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Trial record 13 of 52 for:    rett

Treatment of Rett Syndrome With Recombinant Human IGF-1

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01777542
Recruitment Status : Completed
First Posted : January 29, 2013
Results First Posted : March 26, 2018
Last Update Posted : March 26, 2018
Sponsor:
Collaborator:
International Rett Syndrome Foundation
Information provided by (Responsible Party):
Mustafa Sahin, Harvard Medical School

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Rett Syndrome
Interventions Drug: Recombinant Human Insulin Growth Factor 1 (rhIGF-1)
Drug: Placebo
Enrollment 30

Recruitment Details  
Pre-assignment Details

Assessments findings during the Screening Visit that could result in ineligibility after informed consent include:

  1. Scoliosis curve > 40º by x-ray
  2. Bone age > 11 years by x-ray
  3. Cardiomegaly by echocardiogram
  4. Tanner Stage II breast development by physical exam
  5. Prolonged QTc by ECG
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo). One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Period Title: First Intervention (20 Weeks)
Started 15 15
Completed 15 15
Not Completed 0 0
Period Title: Washout Period (28 +/- 2 Weeks)
Started 15 15
Completed 15 15
Not Completed 0 0
Period Title: Second Intervention (20 Weeks)
Started 15 15
Completed 15 14
Not Completed 0 1
Reason Not Completed
Adverse Event             0             1
Arm/Group Title All Participants
Hide Arm/Group Description All 30 subjects enrolled and randomized in the study
Overall Number of Baseline Participants 30
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants
<=18 years
30
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants
Female
30
 100.0%
Male
0
   0.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants
Hispanic or Latino
26
  86.7%
Not Hispanic or Latino
3
  10.0%
Unknown or Not Reported
1
   3.3%
1.Primary Outcome
Title Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale
Hide Description

The RSBQ is an informant/parent-completed measure of abnormal behaviors typically observed in individuals with RTT, which is completed by a parent/caregiver/LAR. Each item, grouped into eight domains/factors: General mood, Breathing problems, Body rocking and expressionless face, Hand behaviors, Repetitive face movements, Night-time behaviors, Fear/anxiety and Walking/standing), is scored on a Likert scale of 0-2, according to how well the item describes the individual’s behavior. A score of “0” indicates the described item is “not true,” a score of “1” indicates the described item is “somewhat or sometimes true,” and a score of “2” indicates the described item is “very true or often true.”

The total sum of items in each subscale is reported.

For the fear/anxiety subscale, the sum total could be between 0-8. The higher the sum total score, the greater the frequency of fear/anxiety behaviors.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 6.00)
5.00
(2.00 to 6.00)
Visit 2 - First Intervention Number Analyzed 15 participants 15 participants
5.00
(3.00 to 6.00)
3.00
(2.00 to 5.00)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 5.00)
3.00
(2.00 to 5.00)
Visit 4 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(2.00 to 5.00)
3.00
(1.00 to 6.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
3.00
(2.00 to 5.00)
3.00
(1.00 to 5.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(2.00 to 5.00)
4.00
(2.00 to 6.00)
Visit 7 - Second Intervention Number Analyzed 14 participants 15 participants
4.00
(2.00 to 4.00)
3.00
(2.00 to 5.00)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
3.00
(3.00 to 5.00)
4.00
(1.00 to 5.00)
Visit 9 - Second Intervention Number Analyzed 15 participants 15 participants
3.00
(2.00 to 4.00)
3.00
(1.00 to 5.00)
Visit 10 - Second Intervention Number Analyzed 14 participants 14 participants
4.00
(2.00 to 5.00)
3.00
(2.00 to 4.00)
Follow-up Number Analyzed 14 participants 14 participants
3.50
(2.00 to 5.00)
3.50
(2.00 to 5.00)
2.Primary Outcome
Title Anxiety, Depression, and Mood Scale (ADAMS) - Social Avoidance Subscale
Hide Description

The ADAMS is completed by the parent/caregiver/LAR and consists of 29 items which are scored on a 4-point rating scale that combines frequency and severity ratings. The instructions ask the rater to describe the individual's behavior over the last six months on the following scale: "0" if the behavior has not occurred, "1" if the behavior occurs occasionally or is a mild problem, "2" if the behavior occurs quite often or is moderate problem, or "3" if the behavior occurs a lot or is a severe problem.

The Social Avoidance subscale of the ADAMS will be used as a primary outcome measure for this trial. The range for this subscale is 0-21. The higher the subscale score, the more problematic the behavior.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
6.00
(5.00 to 8.00)
4.00
(2.00 to 8.00)
Visit 2 - First Intervention Number Analyzed 15 participants 15 participants
5.00
(3.00 to 7.00)
5.00
(3.00 to 7.00)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
5.00
(4.00 to 7.00)
4.00
(2.00 to 7.00)
Visit 4 - First Intervention Number Analyzed 15 participants 15 participants
6.00
(3.00 to 7.00)
4.00
(3.00 to 7.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
5.00
(2.00 to 6.00)
3.00
(2.00 to 5.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 5.00)
4.00
(3.00 to 6.00)
Visit 7 - Second Intervention Number Analyzed 14 participants 15 participants
4.00
(2.00 to 7.00)
4.00
(3.00 to 5.00)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(2.00 to 6.00)
4.00
(3.00 to 5.00)
Visit 9 - Second Intervention Number Analyzed 15 participants 15 participants
3.00
(1.00 to 7.00)
3.00
(2.00 to 5.00)
Visit 10 - Second Intervention Number Analyzed 14 participants 14 participants
3.50
(2.00 to 5.00)
3.50
(2.00 to 5.00)
Follow-up Number Analyzed 14 participants 14 participants
4.00
(2.00 to 6.00)
3.00
(2.00 to 4.00)
3.Primary Outcome
Title Clinical Global Impression – Severity (CGI-S)
Hide Description

This scale is used to judge the severity of the subject’s disease prior to entry into the study. The clinician will rate the severity of behavioral symptoms at baseline on a 7-point scale from not impaired to the most impaired.

The scores that correspond to each possible grouping are as follows: 1=Normal, not at all impaired; 2=Borderline impaired; 3=Mildly impaired; 4=Moderately impaired; 5=Markedly impaired; 6=Severely impaired; 7=The most impaired.

The possible range for reported scores is 1-7.

Time Frame Every 10 weeks during each of the two 20-week treatment periods
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
4.00
(3.00 to 4.00)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 4.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
4.00
(4.00 to 5.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 5.00)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 5.00)
Visit 10 - Second Intervention Number Analyzed 15 participants 14 participants
4.00
(4.00 to 4.00)
4.50
(4.00 to 5.00)
4.Primary Outcome
Title Clinical Global Impression – Improvement (CGI-I)
Hide Description

Each time the patient was seen after the study intervention was initiated, the clinician compared the patient's overall clinical condition to the CGI-S score obtained at the baseline (visit 1) visit. Based on information collected, the clinician determined if any improvement occurred on the following 7-point scale: 1=Very much improved since the initiation of treatment; 2=Much improved; 3=Minimally improved; 4=No change from baseline (the initiation of treatment); 5=Minimally worse; 6=Much worse; 7=Very much worse since the initiation of treatment.

The possible range for reported scores is 1-7.

Time Frame Every 10 weeks during each of the two 20-week treatment periods
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

Data was not collected at Visit 1 because participants had not yet been exposed to either intervention.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
4.00
(4.00 to 4.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
4.00
(4.00 to 4.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 5.00)
4.00
(4.00 to 5.00)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
4.00
(4.00 to 5.00)
Visit 10 - Second Intervention Number Analyzed 15 participants 14 participants
4.00
(3.00 to 4.00)
4.00
(3.00 to 5.00)
5.Primary Outcome
Title Parental Global Impression – Severity (PGI-S)
Hide Description

The PGI-S is the parent version of the CGI-S. Parents/caregivers/LAR are asked to rate the severity of their child’s symptoms at baseline on a 7-point scale from not at all impaired to the most impaired. The parents/caregivers/LAR will complete the PGI-S at each study visit.

The scores that correspond to each possible grouping are as follows:

1=Normal, not at all impaired; 2=Borderline impaired; 3=Mildly impaired; 4=Moderately impaired; 5=Markedly impaired; 6=Severely impaired; 7=The most impaired.

