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Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

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ClinicalTrials.gov Identifier: NCT02153723
Recruitment Status : Completed
First Posted : June 3, 2014
Results First Posted : November 5, 2018
Last Update Posted : November 5, 2018
Sponsor:
Collaborator:
Rett Syndrome Research Trust
Information provided by (Responsible Party):
Aleksandra Djukic, Montefiore Medical Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Rett Syndrome
Intervention Drug: Glatiramer Acetate
Enrollment 10
Recruitment Details

All participants were recruited between Aug 2014-Jan 2015, from the population treated at the Rett Center at Montefiore.

Of 11 screened subjects, 10 met the inclusion/exclusion criteria and completed the trial. One patient was excluded due to prolonged QTc.

Data analysis was performed after the first 10 participants completed the study.

Pre-assignment Details  
Arm/Group Title Copaxone
Hide Arm/Group Description

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Period Title: Overall Study
Started 10
Completed 10
Not Completed 0
Arm/Group Title Copaxone
Hide Arm/Group Description

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
All enrolled participants were female, ambulatory, and had been previously diagnosed with Rett syndrome.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
9
  90.0%
Between 18 and 65 years
1
  10.0%
>=65 years
0
   0.0%
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
10
 100.0%
Male
0
   0.0%
[1]
Measure Description: All participants were female
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
1.Primary Outcome
Title Gait Velocity as Measured by GAITRite System
Hide Description To perform quantitative gait assessments a computerized walkway (457 × 90.2 × 0.64cm) with embedded pressure sensors (GAIT Rite system) was used. Subjects walked on the walkway for two trials, while wearing comfortable footwear.
Time Frame Baseline and Final week of treatment (week 32)
Hide Outcome Measure Data
Hide Analysis Population Description
All 10 patients were females with genetically confirmed Rett syndrome. All were at least 10 years old and ambulatory (walking without assistance at the time of their enrollment).
Arm/Group Title Copaxone
Hide Arm/Group Description:

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Participants Analyzed 10
Median (Inter-Quartile Range)
Unit of Measure: cm/sec
Baseline
62.7
(42.3 to 103.2)
Final week of treatment (week 32)
84.3
(58.1 to 119.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Copaxone
Comments [Not Specified]
Type of Statistical Test Other
Comments Wilcoxon signed rank sum test
Statistical Test of Hypothesis P-Value 0.03
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value 21.6
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Breath Hold Index (Number of Breath Holds Per Hour; Assessed in the Sleep Monitoring Lab)
Hide Description Breath hold index is defined as number of breath holds/hour. Respirations were monitored with sleep monitoring equipment during the daytime at the polysomnography laboratory with additional oronasal airflow, electromyography (EMG), EEG and video monitoring to confirm wakefulness during the period of study.
Time Frame Baseline and during final week of treatment (week 32)
Hide Outcome Measure Data
Hide Analysis Population Description
One of the 10 enrolled patients experienced panic attack during the respiratory function testing so that session was discontinued. This left 9 participants for final analysis.
Arm/Group Title Copaxone
Hide Arm/Group Description:

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Participants Analyzed 9
Median (Inter-Quartile Range)
Unit of Measure: number of breath holds/hour
Baseline
3.8
(2.3 to 7.3)
Final week of treatment (week 32)
1.6
(0.3 to 2.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Copaxone
Comments [Not Specified]
Type of Statistical Test Other
Comments Wilcoxon signed rank sum test
Statistical Test of Hypothesis P-Value 0.03
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value -2.0
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Breath Hold Time (Assessed in the Sleep Monitoring Lab)
Hide Description Breath Hold Time is defined as percentage of time spent holding the breath in a specific time unit. It is measured by a standard medical technique where belts are placed on the chest and abdomen to record movement and sensors are used to record nasal flow. Wake respiration was monitored with sleep monitoring equipment during the daytime at the polysomnography laboratory with additional oronasal airflow, EMG, EEG and video monitoring to confirm wakefulness during the period of study.
Time Frame Baseline and Final week of treatment (week 32)
Hide Outcome Measure Data
Hide Analysis Population Description
One of the 10 enrolled patients experienced panic attack during the respiratory function testing so that session was discontinued. This left 9 participants for final analysis.
Arm/Group Title Copaxone
Hide Arm/Group Description:

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Participants Analyzed 9
Median (Inter-Quartile Range)
Unit of Measure: percentage of time
Baseline
1.8
(0.8 to 2.3)
Final week of treatment (week 32)
0.4
(0.1 to 0.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Copaxone
Comments [Not Specified]
Type of Statistical Test Other
Comments Wilcoxon signed rank sum test
Statistical Test of Hypothesis P-Value 0.004
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value -0.1
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Visual Memory Novelty Score as Assessed by TX300 Tobii Computer.
Hide Description Eye-tracking is considered an indication of visual memory. Eye-tracking data was recorded at 300 Hz sampling rate using a Tobii T300 computer (Tobii Technology, Danderyd, Sweden). The actual data given by the computer represents the percentage of time spent looking at a novel visual target - this is called the novelty score. Visual memory, as indexed by the novelty score, is the percentage of time spent looking at a novel target during the test ("visual paired comparison paradigm"). Duration of testing was 2 minutes.
Time Frame Baseline and Final week of treatment (week 32)
Hide Outcome Measure Data
Hide Analysis Population Description
Only 7 of the 10 recruited participants were able to complete the cognitive assessment. The remaining 3 participants could not be tested due to technical reasons.
Arm/Group Title Copaxone
Hide Arm/Group Description:

