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

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Brief Title

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

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.

        5. Change in height and weight. Population:Ten girls, 6 to 15 years old, diagnosed with
           Rett syndrome (RTT) Study Design:This is a single - center, exploratory, open-label,
           study in 10 girls diagnosed with RTT. The study will consist of four parts: Screening
           and baseline assessments, initial and final dose-setting period, treatment period and
           end-of study follow-up.

      Investigational Product:Glatiramer Acetate (Copaxone® , Teva Pharmaceutical Industries Ltd.)
      Sample Size Consideration: The planned sample size of 10 patients was considered adequate by
      the investigator for this phase I exploratory proof-of-concept study. The study is not
      expected to show statistical significance or statistical power, only a trend for the study
      endpoints. Each patient will serve as her own control.

      Duration of Study: Approximately 8 months per patient (including up to 2 weeks pre-treatment
      assessment, 6 months initial dose and treatment periods and end-of study visit).

      Overall study duration: the study is expected to be completed within 12 months (dependent on
      rate of recruitment).
    


Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

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

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.

Condition

Rett Syndrome

Intervention

Glatiramer Acetate (Copaxone®)

Study Arms / Comparison Groups

 Copaxone
Description:  Glatiramer Acetate (Copaxone® , Teva Pharmaceutical Industries Ltd.) 20 mg daily or in an interval determined in the Dose Setting period. Administration will be subcutaneous to various areas on the body: back of the upper arms (2 areas), front and outside of thighs (2 areas), upper buttocks/rear hips (2 areas), and stomach (the abdomen).

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

January 2014

Completion Date

February 2015

Primary Completion Date

September 2014

Eligibility Criteria

        Inclusion Criteria:

          1. Females, age 6-15 years (inclusive).

          2. Patients whose parents or legal custodians have provided written informed consent to
             participate in the study.

          3. A diagnosis of RTT (classical or variant), defined according to the internationally
             agreed 2010 RetSearch criteria [4].

          4. Evidence of a genetically defined pathological change in the MECP2 gene (point
             mutation or deletion)

          5. Patients with known epileptiform activity as recorded on EEG.

          6. Blood pressure and heart rate within normal limits (blood pressure: systolic 90-140
             mmHg; diastolic 50-90 mmHg, heart rate 40-120 beats per minute

          7. An electrocardiogram (ECG) which, according to the Investigator's judgment does not
             contraindicate participation in the study.

          8. No clinically significant abnormalities in haematology, blood chemistry lab tests at
             screening.

          9. Parents must be able to understand the requirements of the study and must be willing
             to comply with the requirements of the study

        Exclusion Criteria:

          1. Any medical problem or chronic illness beyond those known to be associated with Rett
             Syndrome which, in the investigator's judgment, contraindicates administration of the
             study medication.

          2. Severe respiratory dysfunction (defined as tracheostomy and/or chronic oxygen therapy
             at least 4 hours a day and/or repeated aspiration pneumonia - at least 4 in the last
             year).

          3. Intractable seizures that started during the last 6 months prior to beginning of the
             study.

          4. Known hypersensitivity to glatiramer or mannitol.

          5. Participation in another clinical study.

          6. Parents of a patient who are unable to communicate well with the investigator and
             staff and comply with study procedures and follow-up

          7. Parents of a patient who are unwilling to sign consent form.
      

Gender

Female

Ages

6 Years - 15 Years

Accepts Healthy Volunteers

No

Contacts

Bruria Ben Zeev, prof., +972 3 5302687, [email protected]

Location Countries

Israel

Location Countries

Israel

Administrative Informations


NCT ID

NCT02023424

Organization ID

SHEBA-12-9855-BBZ-CTIL


Responsible Party

Principal Investigator

Study Sponsor

Sheba Medical Center


Study Sponsor

Bruria Ben Zeev, prof., Principal Investigator, Sheba Medical Center


Verification Date

February 2014