Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

• Status: Completed
• Trial end date: 2016-01-01
• Target enrollment: 0
• Participant gender: Female

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Montefiore Medical Center
• Collaborator: Rett Syndrome Research Trust
• Treatments: (T,G)-A-L; Glatiramer Acetate

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