Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

NCT02153723 | Completed Phase 2 Results

• 1 other identifier: 13-05-117
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Rett Syndrome

Keywords

Treatment trial, Rett syndrome, Glatiramer acetate

Outcome Measures

Primary Outcomes (1)

• Gait Velocity as Measured by GAITRite System

  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.

  Baseline and Final week of treatment (week 32)

Secondary Outcomes (5)

• Breath Hold Index (Number of Breath Holds Per Hour; Assessed in the Sleep Monitoring Lab)

  Baseline and during final week of treatment (week 32)

• Breath Hold Time (Assessed in the Sleep Monitoring Lab)

  Baseline and Final week of treatment (week 32)

• Visual Memory Novelty Score as Assessed by TX300 Tobii Computer.

  Baseline and Final week of treatment (week 32)

• Visual Attention (Number of Fixations) Assessed by Eye-tracking TX300 Tobii Computer.

  Baseline and Final week of treatment (week 32)

• Visual Attention (Fixation Length) Assessed by Eye-tracking TX300 Tobii Computer.

  Baseline and Final week of treatment (week 32)

Study Arms (1)

Copaxone

EXPERIMENTAL

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.

Drug: Glatiramer Acetate

Interventions

Glatiramer Acetate DRUG

Also known as: Copaxone

Copaxone

Eligibility Criteria

• Age: 10 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Montefiore Medical Center: lead
• Rett Syndrome Research Trust: collaborator

Study Sites (1)

Montefiore Medical center

The Bronx, New York, 10467, United States

Location

Related Publications (1)

• 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.

  PMID: 27363291 RESULT

MeSH Terms

Conditions

Rett Syndrome

Interventions

Glatiramer Acetate

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Intervention Hierarchy (Ancestors)

Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Aleksandra Djukic
• Organization: Montefiore Medical Center

adjukic@montefiore.org

718 920 4378

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Clinical Neurology and Clinical Pediatrics, Director, Tri State Rett Syndrome Center

Study Record Dates

• First Submitted: May 26, 2014
• First Posted: June 3, 2014
• Study Start: August 1, 2013
• Primary Completion: August 1, 2014
• Study Completion: January 1, 2016
• Last Updated: November 5, 2018
• Results First Posted: November 5, 2018

Record last verified: 2018-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)