The possible range for reported scores is 1-7.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 4.00)
6.00
(4.00 to 6.00)
Visit 2 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 6.00)
4.00
(4.00 to 6.00)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 6.00)
Visit 4 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
4.00
(4.00 to 6.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 6.00)
4.00
(4.00 to 6.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 6.00)
4.00
(4.00 to 6.00)
Visit 7 - Second Intervention Number Analyzed 14 participants 15 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 6.00)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
6.00
(4.00 to 6.00)
Visit 9 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(4.00 to 4.00)
6.00
(4.00 to 6.00)
Visit 10 - Second Intervention Number Analyzed 14 participants 14 participants
4.00
(3.00 to 4.00)
5.00
(4.00 to 6.00)
Follow-up Number Analyzed 14 participants 14 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 6.00)
6.Primary Outcome
Title Parental Global Impression – Improvement (PGI-I)
Hide Description

As part of each visit after the study intervention was initiated, the parent/caregiver was asked to compare the patient's overall clinical condition to the score obtained at the baseline (visit 1) visit. Based on information collected, the clinician determined if any improvement occurred on the following 7-point scale: 1=Very much improved since the initiation of treatment; 2=Much improved; 3=Minimally improved; 4=No change from baseline (the initiation of treatment); 5=Minimally worse; 6=Much worse; 7=Very much worse since the initiation of treatment.

The possible range for reported scores is 1-7.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

Data was not collected at visit 1 because participants had not yet been exposed to either intervention.

In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 2 - First Intervention Number Analyzed 15 participants 15 participants
4.00
(3.00 to 4.00)
4.00
(4.00 to 4.00)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
3.00
(3.00 to 4.00)
4.00
(3.00 to 4.00)
Visit 4 - First Intervention Number Analyzed 15 participants 15 participants
3.00
(3.00 to 4.00)
4.00
(3.00 to 4.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
3.00
(3.00 to 4.00)
3.00
(3.00 to 4.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
4.00
(2.00 to 4.00)
3.00
(3.00 to 4.00)
Visit 7 - Second Intervention Number Analyzed 14 participants 15 participants
3.00
(3.00 to 4.00)
3.00
(3.00 to 3.00)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
3.00
(3.00 to 4.00)
3.00
(3.00 to 3.00)
Visit 9 - Second Intervention Number Analyzed 15 participants 15 participants
3.00
(3.00 to 5.00)
3.00
(3.00 to 3.00)
Visit 10 - Second Intervention Number Analyzed 14 participants 14 participants
3.00
(3.00 to 3.00)
3.00
(3.00 to 3.00)
Follow-up Number Analyzed 14 participants 14 participants
3.00
(2.00 to 3.00)
3.00
(3.00 to 3.00)
7.Primary Outcome
Title Parent Targeted Visual Analog Scale (PTSVAS) - Scale 1
Hide Description

The parent or caretaker identifies the three most troublesome, RTT-specific, “target” symptoms, such as inattention or breath-holding. This allows the problems that are of concern to parents and the family to be targeted in the trial. In this study the caregiver will choose three target symptoms at baseline and then rate changes in severity of each target symptom on a visual analog scale (VAS).

The VAS is a 10 cm line, where a target symptom is anchored on one end with the description "the best it has ever been" and on the other with the description "the worst it has ever been." The parent was asked to marked on the line where they felt their child's symptoms currently fit best. This mark was measured as recorded as a numeric value from 0.00-10.00 cm. The higher the value, the worse the symptom.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
6.50
(3.25 to 8.40)
8.80
(5.05 to 9.85)
Visit 2 - First Intervention Number Analyzed 13 participants 13 participants
4.70
(3.45 to 7.70)
4.80
(2.30 to 8.95)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
5.65
(3.55 to 8.90)
5.35
(3.80 to 9.35)
Visit 4 - First Intervention Number Analyzed 11 participants 11 participants
5.05
(2.55 to 7.70)
5.10
(4.80 to 9.10)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
4.80
(2.00 to 7.50)
5.15
(2.90 to 9.55)
Visit 6 - Second Intervention Number Analyzed 14 participants 15 participants
4.95
(3.75 to 8.50)
5.20
(4.70 to 9.25)
Visit 7 - Second Intervention Number Analyzed 11 participants 13 participants
4.55
(2.55 to 6.00)
4.65
(3.85 to 7.25)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
5.65
(3.70 to 7.55)
5.00
(3.65 to 7.45)
Visit 9 - Second Intervention Number Analyzed 13 participants 12 participants
4.15
(2.45 to 6.45)
5.15
(3.63 to 8.58)
Visit 10 - Second Intervention Number Analyzed 14 participants 14 participants
4.80
(2.50 to 6.90)
5.05
(4.20 to 8.50)
Follow-up Number Analyzed 9 participants 12 participants
5.60
(1.40 to 6.25)
5.08
(4.13 to 8.13)
8.Primary Outcome
Title Parent Targeted Visual Analog Scale (PTSVAS) - Scale 2
Hide Description

The parent or caretaker identifies the three most troublesome, RTT-specific, “target” symptoms, such as inattention or breath-holding. This allows the problems that are of concern to parents and the family to be targeted in the trial. In this study the caregiver will choose three target symptoms at baseline and then rate changes in severity of each target symptom on a visual analog scale (VAS).

The VAS is a 10 cm line, where a target symptom is anchored on one end with the description "the best it has ever been" and on the other with the description "the worst it has ever been." The parent was asked to marked on the line where they felt their child's symptoms currently fit best. This mark was measured as recorded as a numeric value from 0.00-10.00 cm. The higher the value, the worse the symptom.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention
7.75
(4.80 to 9.45)
6.35
(5.20 to 9.85)
Visit 2 - First Intervention
4.50
(3.75 to 5.00)
5.25
(4.85 to 8.40)
Visit 3 - First Intervention
5.85
(4.30 to 7.25)
5.95
(4.95 to 8.90)
Visit 4 - First Intervention
5.00
(3.05 to 6.20)
5.40
(4.90 to 6.45)
Visit 5 - First Intervention
5.00
(3.30 to 6.10)
5.45
(1.40 to 7.80)
Visit 6 - Second Intervention
5.35
(4.95 to 7.85)
7.10
(4.95 to 9.25)
Visit 7 - Second Intervention
5.50
(3.85 to 6.20)
5.85
(3.40 to 6.90)
Visit 8 - Second Intervention
5.15
(3.80 to 7.20)
5.00
(3.00 to 7.60)
Visit 9 - Second Intervention
3.80
(1.70 to 6.35)
5.13
(3.43 to 7.05)
Visit 10 - Second Intervention
4.90
(2.60 to 6.80)
4.95
(2.25 to 8.15)
Follow-up
5.15
(3.85 to 6.65)
5.20
(3.60 to 7.95)
9.Primary Outcome
Title Parent Targeted Visual Analog Scale (PTSVAS) - Scale 3
Hide Description

The parent or caretaker identifies the three most troublesome, RTT-specific, “target” symptoms, such as inattention or breath-holding. This allows the problems that are of concern to parents and the family to be targeted in the trial. In this study the caregiver will choose three target symptoms at baseline and then rate changes in severity of each target symptom on a visual analog scale (VAS).

The VAS is a 10 cm line, where a target symptom is anchored on one end with the description "the best it has ever been" and on the other with the description "the worst it has ever been." The parent was asked to marked on the line where they felt their child's symptoms currently fit best. This mark was measured as recorded as a numeric value from 0.00-10.00 cm. The higher the value, the worse the symptom.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
7.85
(5.70 to 8.70)
5.70
(4.50 to 9.65)
Visit 2 - First Intervention Number Analyzed 12 participants 13 participants
4.70
(4.48 to 8.30)
5.00
(2.55 to 5.40)
Visit 3 - First Intervention Number Analyzed 15 participants 15 participants
5.65
(3.65 to 9.00)
5.20
(3.50 to 9.40)
Visit 4 - First Intervention Number Analyzed 11 participants 11 participants
4.15
(2.90 to 6.05)
5.35
(3.50 to 8.65)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
5.00
(4.40 to 7.40)
5.10
(4.00 to 8.20)
Visit 6 - Second Intervention Number Analyzed 14 participants 15 participants
6.20
(4.70 to 8.35)
5.35
(4.05 to 9.05)
Visit 7 - Second Intervention Number Analyzed 11 participants 13 participants
4.80
(3.15 to 7.30)
4.95
(2.95 to 7.05)
Visit 8 - Second Intervention Number Analyzed 15 participants 15 participants
4.85
(3.80 to 8.15)
5.15
(1.75 to 7.10)
Visit 9 - Second Intervention Number Analyzed 13 participants 12 participants
4.60
(3.15 to 7.90)
5.25
(3.35 to 7.10)
Visit 10 - Second Intervention Number Analyzed 14 participants 14 participants
4.13
(1.10 to 8.45)
4.55
(2.95 to 5.45)
Follow-up Number Analyzed 9 participants 12 participants
4.55
(2.20 to 7.00)
5.10
(3.20 to 8.03)
10.Primary Outcome
Title Kerr Clinical Severity Scale
Hide Description

The Kerr clinical severity scale (Kerr scale) is a quantitative measure of global disease severity. The Kerr scale is a summation of individual items related to Rett syndrome phenotypic characteristics. The items are based on the severity or degree of abnormality of each characteristic on a discrete scale (0, 1, 2) with the highest level corresponding to the most severe or most abnormal presentations.

