Phase 2
Completed N=10
Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)
Source: ClinicalTrials.gov NCT02153723 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Nov 2018
Primary outcomePrimary: Gait Velocity as Measured by GAITRite System — 62.7; 84.3 cm/sec — p=0.03
Summary
A phase 2 open label trial to test a potential drug treatment for Rett syndrome, the leading known genetic cause of severe neurological impairment in girls. The drug, Copaxone (generic name - Glatiramer acetate) is medication FDA approved for the treatment of multiple sclerosis. Copaxone's high safety profile has been documented in large cohorts of patients for more than 12 years.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Gait Velocity as Measured by GAITRite System |
62.7; 84.3 | 0.03 sig |
| SECONDARY Breath Hold Index (Number of Breath Holds Per Hour; Assessed in the Sleep Monitoring Lab) |
3.8; 1.6 | 0.03 sig |
| SECONDARY Breath Hold Time (Assessed in the Sleep Monitoring Lab) |
1.8; 0.4 | 0.004 sig |
| SECONDARY Visual Memory Novelty Score as Assessed by TX300 Tobii Computer. |
42.4; 62.4 | 0.08 |
| SECONDARY Visual Attention (Number of Fixations) Assessed by Eye-tracking TX300 Tobii Computer. |
19.4; 16.2 | 0.86 |
| SECONDARY Visual Attention (Fixation Length) Assessed by Eye-tracking TX300 Tobii Computer. |
0.46; 0.35 | 0.44 |
Eligibility Criteria
Inclusion Criteria
- Female patients with genetically confirmed Rett Syndrome (RTT)
- Age: 10 or more years old. Selection of the age is based on the available evidence of the safety of Glatiramer Acetate (GA) in this group, and the relative homogeneity/stability of the phenotype, which is not expected to spontaneously change within a 6 month period at this age
- Ambulatory (with our without support)
Exclusion Criteria
- Prolonged Qtc (obtained within 30 days prior to enrollment)
- Presence of co morbid non-Rett related disease
- Presence of immunodeficiency requiring intravenous immunoglobulin 3 (IVIG 3) months prior to enrollment
- Allergy/sensitivity to GA or mannitol
- Inability or unwillingness of legal guardians to give written informed consent
Data sourced from ClinicalTrials.gov (NCT02153723). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.