Clinical Trial: An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: An Open Label, Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate (Copaxone ®) on Girls Woth Rett Syndrome

Brief Summary:

Primary Objective: To test the hypothesis that 6 months treatment with glatiramer acetate (GA) decreases epileptiform activity in young girls with Rett syndrome.

Primary Safety Objective:To evaluate the safety and tolerability of 6 months treatment with GA in these patients.

Secondary Objectives:

  1. To test the hypothesis that 6 months treatment with glatiramer acetate (GA) improves respiratory dysfunction.
  2. To evaluate the effect of GA treatment on general behaviour communication, hand stereotyping, feeding, sleep and other autonomic symptoms: gastrointestinal and cardiac.
  3. To assess the effect of GA treatment on bodily development.

Primary Endpoint:Improvement of epileptiform activity as recorded in a 24-hours EEG.

Primary Safety Endpoint:Frequency and severity of treatment-related AEs (including safety lab parameters).

Secondary Endpoints:

  1. Improvement in the scoring of breath holds and hyperventilation, as measured with non-invasive respiratory inductance plethysmography (NoxT3 device) and parents' diaries.
  2. Changes in general behaviour, communication, feeding and motor skills as assessed by the investigator (based on Kerr and Naidu validated severity scores) and recorded in parents' diary.
  3. Decrease in seizure frequency as reported in parents' diary.
  4. Improvement in sleep schedule as recorded in a sleep diary.
  6. Improvement of epileptiform activity as recorded in a 24-hours EEG. [ Time Frame: 6 months ]
  7. Frequency and severity of treatment-related AEs (including safety lab parameters). [ Time Frame: 8 months ]


    8. Current Secondary Outcome:

    • 1.Improvement in the scoring of breath holds and hyperventilation, as measured with non-invasive respiratory inductance plethysmography (NoxT3 device) and parents' diaries. [ Time Frame: 8 months ]
    • 2. Changes in general behaviour, communication, feeding and motor skills as assessed by the investigator (based on Kerr and Naidu validated severity scores) and recorded in parents' diary. [ Time Frame: 8 months ]
    • Decrease in seizure frequency as reported in parents' diary. [ Time Frame: 8 months ]
    • Improvement in sleep schedule as recorded in a sleep diary. [ Time Frame: 8 months ]
    • Change in height and weight [ Time Frame: 8 months ]


    Original Secondary Outcome: Same as current

    Information By: Sheba Medical Center

    Dates:
    Date Received: December 23, 2013
    Date Started: January 2014
    Date Completion: February 2015
    Last Updated: February 3, 2014
    Last Verified: February 2014