The possible range of scores is 0-48. The higher the score, the more severe the symptoms.

Time Frame At the start and end of each 20-week treatment period
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention Number Analyzed 15 participants 15 participants
16.50
(13.00 to 20.00)
18.00
(12.00 to 22.00)
Visit 5 - First Intervention Number Analyzed 15 participants 15 participants
15.00
(13.00 to 21.00)
18.00
(14.00 to 22.00)
Visit 6 - Second Intervention Number Analyzed 15 participants 15 participants
15.00
(12.00 to 20.00)
19.00
(16.00 to 22.00)
Visit 10 - Second Intervention Number Analyzed 15 participants 14 participants
14.00
(13.00 to 22.00)
20.00
(17.00 to 22.00)
11.Secondary Outcome
Title Rett Syndrome Behavior Questionnaire (RSBQ)
Hide Description

The RSBQ is a parent-completed measure of abnormal behaviors typically observed in individuals with RTT. Each item, grouped into eight subscales, is scored on a Likert scale of 0-2, according to how well the item describes the individual’s behavior. A score of “0” indicates the described item is “not true,” a score of “1” indicates the described item is “somewhat or sometimes true,” and a score of “2” indicates the described item is “very true or often true.”

The total sum of each subscale is reported. The higher the score, the more severe the symptoms of that subscale in the participant.

The range for each subscale is as follows:

General Mood: 0-16 Body rocking and expressionless face: 0-14 Hand behaviors: 0-12 Breathing Problems: 0-10 Repetitive Face Movements: 0-8 Night-time behaviors: 0-6 Walking Standing: 0-4

The fear/anxiety subscale was used as a primary outcome measure in this study and results can be found in that section.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1- First Intervention: General Mood Number Analyzed 15 participants 15 participants
7.00
(4.00 to 11.00)
4.00
(3.00 to 7.00)
Visit 2- First Intervention: General Mood Number Analyzed 15 participants 15 participants
5.00
(3.00 to 8.00)
3.00
(1.00 to 4.00)
Visit 3- First Intervention: General Mood Number Analyzed 15 participants 15 participants
6.00
(1.00 to 8.00)
2.00
(1.00 to 4.00)
Visit 4- First Intervention: General Mood Number Analyzed 15 participants 15 participants
5.00
(3.00 to 8.00)
2.00
(1.00 to 5.00)
Visit 5- First Intervention: General Mood Number Analyzed 15 participants 15 participants
5.00
(1.00 to 8.00)
3.00
(1.00 to 6.00)
Visit 6- Second Intervention: General Mood Number Analyzed 15 participants 15 participants
4.00
(2.00 to 8.00)
4.00
(2.00 to 6.00)
Visit 7- Second Intervention: General Mood Number Analyzed 14 participants 15 participants
5.50
(2.00 to 7.00)
2.00
(1.00 to 5.00)
Visit 8- Second Intervention: General Mood Number Analyzed 15 participants 15 participants
5.00
(2.00 to 8.00)
2.00
(1.00 to 4.00)
Visit 9- Second Intervention: General Mood Number Analyzed 15 participants 15 participants
6.00
(3.00 to 8.00)
1.00
(1.00 to 5.00)
Visit 10- Second Intervention: General Mood Number Analyzed 14 participants 14 participants
4.00
(2.00 to 8.00)
2.50
(1.00 to 6.00)
Follow-up: General Mood Number Analyzed 14 participants 14 participants
5.50
(3.00 to 6.00)
2.00
(1.00 to 4.00)
Visit 1- First Intervention: Body Rocking Number Analyzed 15 participants 15 participants
6.00
(5.00 to 10.00)
4.00
(4.00 to 6.00)
Visit 2- First Intervention: Body Rocking Number Analyzed 15 participants 15 participants
5.00
(4.00 to 7.00)
4.00
(3.00 to 6.00)
Visit 3- First Intervention: Body Rocking Number Analyzed 15 participants 15 participants
5.00
(3.00 to 8.00)
3.00
(2.00 to 6.00)
Visit 4- First Intervention: Body Rocking Number Analyzed 15 participants 15 participants
6.00
(4.00 to 7.00)
4.00
(2.00 to 5.00)
Visit 5- First Intervention: Body Rocking Number Analyzed 15 participants 15 participants
5.00
(3.00 to 8.00)
4.00
(2.00 to 5.00)
Visit 6- Second Intervention: Body Rocking Number Analyzed 15 participants 15 participants
4.00
(3.00 to 7.00)
4.00
(3.00 to 5.00)
Visit 7- Second Intervention: Body Rocking Number Analyzed 14 participants 15 participants
5.00
(3.00 to 7.00)
3.00
(3.00 to 5.00)
Visit 8- Second Intervention: Body Rocking Number Analyzed 15 participants 15 participants
5.00
(3.00 to 6.00)
4.00
(2.00 to 5.00)
Visit 9- Second Intervention: Body Rocking Number Analyzed 15 participants 15 participants
4.00
(3.00 to 7.00)
3.00
(2.00 to 5.00)
Visit 10- Second Intervention: Body Rocking Number Analyzed 14 participants 14 participants
5.00
(3.00 to 7.00)
4.00
(2.00 to 5.00)
Followup: Body Rocking Number Analyzed 14 participants 14 participants
4.50
(3.00 to 7.00)
4.00
(2.00 to 5.00)
Visit 1- First Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
8.00
(7.00 to 10.00)
8.00
(5.00 to 10.00)
Visit 2- First Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
9.00
(6.00 to 9.00)
8.00
(6.00 to 10.00)
Visit 3- First Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
8.00
(7.00 to 10.00)
8.00
(5.00 to 9.00)
Visit 4- First Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
8.00
(6.00 to 9.00)
9.00
(6.00 to 9.00)
Visit 5- First Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
8.00
(6.00 to 10.00)
9.00
(5.00 to 10.00)
Visit 6- Second Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
9.00
(6.00 to 10.00)
8.00
(7.00 to 10.00)
Visit 7- Second Intervention: Hand Behaviors Number Analyzed 14 participants 15 participants
8.00
(5.00 to 10.00)
9.00
(6.00 to 9.00)
Visit 8- Second Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
8.00
(6.00 to 9.00)
9.00
(6.00 to 9.00)
Visit 9- Second Intervention: Hand Behaviors Number Analyzed 15 participants 15 participants
8.00
(5.00 to 9.00)
7.00
(6.00 to 10.00)
Visit 10- Second Intervention: Hand Behaviors Number Analyzed 14 participants 14 participants
7.00
(6.00 to 9.00)
9.00
(6.00 to 10.00)
Follow-up: Hand Behaviors Number Analyzed 14 participants 14 participants
7.50
(7.00 to 9.00)
8.50
(6.00 to 10.00)
Visit 1- First Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
6.00
(3.00 to 9.00)
4.00
(3.00 to 7.00)
Visit 2- First Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
4.00
(2.00 to 8.00)
4.00
(2.00 to 6.00)
Visit 3- First Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
5.00
(2.00 to 8.00)
4.00
(2.00 to 6.00)
Visit 4- First Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
5.00
(3.00 to 7.00)
5.00
(2.00 to 7.00)
Visit 5- First Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
5.00
(1.00 to 8.00)
4.00
(2.00 to 7.00)
Visit 6- Second Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
6.00
(2.00 to 9.00)
4.00
(1.00 to 8.00)
Visit 7- Second Intervention: Breathing Problems Number Analyzed 14 participants 15 participants
4.50
(2.00 to 7.00)
3.00
(1.00 to 7.00)
Visit 8- Second Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
6.00
(3.00 to 7.00)
3.00
(1.00 to 6.00)
Visit 9- Second Intervention: Breathing Problems Number Analyzed 15 participants 15 participants
5.00
(2.00 to 7.00)
3.00
(0.00 to 5.00)
Visit 10- Second Intervention: Breathing Problems Number Analyzed 14 participants 14 participants
6.00
(3.00 to 7.00)
4.00
(1.00 to 5.00)
Follow-up: Breathing Problems Number Analyzed 14 participants 14 participants
5.00
(2.00 to 7.00)
3.00
(0.00 to 4.00)
Visit 1- First Intervention: Repetitive Face Movem Number Analyzed 15 participants 15 participants
2.00
(1.00 to 5.00)
2.00
(2.00 to 4.00)
Visit 2- First Intervention: Repetitive Face Movem Number Analyzed 15 participants 15 participants
2.00
(0.00 to 4.00)
2.00
(2.00 to 4.00)
Visit 3- First Intervention: Repetitive Face Movem Number Analyzed 15 participants 15 participants
3.00
(1.00 to 4.00)
3.00
(2.00 to 4.00)
Visit 4- First Intervention: Repetitive Face Movem Number Analyzed 15 participants 15 participants
2.00
(1.00 to 5.00)
2.00
(1.00 to 4.00)
Visit 5- First Intervention: Repetitive Face Movem Number Analyzed 15 participants 15 participants
3.00
(0.00 to 5.00)
2.00
(0.00 to 4.00)
Visit 6- Second Intervention: Repetitive Face Mov Number Analyzed 15 participants 15 participants
3.00
(2.00 to 5.00)
3.00
(2.00 to 4.00)
Visit 7- Second Intervention: Repetitive Face Mov Number Analyzed 14 participants 15 participants
3.00
(1.00 to 5.00)
2.00
(0.00 to 4.00)
Visit 8- Second Intervention: Repetitive Face Mov Number Analyzed 15 participants 15 participants
3.00
(2.00 to 4.00)
2.00
(1.00 to 4.00)
Visit 9- Second Intervention: Repetitive Face Mov Number Analyzed 15 participants 15 participants
3.00
(1.00 to 5.00)
2.00
(0.00 to 3.00)
Visit 10- Second Intervention: Repetitive Face Mov Number Analyzed 14 participants 14 participants
3.00
(1.00 to 5.00)
1.50
(1.00 to 4.00)
Follow-up: Repetitive Face Movements Number Analyzed 14 participants 14 participants
2.00
(1.00 to 4.00)
2.00
(0.00 to 2.00)
Visit 1- First Intervention: Night time Behaviors Number Analyzed 15 participants 15 participants
0.00
(0.00 to 2.00)
1.00
(0.00 to 2.00)
Visit 2- First Intervention: Night time Behaviors Number Analyzed 15 participants 15 participants
0.00
(0.00 to 3.00)
1.00
(0.00 to 2.00)
Visit 3- First Intervention: Night time Behaviors Number Analyzed 15 participants 15 participants
0.00
(0.00 to 0.00)
0.00
(0.00 to 2.00)
Visit 4- First Intervention: Night time Behaviors Number Analyzed 15 participants 15 participants
0.00
(0.00 to 2.00)
0.00
(0.00 to 1.00)
Visit 5- First Intervention: Night time Behaviors Number Analyzed 15 participants 15 participants
0.00
(0.00 to 2.00)
1.00
(0.00 to 2.00)
Visit 6- Second Intervention: Night time Behavior Number Analyzed 15 participants 15 participants
0.00
(0.00 to 2.00)
1.00
(0.00 to 2.00)
Visit 7- Second Intervention: Night time Behavior Number Analyzed 14 participants 15 participants
0.00
(0.00 to 2.00)
0.00
(0.00 to 1.00)
Visit 8- Second Intervention: Night time Behavior Number Analyzed 15 participants 15 participants
1.00
(0.00 to 2.00)
0.00
(0.00 to 1.00)
Visit 9- Second Intervention: Night time Behavior Number Analyzed 15 participants 15 participants
0.00
(0.00 to 3.00)
0.00
(0.00 to 1.00)
Visit 10- Second Intervention: Night time Behavior Number Analyzed 14 participants 14 participants
0.00
(0.00 to 1.00)
0.00
(0.00 to 1.00)
Follow-up: Night time Behaviors Number Analyzed 14 participants 14 participants
0.00
(0.00 to 1.00)
0.00
(0.00 to 1.00)
Visit 1- First Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(2.00 to 3.00)
2.00
(1.00 to 3.00)
Visit 2- First Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(1.00 to 3.00)
2.00
(1.00 to 3.00)
Visit 3- First Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(2.00 to 2.00)
2.00
(1.00 to 3.00)
Visit 4- First Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(1.00 to 3.00)
2.00
(1.00 to 3.00)
Visit 5- First Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(1.00 to 3.00)
2.00
(1.00 to 3.00)
Visit 6- Second Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(2.00 to 3.00)
2.00
(1.00 to 2.00)
Visit 7- Second Intervention: Walking/Standing Number Analyzed 14 participants 15 participants
2.00
(2.00 to 3.00)
2.00
(1.00 to 3.00)
Visit 8- Second Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
2.00
(1.00 to 3.00)
2.00
(1.00 to 3.00)
Visit 9- Second Intervention: Walking/Standing Number Analyzed 15 participants 15 participants
3.00
(1.00 to 3.00)
2.00
(1.00 to 2.00)
Visit 10- Second Intervention: Walking/Standing Number Analyzed 14 participants 14 participants
1.50
(1.00 to 3.00)
2.00
(1.00 to 2.00)
Follow-up: Walking/Standing Number Analyzed 14 participants 14 participants
2.00
(1.00 to 3.00)
2.00
(2.00 to 3.00)
12.Secondary Outcome
Title Anxiety, Depression, and Mood Scale (ADAMS)
Hide Description

