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Register:	ClinicalTrials.gov
Last refreshed on:	19 February 2015
Main ID:	NCT02023424
Date of registration:	23/12/2013
Prospective Registration:	Yes
Primary sponsor:	Sheba Medical Center
Public title:	An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome
Scientific title:	An Open Label, Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate (Copaxone ®) on Girls Woth Rett Syndrome
Date of first enrolment:	January 2014
Target sample size:	10
Recruitment status:	Recruiting
URL:	http://clinicaltrials.gov/show/NCT02023424
Study type:	Interventional
Study design:	Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Phase:	Phase 1

Countries of recruitment
Israel

Contacts

Name:	Irit Avisar, R.N M.A
Address:
Telephone:
Email:
Affiliation:	Sheba Medical Center

Name:	Andreea Nissenkorn, Dr.
Address:
Telephone:
Email:
Affiliation:	Sheba Medical Center

Name:	BRURIA BEN ZEEV, MD
Address:
Telephone:	+972 3 5302687
Email:	Bruria.BenZeev@sheba.health.gov.il
Affiliation:

Name:	BRURIA BEN ZEEV, MD
Address:
Telephone:	+972 3 5302687
Email:	Bruria.BenZeev@sheba.health.gov.il
Affiliation:

Name:	Bruria Ben Zeev, prof.
Address:
Telephone:
Email:
Affiliation:	Sheba Medical Center

Key inclusion & exclusion 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.

Age minimum: 6 Years
Age maximum: 15 Years
Gender: Female

Health Condition(s) or Problem(s) studied

Rett Syndrome

Intervention(s)

Drug: Glatiramer Acetate (Copaxone®)

Primary Outcome(s)

Improvement of epileptiform activity as recorded in a 24-hours EEG. [Time Frame: 6 months]

Secondary Outcome(s)

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]

Change in height and weight [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]

Secondary ID(s)

SHEBA-12-9855-BBZ-CTIL

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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