Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

Phase 2 Completed 10 enrolled Montefiore Medical Center Rett Syndrome Research Trust · collab

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2013-08
Primary completion: 2014-08
Completion: 2016-01

Outcome

Met primary endpoint

paper Gait velocity improved significantly (improvement range 13%-95%, p=0.03 for both tests) PMID 27363291 ↗

registry MEDIAN Copaxone: Final week of treatment (week 32): 84.3 (58.1 to 119.3) cm/sec NCT02153723 ↗

registry analysis (other test); Copaxone; p = 0.03; Median Difference (Final Values) 21.6; Wilcoxon (Mann-Whitney) NCT02153723 ↗

Publications

Results Djukic A, Holtzer R, Shinnar S, Muzumdar H, Rose SA, Mowrey W, Galanopoulou AS, Shinnar R, Jankowski JJ, Feldman JF, Pillai S, Moshe SL. Pharmacologic Treatment of Rett Syndrome With Glatiramer Acetate. Pediatr Neurol. 2016 Aug;61:51-7. doi: 10.1016/j.pediatrneurol.2016.05.010. Epub 2016 May 27.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Glatiramer Acetate	Small molecule	—	Subcutaneous