Remaining subscales of the ADAMS that are not primary outcome measures include: Manic/hyperactive, Depressed mood, General anxiety, Obsessive/compulsive behavior.

The range for each subscale is as follows:

Manic/Hyperactive Behavior: 0-15 Depressed Mood: 0-21 General Anxiety: 0-21 Obsessive/Compulsive Behavior: 0-9

The higher the score for each subscale, the more problematic the behavior.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1- First Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
8.00
(5.00 to 11.00)
7.00
(5.00 to 9.00)
Visit 2- First Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
7.00
(3.00 to 10.00)
7.00
(4.00 to 9.00)
Visit 3- First Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
7.00
(3.00 to 9.00)
6.00
(4.00 to 7.00)
Visit 4- First Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
7.00
(5.00 to 9.00)
5.00
(4.00 to 7.00)
Visit 5- First Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
7.00
(4.00 to 9.00)
4.00
(3.00 to 7.00)
Visit 6- Second Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
8.00
(3.00 to 9.00)
6.00
(5.00 to 8.00)
Visit 7- Second Intervention: Manic/Hyperactive Number Analyzed 14 participants 15 participants
6.50
(3.00 to 9.00)
5.00
(3.00 to 7.00)
Visit 8- Second Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
6.00
(3.00 to 8.00)
5.00
(3.00 to 6.00)
Visit 9- Second Intervention: Manic/Hyperactive Number Analyzed 15 participants 15 participants
6.00
(4.00 to 9.00)
4.00
(1.00 to 6.00)
Visit 10- First Intervention: Manic/Hyperactive Number Analyzed 14 participants 14 participants
5.00
(2.00 to 7.00)
4.50
(2.00 to 7.00)
Follow-up: Manic/Hyperactive Subscale Number Analyzed 14 participants 14 participants
5.00
(3.00 to 9.00)
5.00
(3.00 to 7.00)
Visit 1- First Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
2.00
(2.00 to 7.00)
4.00
(3.00 to 6.00)
Visit 2- First Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
4.00
(1.00 to 5.00)
5.00
(2.00 to 6.00)
Visit 3- First Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
3.00
(1.00 to 5.00)
3.00
(2.00 to 5.00)
Visit 4- First Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
2.00
(1.00 to 4.00)
3.00
(1.00 to 5.00)
Visit 5- First Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
2.00
(1.00 to 4.00)
4.00
(0.00 to 5.00)
Visit 6- Second Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
2.00
(0.00 to 5.00)
4.00
(3.00 to 6.00)
Visit 7- Second Intervention: Depressed Mood Number Analyzed 14 participants 15 participants
3.00
(0.00 to 6.00)
3.00
(2.00 to 5.00)
Visit 8- Second Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
2.00
(1.00 to 5.00)
3.00
(1.00 to 5.00)
Visit 9- Second Intervention: Depressed Mood Number Analyzed 15 participants 15 participants
3.00
(0.00 to 4.00)
2.00
(1.00 to 5.00)
Visit 10- Second Intervention: Depressed Mood Number Analyzed 14 participants 14 participants
2.00
(1.00 to 4.00)
3.00
(2.00 to 4.00)
Follow-up: Depressed Mood Subscale Number Analyzed 14 participants 14 participants
2.00
(0.00 to 5.00)
3.50
(0.00 to 4.00)
Visit 1- First Intervention: General Anxiety Number Analyzed 15 participants 15 participants
8.00
(5.00 to 12.00)
6.00
(5.00 to 12.00)
Visit 2- First Intervention: General Anxiety Number Analyzed 15 participants 15 participants
6.00
(4.00 to 12.00)
7.00
(3.00 to 10.00)
Visit 3- First Intervention: General Anxiety Number Analyzed 15 participants 15 participants
6.00
(3.00 to 9.00)
6.00
(3.00 to 9.00)
Visit 4- First Intervention: General Anxiety Number Analyzed 15 participants 15 participants
5.00
(2.00 to 11.00)
5.00
(2.00 to 8.00)
Visit 5- First Intervention: General Anxiety Number Analyzed 15 participants 15 participants
5.00
(2.00 to 10.00)
5.00
(3.00 to 7.00)
Visit 6- Second Intervention: General Anxiety Number Analyzed 15 participants 15 participants
6.00
(3.00 to 9.00)
7.00
(3.00 to 11.00)
Visit 7- Second Intervention: General Anxiety Number Analyzed 14 participants 15 participants
6.00
(2.00 to 8.00)
5.00
(1.00 to 7.00)
Visit 8- Second Intervention: General Anxiety Number Analyzed 15 participants 15 participants
6.00
(3.00 to 9.00)
4.00
(2.00 to 8.00)
Visit 9- Second Intervention: General Anxiety Number Analyzed 15 participants 15 participants
4.00
(2.00 to 8.00)
3.00
(1.00 to 6.00)
Visit 10- Second Intervention: General Anxiety Number Analyzed 14 participants 14 participants
4.00
(2.00 to 7.00)
4.00
(2.00 to 5.00)
Follow-up: General Anxiety Subscale Number Analyzed 14 participants 14 participants
5.50
(3.00 to 9.00)
4.00
(1.00 to 6.00)
Visit 1- First Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
4.00
(3.00 to 6.00)
3.00
(0.00 to 5.00)
Visit 2- First Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
4.00
(2.00 to 4.00)
4.00
(1.00 to 4.00)
Visit 3- First Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
4.00
(1.00 to 4.00)
4.00
(1.00 to 4.00)
Visit 4- First Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
3.00
(3.00 to 4.00)
3.00
(1.00 to 4.00)
Visit 5- First Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
3.00
(2.00 to 4.00)
3.00
(0.00 to 4.00)
Visit 6- Second Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
3.00
(1.00 to 5.00)
3.00
(1.00 to 5.00)
Visit 7- Second Intervention: Obsessive Compulsive Number Analyzed 14 participants 15 participants
3.00
(2.00 to 6.00)
3.00
(0.00 to 4.00)
Visit 8- Second Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
3.00
(2.00 to 4.00)
3.00
(0.00 to 4.00)
Visit 9- Second Intervention: Obsessive Compulsive Number Analyzed 15 participants 15 participants
3.00
(1.00 to 3.00)
2.00
(0.00 to 4.00)
Visit 10- First Intervention: Obsessive Compulsive Number Analyzed 14 participants 14 participants
2.00
(1.00 to 4.00)
2.50
(0.00 to 5.00)
Follow-up: Obsessive Compulsive Behavior Subscale Number Analyzed 14 participants 14 participants
3.50
(2.00 to 4.00)
3.00
(0.00 to 4.00)
13.Secondary Outcome
Title Mullen Scales of Early Learning (MSEL)
Hide Description

