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Trial record 13 of 48 for:    rett

Treatment of Rett Syndrome With Recombinant Human IGF-1

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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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment

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

Reporting Groups
  Description
Placebo First, Then rhIGF-1 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).
rhIGF-1 First, Then 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.

Participant Flow for 3 periods

Period 1:   First Intervention (20 Weeks)
    Placebo First, Then rhIGF-1   rhIGF-1 First, Then Placebo
STARTED   15   15 
COMPLETED   15   15 
NOT COMPLETED   0   0 

Period 2:   Washout Period (28 +/- 2 Weeks)
    Placebo First, Then rhIGF-1   rhIGF-1 First, Then Placebo
STARTED   15   15 
COMPLETED   15   15 
NOT COMPLETED   0   0 

Period 3:   Second Intervention (20 Weeks)
    Placebo First, Then rhIGF-1   rhIGF-1 First, Then Placebo
STARTED   15   15 
COMPLETED   15   14 
NOT COMPLETED   0   1 
Adverse Event                0                1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
All Participants All 30 subjects enrolled and randomized in the study

Baseline Measures
   All Participants 
Overall Participants Analyzed 
[Units: Participants]
 30 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      30 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      30 100.0% 
Male      0   0.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      26  86.7% 
Not Hispanic or Latino      3  10.0% 
Unknown or Not Reported      1   3.3% 


  Outcome Measures

1.  Primary:   Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

2.  Primary:   Anxiety, Depression, and Mood Scale (ADAMS) - Social Avoidance Subscale   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

3.  Primary:   Clinical Global Impression – Severity (CGI-S)   [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ]

4.  Primary:   Clinical Global Impression – Improvement (CGI-I)   [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ]

5.  Primary:   Parental Global Impression – Severity (PGI-S)   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

6.  Primary:   Parental Global Impression – Improvement (PGI-I)   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

7.  Primary:   Parent Targeted Visual Analog Scale (PTSVAS) - Scale 1   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

8.  Primary:   Parent Targeted Visual Analog Scale (PTSVAS) - Scale 2   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

9.  Primary:   Parent Targeted Visual Analog Scale (PTSVAS) - Scale 3   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

10.  Primary:   Kerr Clinical Severity Scale   [ Time Frame: At the start and end of each 20-week treatment period ]

11.  Secondary:   Rett Syndrome Behavior Questionnaire (RSBQ)   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

12.  Secondary:   Anxiety, Depression, and Mood Scale (ADAMS)   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

13.  Secondary:   Mullen Scales of Early Learning (MSEL)   [ Time Frame: At the start and end of each 20-week treatment period ]

14.  Secondary:   Vineland Adaptive Behavior Scales, Second Edition (VABS-II)   [ Time Frame: At the start and end of each 20-week treatment period ]

15.  Secondary:   Communication and Symbolic Behavior Scales – Developmental Profile (CSBS-DP)   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

16.  Secondary:   Aberrant Behavior Checklist – Community Edition (ABC-C)   [ Time Frame: Every 5 weeks during each of the two 20-week treatment periods, and once 4 weeks after final treatment ends ]

17.  Secondary:   Quantitative Measures of Respiration: Apnea Index   [ Time Frame: Every 10 weeks during each of the two 20-week treatment periods ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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
e-mail: ResearchTNC@childrens.harvard.edu


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