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Participants Analyzed 7
Median (Inter-Quartile Range)
Unit of Measure: percentage of time
Baseline
42.4
(39.5 to 61.0)
Final week of treatment (week 32)
62.4
(55.7 to 65.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Copaxone
Comments [Not Specified]
Type of Statistical Test Other
Comments Wilcoxon signed rank sum test
Statistical Test of Hypothesis P-Value 0.08
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value 15.9
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Visual Attention (Number of Fixations) Assessed by Eye-tracking TX300 Tobii Computer.
Hide Description

Visual attention is indexed by duration and number of fixations on novel target on testing. The standard method of assessing visual attention in neuropsychology is by measuring:

A)number of fixations (how many times the subject looks at each of the 2 visual targets). The higher number of fixations, the more attentive the subject to that visual target.

B) duration of fixations in seconds (the longer the fixation the more attentive). Duration of fixations correlates with intelligence: the smarter the person is the shorter his fixations are.

Eye-tracking data was recorded at 300 Hz sampling rate using a Tobii T300 (Tobii Technology AB, Danderyd, Sweden). The measured index is called the Novelty Score which indicates the percentage of time spent looking at novel visual target. Duration of testing session was 2 minutes.

Time Frame Baseline and Final week of treatment (week 32)
Hide Outcome Measure Data
Hide Analysis Population Description
Only 7 of the 10 recruited participants were able to complete the cognitive assessment. The remaining 3 participants could not be tested due to technical reasons.
Arm/Group Title Copaxone
Hide Arm/Group Description:

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Participants Analyzed 7
Median (Inter-Quartile Range)
Unit of Measure: number of fixations
Baseline
19.4
(8.0 to 20.8)
Final week of treatment (week 32)
16.2
(11.0 to 27.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Copaxone
Comments [Not Specified]
Type of Statistical Test Other
Comments Wilcoxon signed rank sum test
Statistical Test of Hypothesis P-Value 0.86
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.8
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Visual Attention (Fixation Length) Assessed by Eye-tracking TX300 Tobii Computer.
Hide Description

The standard method of assessing visual attention in neuropsychology is by measuring:

A)number of fixations (how many times the subject looks at each of the 2 visual targets). The higher number of fixations, the more attentive the subject to that visual target.

B) duration of fixations in seconds (the longer the fixation the more attentive). Duration of fixations correlates with intelligence: the smarter the person is the shorter his fixations are.

Eye-tracking data was recorded at 300 Hz sampling rate using a Tobii T300 (Tobii Technology AB, Danderyd, Sweden). The measured index is called the Novelty Score which indicates the percentage of time spent looking at novel visual target. Visual attention is indexed by number of fixations on novel target on test.

Duration of testing session was 2 minutes.

Time Frame Baseline and Final week of treatment (week 32)
Hide Outcome Measure Data
Hide Analysis Population Description
Only 7 of the 10 recruited participants were able to complete the cognitive assessment. The remaining 3 participants could not be tested due to technical reasons.
Arm/Group Title Copaxone
Hide Arm/Group Description:

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

Overall Number of Participants Analyzed 7
Median (Inter-Quartile Range)
Unit of Measure: seconds
Baseline
0.46
(0.26 to 0.64)
Final week of treatment (week 32)
0.35
(0.18 to 0.46)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Copaxone
Comments [Not Specified]
Type of Statistical Test Other
Comments Wilcoxon signed rank sum test
Statistical Test of Hypothesis P-Value 0.44
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value 0.00
Estimation Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Copaxone
Hide Arm/Group Description

Dose escalation:

Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection.

Glatiramer Acetate

All-Cause Mortality
Copaxone
Affected / at Risk (%)
Total   0/10 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Copaxone
Affected / at Risk (%) # Events
Total   0/10 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Copaxone
Affected / at Risk (%) # Events
Total   1/10 (10.00%)    
Musculoskeletal and connective tissue disorders   
laboratory result (elevated CPK) without clinical correlate, transitory  [1]  1/10 (10.00%)  1
Indicates events were collected by systematic assessment
[1]
not sympromatic
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Aleksandra Djukic
Organization: Montefiore Medical Center
Phone: 718 920 4378
EMail: adjukic@montefiore.org
Layout table for additonal information
Responsible Party: Aleksandra Djukic, Montefiore Medical Center
ClinicalTrials.gov Identifier: NCT02153723     History of Changes
Other Study ID Numbers: 13-05-117
First Submitted: May 26, 2014
First Posted: June 3, 2014
Results First Submitted: July 20, 2015
Results First Posted: November 5, 2018
Last Update Posted: November 5, 2018