The MSEL is a standardized developmental test for children ages 3 to 68 months consisting of five subscales: gross motor, fine motor, visual reception, expressive language, and receptive language.

The raw score is reported for each subscale domain. The potential score ranges are as follows:

Visual Reception: 33 items, score range=0-50, Fine Motor: 30 items, score range= 0-49, Receptive Language: 33 items, score range= 0-48, Expressive Language: 28 items, score range= 0-50. The gross motor subscale was not included in this population.

A higher raw score indicates more advanced abilities in that section.

Time Frame At the start and end of each 20-week treatment period
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

In some cases, the number of participants analyzed is less than 15 for each group due to subject's inability or unwillingness to complete the testing.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1- First Intervention: Visual Reception Number Analyzed 15 participants 15 participants
17.00
(15.00 to 25.00)
26.00
(18.00 to 42.00)
Visit 5- First Intervention: Visual Reception Number Analyzed 15 participants 14 participants
26.00
(16.00 to 33.00)
39.50
(20.00 to 49.00)
Visit 6- Second Intervention: Visual Reception Number Analyzed 15 participants 15 participants
23.00
(20.00 to 36.00)
42.00
(23.00 to 48.00)
Visit 10: Visual Reception Raw Score Number Analyzed 15 participants 13 participants
28.00
(25.00 to 41.00)
44.00
(28.00 to 48.00)
Visit 1- First Intervention: Fine Motor Number Analyzed 15 participants 15 participants
10.00
(7.00 to 14.00)
7.00
(6.00 to 17.00)
Visit 5- First Intervention: Fine Motor Number Analyzed 15 participants 15 participants
9.00
(6.00 to 13.00)
7.00
(3.00 to 15.00)
Visit 6- Second Intervention: Fine Motor Number Analyzed 15 participants 15 participants
11.00
(7.00 to 13.00)
10.00
(5.00 to 17.00)
Visit 10- Second Intervention: Fine Motor Number Analyzed 15 participants 14 participants
9.00
(6.00 to 13.00)
8.50
(4.00 to 13.00)
Visit 1- First Intervention: Receptive Language Number Analyzed 15 participants 14 participants
20.00
(17.00 to 31.00)
25.50
(17.00 to 40.00)
Visit 5- First Intervention: Receptive Language Number Analyzed 15 participants 15 participants
30.00
(19.00 to 33.00)
32.00
(25.00 to 44.00)
Visit 6- Second Intervention: Receptive Language Number Analyzed 15 participants 15 participants
31.00
(24.00 to 35.00)
38.00
(33.00 to 47.00)
Visit 10- Second Intervention: Receptive Language Number Analyzed 15 participants 14 participants
31.00
(26.00 to 38.00)
36.50
(27.00 to 44.00)
Visit 1- First Intervention: Expressive Language Number Analyzed 15 participants 15 participants
8.00
(5.00 to 9.00)
9.00
(6.00 to 11.00)
Visit 5- First Intervention: Expressive Language Number Analyzed 15 participants 15 participants
9.00
(5.00 to 11.00)
8.00
(5.00 to 11.00)
Visit 6- Second Intervention: Expressive Language Number Analyzed 15 participants 15 participants
6.00
(5.00 to 9.00)
10.00
(5.00 to 12.00)
Visit 10- Second Intervention: Expressive Language Number Analyzed 15 participants 14 participants
8.00
(5.00 to 10.00)
8.00
(6.00 to 12.00)
14.Secondary Outcome
Title Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
Hide Description

The VABS-II is a survey designed to assess personal and social functioning. Within each domain (Communication, Daily Living Skills, Socialization, and Motor Skills), items can given a score of "2" if the participant successfully performs the activity usually; a "1" if the participant successfully performs the activity sometimes, or needs reminders; a "0" if the participant never performs the activity, and a "DK" if the parent/caregiver is unsure of the participant's ability for an item.

The raw scores in each sub-domain are reported and the ranges for these are as follows: [Communication Domain], Receptive Language=0-40, Expressive Language=0-108, Written Language=0-50; [Daily Living Skills Domain], Personal=0-82, Domestic=0-48, Community=0-88; [Socialization Domain], Interpersonal Relationships=0-76, Play and Leisure Time=0-62, Coping Skills=0-60; [Motor Skills Domain]: Gross Motor Skills=0-80, Fine Motor Skills=0-72.

A higher score indicates more advanced abilities.

