Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

Completed · Phase 2

Conditions studied: Rett Syndrome

In brief

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.

Key facts

Study ID

NCT02153723

Run by

Montefiore Medical Center

People needed

10

Starts

2013-08-01

Expected to finish

2016-01-01

Last updated by the study team

2018-11-05

Who can join

Age: 10 and older. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Montefiore Medical center — The Bronx, New York, United States

Full record on ClinicalTrials.gov

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