Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

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Brief Title

Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

Official Title

Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

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.
    

Detailed Description

      Background/rationale for the study:

      In Rett syndrome brain cells aren't actually lost, instead poor maturation of connections
      between brain cells (synapses) prevents effective neurological functioning, and is the main
      morphological feature of the disease. The MeCP2 gene plays a major role in transcriptional
      regulation of other genes, one of which is the gene encoding brain-derived neurotrophic
      factor (BDNF).

      The disease progression and severity of symptoms is directly affected by the level of BDNF
      expression. An increase of BDNF levels (by genetic manipulations or pharmacological agents)
      leads to delayed onset of Rett syndrome-like symptoms in experimental models; rescued
      gait/mobility, improved quality of life and increased survival rates.

      Copaxone treatment by subcutaneous injection caused elevation of BDNF levels. Quantitative
      immunofluorescence assays showed about a twofold increase in neuronal expression of BDNF
      following Copaxone treatment.

      We expect that an increase in BDNF levels with Copaxone administration will stimulate
      communication between brain cells (synaptic maturation), which will lead to amelioration of
      symptoms (motor functions/gait, cognitive functions, breathing, encephalopathy and improve
      quality of life) for girls with Rett syndrome.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Gait Velocity as Measured by GAITRite System

Secondary Outcome

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

Condition

Rett Syndrome

Intervention

Glatiramer Acetate

Study Arms / Comparison Groups

 Copaxone
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

10

Start Date

August 2013

Completion Date

January 2016

Primary Completion Date

August 2014

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
      

Gender

Female

Ages

10 Years - N/A

Accepts Healthy Volunteers

No

Contacts

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Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02153723

Organization ID

13-05-117


Responsible Party

Principal Investigator

Study Sponsor

Montefiore Medical Center

Collaborators

 Rett Syndrome Research Trust

Study Sponsor

, , 


Verification Date

October 2018