Time Frame At the start and end of each 20-week treatment period
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.
Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention: Receptive Number Analyzed 15 participants 15 participants
13.00
(9.00 to 16.00)
18.00
(13.00 to 20.00)
Visit 5 - First Intervention: Receptive Number Analyzed 15 participants 15 participants
15.00
(14.00 to 20.00)
21.00
(17.00 to 23.00)
Visit 6 - Second Intervention: Receptive Language Number Analyzed 15 participants 14 participants
18.00
(13.00 to 20.00)
22.00
(20.00 to 24.00)
Visit 10 - Second Intervention: Receptive Language Number Analyzed 15 participants 14 participants
20.00
(15.00 to 24.00)
24.50
(20.00 to 29.00)
Visit 1 - First Intervention: Expressive Number Analyzed 15 participants 15 participants
16.00
(12.00 to 17.00)
18.00
(13.00 to 25.00)
Visit 5 - First Intervention: Expressive Number Analyzed 15 participants 15 participants
17.00
(13.00 to 23.00)
22.00
(18.00 to 29.00)
Visit 6 - Second Intervention: Expressive Lang. Number Analyzed 15 participants 14 participants
18.00
(11.00 to 23.00)
25.00
(18.00 to 31.00)
Visit 10 - Second Intervention: Expressive Lang. Number Analyzed 15 participants 14 participants
20.00
(15.00 to 28.00)
24.00
(23.00 to 34.0)
Visit 1 - First Intervention: Written Number Analyzed 15 participants 15 participants
0.00
(0.00 to 1.00)
4.00
(0.00 to 7.00)
Visit 5 - First Intervention: Written Number Analyzed 15 participants 15 participants
0.00
(0.00 to 5.00)
5.00
(0.00 to 9.00)
Visit 6: - Second Intervention Written Language Number Analyzed 15 participants 14 participants
4.00
(1.00 to 7.00)
7.00
(2.00 to 9.00)
Visit 10 - Second Intervention: Written Language Number Analyzed 15 participants 14 participants
6.00
(0.00 to 8.00)
7.00
(4.00 to 10.00)
Visit 1 - First Intervention: Personal Number Analyzed 15 participants 15 participants
9.00
(7.00 to 10.00)
8.00
(6.00 to 10.00)
Visit 5 - First Intervention: Personal Number Analyzed 15 participants 15 participants
10.00
(8.00 to 13.00)
9.00
(7.00 to 13.00)
Visit 6 - Second Intervention: Personal Number Analyzed 15 participants 14 participants
9.00
(8.00 to 14.00)
8.50
(8.00 to 16.00)
Visit 10 - Second Intervention: Personal Number Analyzed 15 participants 14 participants
10.00
(7.00 to 14.00)
9.50
(8.00 to 10.00)
Visit 1 - First Intervention: Domestic Number Analyzed 15 participants 15 participants
0.00
(0.00 to 0.00)
0.00
(0.00 to 1.00)
Visit 5 - First Intervention: Domestic Number Analyzed 15 participants 15 participants
0.00
(0.00 to 0.00)
0.00
(0.00 to 1.00)
Visit 6 - Second Intervention: Domestic Number Analyzed 15 participants 14 participants
0.00
(0.00 to 0.00)
0.00
(0.00 to 2.00)
Visit 10 - Second Intervention: Domestic Number Analyzed 15 participants 14 participants
0.00
(0.00 to 1.00)
0.00
(0.00 to 1.00)
Visit 1 - First Intervention: Community Number Analyzed 15 participants 15 participants
0.00
(0.00 to 1.00)
3.00
(2.00 to 6.00)
Visit 5 - First Intervention: Community Number Analyzed 15 participants 15 participants
1.00
(0.00 to 2.00)
3.00
(2.00 to 4.00)
Visit 6 - Second Intervention: Community Number Analyzed 15 participants 14 participants
1.00
(0.00 to 3.00)
5.00
(2.00 to 7.00)
Visit 10 - Second Intervention: Community Number Analyzed 15 participants 14 participants
2.00
(0.00 to 5.00)
5.00
(2.00 to 7.00)
Visit 1 - First Intervention: Interpersonal Rel. Number Analyzed 15 participants 15 participants
18.00
(15.00 to 21.00)
21.00
(17.00 to 24.00)
Visit 5 - First Intervention: Interpersonal Rel. Number Analyzed 15 participants 15 participants
18.00
(15.00 to 21.00)
22.00
(17.00 to 25.00)
Visit 6 - Second Intervention: Interpersonal Rel. Number Analyzed 15 participants 14 participants
19.00
(15.00 to 21.00)
21.00
(20.00 to 26.00)
Visit 10 - Second Intervention: Interpersonal Rel. Number Analyzed 15 participants 14 participants
20.00
(16.00 to 23.00)
22.50
(19.00 to 27.00)
Visit 1 - First Intervention: Play and Leisure Number Analyzed 15 participants 15 participants
8.00
(7.00 to 12.00)
13.00
(11.00 to 17.00)
Visit 5 - First Intervention: Play and Leisure Number Analyzed 15 participants 15 participants
11.00
(9.00 to 12.00)
12.00
(11.00 to 16.00)
Visit 6 - Second Intervention: Play and Leisure Number Analyzed 15 participants 14 participants
12.00
(9.00 to 14.00)
13.00
(11.00 to 16.00)
Visit 10 - Second Intervention: Play and Leisure Number Analyzed 15 participants 14 participants
11.00
(9.00 to 15.00)
12.50
(10.00 to 16.00)
Visit 1 - First Intervention: Coping Skills Number Analyzed 15 participants 15 participants
3.00
(2.00 to 4.00)
3.00
(3.00 to 4.00)
Visit 5 - First Intervention: Coping Skills Number Analyzed 15 participants 15 participants
2.00
(2.00 to 4.00)
4.00
(2.00 to 6.00)
Visit 6 - Second Intervention: Coping Skills Number Analyzed 15 participants 14 participants
3.00
(3.00 to 4.00)
6.00
(3.00 to 6.00)
Visit 10 - Second Intervention: Coping Skills Number Analyzed 15 participants 14 participants
4.00
(3.00 to 5.00)
4.50
(3.00 to 8.00)
Visit 1 - First Intervention: Gross Motor Number Analyzed 15 participants 15 participants
31.00
(21.00 to 37.00)
10.00
(7.00 to 43.00)
Visit 5 - First Intervention: Gross Motor Number Analyzed 15 participants 15 participants
34.00
(22.00 to 42.00)
10.00
(8.00 to 36.00)
Visit 6 - Second Intervention: Gross Motor Number Analyzed 15 participants 14 participants
27.00
(12.00 to 45.00)
11.50
(8.00 to 34.00)
Visit 10 - Second Intervention: Gross Motor Number Analyzed 15 participants 14 participants
27.00
(14.00 to 46.00)
10.50
(8.00 to 30.00)
Visit 1 - First Intervention: Fine Motor Number Analyzed 15 participants 15 participants
6.00
(3.00 to 8.00)
2.00
(1.00 to 14.00)
Visit 5 - First Intervention: Fine Motor Number Analyzed 15 participants 15 participants
6.00
(4.00 to 9.00)
3.00
(2.00 to 15.00)
Visit 6 - Second Intervention: Fine Motor Number Analyzed 15 participants 14 participants
7.00
(3.00 to 10.00)
4.00
(2.00 to 16.00)
Visit 10 - Second Intervention: Fine Motor Number Analyzed 15 participants 14 participants
5.00
(2.00 to 9.00)
4.00
(2.00 to 8.00)
15.Secondary Outcome
Title Communication and Symbolic Behavior Scales – Developmental Profile (CSBS-DP)
Hide Description

The CSBS-DP was designed to measure early communication and symbolic skills in infants and young children (that is, functional communication skills of 6 month to 2 year olds). The CSBS-DP measures skills from three composites: (a) Social (emotion, eye gaze, and communication); (b) Speech (sounds and words); and (c) Symbolic (understanding and object use) and asks about developmental milestones. The data reported are the composite scores for these three categories.

The possible scores for the three composite categories are as follows:

Social Composite = 0-48; Speech Composite = 0-40; Symbolic Composite = 0-51.

A higher score indicates more advanced abilities in that area.

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention: Social Number Analyzed 15 participants 15 participants
19.00
(16.00 to 25.00)
22.00
(19.00 to 26.00)
Visit 2: Social Composite Score Number Analyzed 15 participants 15 participants
20.00
(15.00 to 23.00)
24.00
(20.00 to 28.00)
Visit 3: Social Composite Score Number Analyzed 15 participants 15 participants
18.00
(13.00 to 25.00)
24.00
(21.00 to 30.00)
Visit 4: Social Composite Score Number Analyzed 15 participants 15 participants
18.00
(17.00 to 25.00)
24.00
(21.00 to 31.00)
Visit 5: Social Composite Score Number Analyzed 15 participants 15 participants
20.00
(15.00 to 29.00)
23.00
(21.00 to 34.00)
Visit 6 - Second Intervention: Social Number Analyzed 15 participants 15 participants
18.00
(15.00 to 28.00)
28.00
(22.00 to 34.00)
Visit 7 - Second Intervention: Social Number Analyzed 14 participants 15 participants
20.00
(16.00 to 26.00)
25.00
(21.00 to 34.00)
Visit 8 - Second Intervention: Social Number Analyzed 15 participants 14 participants
21.00
(17.00 to 27.00)
27.00
(25.00 to 36.00)
Visit 9 - Second Intervention: Social Number Analyzed 15 participants 15 participants
21.00
(17.00 to 29.00)
29.00
(25.00 to 34.00)
Visit 10 - Second Intervention: Social Number Analyzed 14 participants 14 participants
22.50
(19.00 to 30.00)
27.00
(23.00 to 36.00)
Follow-up: Social Composite Score Number Analyzed 14 participants 15 participants
22.50
(18.00 to 25.00)
28.00
(26.00 to 36.00)
Visit 1 - First Intervention: Speech Number Analyzed 15 participants 15 participants
4.00
(2.00 to 8.00)
7.00
(2.00 to 10.00)
Visit 2 - First Intervention: Speech Number Analyzed 15 participants 15 participants
3.00
(1.00 to 9.00)
5.00
(2.00 to 11.00)
Visit 3 - First Intervention: Speech Number Analyzed 15 participants 15 participants
5.00
(1.00 to 6.00)
8.00
(2.00 to 11.00)
Visit 4 - First Intervention: Speech Number Analyzed 15 participants 15 participants
5.50
(3.00 to 8.50)
5.00
(2.00 to 10.50)
Visit 5 - First Intervention: Speech Number Analyzed 15 participants 15 participants
6.50
(3.00 to 7.50)
8.00
(2.00 to 11.50)
Visit 6 - Second Intervention: Speech Number Analyzed 15 participants 15 participants
4.00
(1.00 to 6.00)
8.50
(2.00 to 11.50)
Visit 7 - Second Intervention: Speech Number Analyzed 14 participants 15 participants
4.00
(2.00 to 5.00)
7.00
(4.00 to 12.00)
Visit 8 - Second Intervention: Speech Number Analyzed 15 participants 14 participants
5.00
(1.00 to 7.00)
6.50
(2.00 to 9.00)
Visit 9 - Second Intervention: Speech Number Analyzed 15 participants 15 participants
5.00
(2.00 to 8.50)
5.00
(2.00 to 10.00)
Visit 10 - Second Intervention: Speech Number Analyzed 14 participants 14 participants
5.00
(3.00 to 7.00)
7.25
(2.50 to 11.00)
Follow-up: Speech Composite Score Number Analyzed 14 participants 14 participants
6.00
(2.00 to 9.50)
6.00
(2.00 to 10.50)
Visit 1 - First Intervention: Symbolic Number Analyzed 15 participants 15 participants
9.50
(6.50 to 12.50)
14.00
(12.50 to 19.50)
Visit 2 - First Intervention: Symbolic Number Analyzed 15 participants 15 participants
10.50
(6.00 to 13.50)
14.50
(11.00 to 17.00)
Visit 3 - First Intervention: Symbolic Number Analyzed 15 participants 15 participants
10.50
(4.00 to 13.00)
15.00
(10.00 to 21.00)
Visit 4 - First Intervention: Symbolic Number Analyzed 15 participants 15 participants
12.00
(5.50 to 13.50)
14.00
(11.50 to 22.00)
Visit 5 - First Intervention: Symbolic Number Analyzed 15 participants 15 participants
11.50
(7.00 to 17.50)
16.50
(11.50 to 23.00)
Visit 6 - Second Intervention: Symbolic Number Analyzed 15 participants 15 participants
13.00
(6.50 to 15.00)
18.50
(12.00 to 23.00)
Visit 7 - Second Intervention: Symbolic Number Analyzed 14 participants 15 participants
10.25
(6.00 to 13.00)
17.00
(10.00 to 22.00)
Visit 8 - Second Intervention: Symbolic Number Analyzed 15 participants 14 participants
11.50
(6.50 to 15.50)
17.00
(9.00 to 20.50)
Visit 9 - Second Intervention: Symbolic Number Analyzed 15 participants 15 participants
11.50
(9.00 to 18.50)
18.00
(12.50 to 20.50)
Visit 10 - Second Intervention: Symbolic Number Analyzed 14 participants 14 participants
13.75
(8.00 to 17.00)
17.00
(11.00 to 19.00)
Follow-up: Symbolic Composite Score Number Analyzed 14 participants 14 participants
14.25
(9.00 to 16.00)
18.00
(13.50 to 19.00)
16.Secondary Outcome
Title Aberrant Behavior Checklist – Community Edition (ABC-C)
Hide Description

The ABC-C is a global behavior checklist implemented for the measurement of drug and other treatment effects in populations with intellectual disability. Behavior based on 58 items that describe various behavioral problems.

Each item is rated on the parents perceived severity of the behavior. The answer options for each item are:

0 = Not a problem

  1. = Problem but slight in degree
  2. = Moderately serious problem
  3. = Severe in degree

The measure is broken down into the following subscales with individual ranges as follows:

Subscale I (Irritability): 15 items, score range = 0-45 Subscale II (Lethargy): 16 items, score range = 0-48 Subscale III (Stereotypy): 7 items, score range = 0-21 Subscale IV (Hyperactivity): 16 items, score range = 0-48 Subscale V (Inappropriate Speech) was not included in the breakdown because it was not applicable (no participants in the study had verbal language).

Time Frame Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

In some cases, the number of participants analyzed is less than 15 for each group due to caregiver(s) not completing the forms.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
Visit 1 - First Intervention: Subscale I Number Analyzed 15 participants 15 participants
9.00
(6.00 to 22.00)
6.00
(3.00 to 12.00)
Visit 3 - First Intervention: Subscale I Number Analyzed 15 participants 15 participants
9.00
(2.00 to 15.00)
4.00
(0.00 to 7.00)
Visit 5 - First Intervention: Subscale I Number Analyzed 15 participants 15 participants
7.00
(0.00 to 16.00)
2.00
(0.00 to 6.00)
Visit 6 - Second Intervention: Subscale I Number Analyzed 15 participants 15 participants
7.00
(3.00 to 13.00)
4.00
(0.00 to 10.00)
Visit 8 - Second Intervention: Subscale I Number Analyzed 15 participants 15 participants
4.00
(1.00 to 17.00)
3.00
(0.00 to 10.00)
Visit 10 - Second Intervention: Subscale I Number Analyzed 15 participants 14 participants
5.00
(2.00 to 14.00)
5.00
(0.00 to 8.00)
Follow-up: Subscale I (Irritability) Number Analyzed 15 participants 13 participants
3.00
(0.00 to 9.00)
2.00
(0.00 to 7.00)
Visit 1 - First Intervention: Subscale II Number Analyzed 15 participants 15 participants
13.00
(5.00 to 19.00)
8.00
(4.00 to 10.00)
Visit 3 - First Intervention: Subscale II Number Analyzed 15 participants 15 participants
11.00
(3.00 to 17.00)
7.00
(3.00 to 8.00)
Visit 5 - First Intervention: Subscale II Number Analyzed 15 participants 15 participants
9.00
(5.00 to 12.00)
6.00
(4.00 to 10.00)
Visit 6 - Second Intervention: Subscale II Number Analyzed 15 participants 15 participants
11.00
(5.00 to 16.00)
5.00
(2.00 to 9.00)
Visit 8 - Second Intervention: Subscale II Number Analyzed 15 participants 15 participants
8.00
(4.00 to 11.00)
5.00
(2.00 to 9.00)
Visit 10 - Second Intervention: Subscale II Number Analyzed 15 participants 14 participants
6.00
(4.00 to 9.00)
4.00
(2.00 to 6.00)
Follow-up: Subscale II (Lethargy) Number Analyzed 15 participants 13 participants
6.00
(2.00 to 11.00)
5.00
(2.00 to 8.00)
Visit 1 - First Intervention: Subscale III Number Analyzed 15 participants 15 participants
13.00
(10.00 to 16.00)
12.00
(9.00 to 14.00)
Visit 3 - First Intervention: Subscale III Number Analyzed 15 participants 15 participants
10.00
(8.00 to 15.00)
10.00
(8.00 to 13.00)
Visit 5 - First Intervention: Subscale III Number Analyzed 15 participants 15 participants
11.00
(9.00 to 16.00)
9.00
(7.00 to 12.00)
Visit 6 - Second Intervention: Subscale III Number Analyzed 15 participants 15 participants
11.00
(7.00 to 16.00)
11.00
(9.00 to 12.00)
Visit 8 - Second Intervention: Subscale III Number Analyzed 15 participants 15 participants
10.00
(7.00 to 15.00)
9.00
(7.00 to 11.00)
Visit 10 - Second Intervention: Subscale III Number Analyzed 15 participants 15 participants
8.00
(6.00 to 12.00)
9.00
(7.00 to 12.00)
Follow-up: Subscale III (Stereotypy) Number Analyzed 15 participants 15 participants
8.00
(6.00 to 11.00)
9.00
(7.00 to 12.00)
Visit 1 - First Intervention: Subscale IV Number Analyzed 15 participants 15 participants
13.00
(8.00 to 22.00)
8.00
(6.00 to 14.00)
Visit 3 - First Intervention: Subscale IV Number Analyzed 15 participants 15 participants
12.00
(7.00 to 15.00)
8.00
(3.00 to 13.00)
Visit 5 - First Intervention: Subscale IV Number Analyzed 15 participants 15 participants
11.00
(8.00 to 26.00)
6.00
(3.00 to 6.00)
Visit 6 - Second Intervention: Subscale IV Number Analyzed 15 participants 15 participants
11.00
(7.00 to 22.00)
7.00
(2.00 to 17.00)
Visit 8 - Second Intervention: Subscale IV Number Analyzed 15 participants 15 participants
7.00
(5.00 to 16.00)
4.00
(0.00 to 11.00)
Visit 10 - Second Intervention: Subscale IV Number Analyzed 15 participants 14 participants
10.00
(4.00 to 20.00)
5.00
(2.00 to 10.00)
Follow-up: Subscale IV (Hyperactivity) Number Analyzed 15 participants 13 participants
9.00
(3.00 to 17.00)
5.00
(1.00 to 9.00)
17.Secondary Outcome
Title Quantitative Measures of Respiration: Apnea Index
Hide Description

Respiratory data was collected using non-invasive respiratory inductance plethysmography from a BioCapture® recording device. BioCapture® is a child-friendly measurement device that can record from 1 to 12 physiological signal transducers in a time-locked manner. It can be configured with the pediatric chest and abdominal plethysmography bands and the 3 lead ECG signals we plan to use for monitoring cardiac safety throughout the study. Each transducer is placed on the patient independently to provide a customized fit that yields the highest signal quality for each patient irrespective of body shape and proportion. The transducer signals captured by the BioCapture® are transmitted wirelessly to a laptop computer where all signals are displayed in real-time.

The apnea index is given as apneas/hour. Data on apneas greater than or equal to 10 seconds are displayed below. The higher the frequency of apnea, the more severe the breathing abnormality.

Time Frame Every 10 weeks during each of the two 20-week treatment periods
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

In some cases, the number of participants analyzed is less than 15 for each group due to inability to complete testing session.

One participant from the "rhIGF-1 First, Then Placebo" group was removed from the study prior to Visit 10, so there is only data for 14 participants included at that time point.

Arm/Group Title Placebo First, Then rhIGF-1 rhIGF-1 First, Then Placebo
Hide Arm/Group Description:
One half of subjects will be randomly assigned to receive placebo during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of a saline solution (placebo).
One half of subjects will be randomly assigned to receive Recombinant Human Insulin Growth Factor 1 (rhIGF-1) during the first arm of the study. Subjects assigned to this group will receive twice daily subcutaneous injections of IGF-1.
Overall Number of Participants Analyzed 15 15
Median (Inter-Quartile Range)
Unit of Measure: Apneas/Hour
Visit 1 - First Intervention: Apnea Index Number Analyzed 15 participants 14 participants
7.58
(1.58 to 10.33)
4.05
(2.07 to 7.91)
Visit 3 - First Intervention: Apnea Index Number Analyzed 15 participants 14 participants
4.80
(0.38 to 9.20)
3.48
(2.00 to 4.19)
Visit 5 - First Intervention: Apnea Index Number Analyzed 15 participants 14 participants
6.93
(3.06 to 9.07)
3.07
(0.40 to 5.11)
Visit 6 - Second Intervention: Apnea Index Number Analyzed 15 participants 14 participants
7.90
(4.30 to 17.60)
3.62
(2.20 to 11.82)
Visit 8 - Second Intervention: Apnea Index Number Analyzed 15 participants 14 participants
7.28
(2.80 to 12.53)
5.55
(1.24 to 12.81)
Visit 10 - Second Intervention: Apnea Index Number Analyzed 15 participants 14 participants
8.91
(4.13 to 14.34)
5.56
(1.57 to 13.47)
Time Frame 80 weeks (from screening visit to 12-week follow up phone call).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo rhIGF-1 Off Treatment
Hide Arm/Group Description This group includes subjects who had a reported Adverse Event (AE) while they were receiving placebo treatment. This group includes subjects who had a reported Adverse Event (AE) while they were receiving rhIGF-1 treatment. This group includes subjects who had a reported Adverse Event (AE) while they were not receiving any treatment. This includes the period between Screening and Baseline, the 20-week washout period between study arms, and the 12-week follow up period after study completion.
All-Cause Mortality
Placebo rhIGF-1 Off Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/30 (0.00%)      0/30 (0.00%)      0/30 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo rhIGF-1 Off Treatment
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/30 (13.33%)      4/30 (13.33%)      2/30 (6.67%)    
Gastrointestinal disorders       
Colitis * 1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/30 (0.00%)  0
General disorders       
Seizure * 1  2/30 (6.67%)  2 1/30 (3.33%)  1 0/30 (0.00%)  0
Infections and infestations       
Pneumonia * 1  2/30 (6.67%)  2 2/30 (6.67%)  3 1/30 (3.33%)  2
Respiratory, thoracic and mediastinal disorders       
Aspiration * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 1/30 (3.33%)  1
1
Term from vocabulary, CTCAE (4.0)
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Placebo rhIGF-1 Off Treatment
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   16/30 (53.33%)      20/30 (66.67%)      15/30 (50.00%)    
Cardiac disorders       
Abnormal ECG - Prolonged QT  1  0/30 (0.00%)  0 0/30 (0.00%)  0 2/30 (6.67%)  2
Other Cardiac Disorder  1  0/30 (0.00%)  0 0/30 (0.00%)  0 1/30 (3.33%)  1
Ear and labyrinth disorders       
Otitis Media  1  2/30 (6.67%)  2 4/30 (13.33%)  4 1/30 (3.33%)  1
Endocrine disorders       
Advanced Bone Age  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Precocious Puberty  1  0/30 (0.00%)  0 1/30 (3.33%)  1 1/30 (3.33%)  1
Gastrointestinal disorders       
Vomiting  1  2/30 (6.67%)  2 1/30 (3.33%)  1 1/30 (3.33%)  1
Vomiting and Diarrhea * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Constipation  1  0/30 (0.00%)  0 2/30 (6.67%)  2 0/30 (0.00%)  0
Diarrhea  1  1/30 (3.33%)  1 1/30 (3.33%)  1 0/30 (0.00%)  0
Dyspepsia * 1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Gastroenteritis  1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
General disorders       
Seizures * 1  3/30 (10.00%)  4 1/30 (3.33%)  1 2/30 (6.67%)  2
Seizure and vomiting * 1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Elevated Alkaline Phosphatase * 1  0/30 (0.00%)  0 0/30 (0.00%)  0 1/30 (3.33%)  1
Alopecia  1  0/30 (0.00%)  0 2/30 (6.67%)  2 0/30 (0.00%)  0
Dysphagia * 1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Productive Cough and Vomiting  1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Car Accident * 1 [1]  0/30 (0.00%)  0 0/30 (0.00%)  0 1/30 (3.33%)  1
Fall  1  0/30 (0.00%)  0 1/30 (3.33%)  1 1/30 (3.33%)  1
Injection Site Reaction  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Minor Head Injury  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Immune system disorders       
Tonsillar Hypertrophy  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Infections and infestations       
Conjunctivitis * 1  0/30 (0.00%)  0 0/30 (0.00%)  0 1/30 (3.33%)  1
Lung Infection  1  0/30 (0.00%)  0 2/30 (6.67%)  2 0/30 (0.00%)  0
Pharyngitis * 1  2/30 (6.67%)  2 1/30 (3.33%)  1 0/30 (0.00%)  0
Pneumonia * 1  1/30 (3.33%)  1 1/30 (3.33%)  1 0/30 (0.00%)  0
Seizure and Fever * 1  0/30 (0.00%)  0 0/30 (0.00%)  0 2/30 (6.67%)  2
Sinusitis * 1  1/30 (3.33%)  1 4/30 (13.33%)  4 1/30 (3.33%)  1
Fever  1  1/30 (3.33%)  1 1/30 (3.33%)  1 0/30 (0.00%)  0
Hand, Foot, and Mouth Disease  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Skin (Eye) Infection  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Upper Respiratory Infection * 1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Metabolism and nutrition disorders       
Anorexia  1  0/30 (0.00%)  0 0/30 (0.00%)  0 1/30 (3.33%)  1
Musculoskeletal and connective tissue disorders       
Fracture  1  1/30 (3.33%)  1 1/30 (3.33%)  1 0/30 (0.00%)  0
Scoliosis  1  1/30 (3.33%)  1 0/30 (0.00%)  0 1/30 (3.33%)  1
Unequal limb length * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Nervous system disorders       
Extrapyramidal Disorder  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Psychiatric disorders       
Anxiety * 1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Personality Disorder * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Sleep Apnea  1  0/30 (0.00%)  0 1/30 (3.33%)  1 1/30 (3.33%)  1
Cough  1  1/30 (3.33%)  1 0/30 (0.00%)  0 0/30 (0.00%)  0
Skin and subcutaneous tissue disorders       
Eczema  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Facial Swelling * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Rash  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Skin Abscess * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Surgical and medical procedures       
Adenoidectomy  1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
Gastrostomy Tube Replacement * 1  0/30 (0.00%)  0 1/30 (3.33%)  1 0/30 (0.00%)  0
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
[1]
Participant was in car accident on way to study visit. No injuries sustained.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Dr. Mustafa Sahin, Director of Translational Neuroscience Center
Organization: Boston Children's Hospital
Phone: 617-919-6258
Publications:
Responsible Party: Mustafa Sahin, Harvard Medical School
ClinicalTrials.gov Identifier: NCT01777542     History of Changes
Other Study ID Numbers: IRB-P00005610
First Submitted: January 23, 2013
First Posted: January 29, 2013
Results First Submitted: November 20, 2017
Results First Posted: March 26, 2018
Last Update Posted: March 26, 2018