Rett SyndromePhase 1NCT02023424
An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls Woth Rett Syndrome
A Phase 1 study testing Glatiramer Acetate (Copaxone®) in people with Rett Syndrome, run by Sheba Medical Center at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 15 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- Glatiramer Acetate (Copaxone®)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteIsrael
- Study dates
- Jan 2014to Feb 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 15 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Females, age 6-15 years (inclusive).
- Patients whose parents or legal custodians have provided written informed consent to participate in the study.
- A diagnosis of RTT (classical or variant), defined according to the internationally agreed 2010 RetSearch criteria [4].
- Evidence of a genetically defined pathological change in the MECP2 gene (point mutation or deletion)
- Patients with known epileptiform activity as recorded on EEG.
- 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
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- An electrocardiogram (ECG) which, according to the Investigator's judgment does not contraindicate participation in the study.
- No clinically significant abnormalities in haematology, blood chemistry lab tests at screening.
- Parents must be able to understand the requirements of the study and must be willing to comply with the requirements of the study
Who cannot join
- 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.
- 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).
- Intractable seizures that started during the last 6 months prior to beginning of the study.
- Known hypersensitivity to glatiramer or mannitol.
- Participation in another clinical study.
- Parents of a patient who are unable to communicate well with the investigator and staff and comply with study procedures and follow-up
- Parents of a patient who are unwilling to sign consent form.
Trial locations and contacts
1 site in 1 country.
Israel1 site, 1 recruiting
- Recruiting
Sheba Medical Center
Ramat Gan
BRURIA BEN ZEEV, MD, +972 3 5302687, Bruria.BenZeev@sheba.health.gov.il
Irit Avisar, R.N M.A, +972544222786, irit.avisar@sheba.health.gov.il
About this study
Official title: An Open Label, Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate (Copaxone ®) on Girls Woth Rett Syndrome
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.
Read more
Secondary Objectives:
- To test the hypothesis that 6 months treatment with glatiramer acetate (GA) improves respiratory dysfunction.
- To evaluate the effect of GA treatment on general behaviour communication, hand stereotyping, feeding, sleep and other autonomic symptoms: gastrointestinal and cardiac.
- 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:
- Improvement in the scoring of breath holds and hyperventilation, as measured with non-invasive respiratory inductance plethysmography (NoxT3 device) and parents' diaries.
- 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.
- Decrease in seizure frequency as reported in parents' diary.
- Improvement in sleep schedule as recorded in a sleep diary.
- 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).
- Conditions
- Rett Syndrome
- Being tested
- Glatiramer Acetate (Copaxone®)
- Keywords
- BDNF, GLATIRAMER ACETATE, RETT SYNDROME, COPAXONE, Bruria Ben-Zeev
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Glatiramer Acetate (Copaxone®)Drug
GA is a potent inducer of Th2-cells and modulates these immune cells to secrete high levels of neurotrophic factors, particularly BDNF. The induced cells cross the blood brain barrier (BBB), accumulate in the CNS and express BDNF and other regulatory substances in situ.
| Study group | Role | What they receive |
|---|---|---|
| Copaxone | Experimental | 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). Receives: Drug: Glatiramer Acetate (Copaxone®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement of epileptiform activity as recorded in a 24-hours EEG.
Measured: 6 months
Secondary outcomes (5)
1.Improvement in the scoring of breath holds and hyperventilation, as measured with non-invasive respiratory inductance plethysmography (NoxT3 device) and parents' diaries.
Measured: 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.
Measured: 8 months
Decrease in seizure frequency as reported in parents' diary.
Measured: 8 months
Improvement in sleep schedule as recorded in a sleep diary.
Measured: 8 months
Change in height and weight
Measured: 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2013 (estimated)
- Study startJan 2014
- Primary completionSep 2014 (estimated)
- Study completionFeb 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- More trials run by Sheba Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sheba Medical Center
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Prof. Bruria Ben-Zeev MD, Sheba Medical Center
- Investigators
- Bruria Ben Zeev, prof., Principal investigator, Sheba Medical CenterAndreea Nissenkorn, Dr., Principal investigator, Sheba Medical CenterIrit Avisar, R.N M.A, Study director, Sheba Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02023424
- Sponsor's protocol ID
- SHEBA-12-9855-BBZ-CTIL
- Status last verified
- Feb 2014
- First submitted
- 23 Dec 2013
- Last updated
- 4 Feb 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Exploratory Study to Investigate the Treatment Effect og Glatiramer Acetate (Copaxone ®) on Girls Woth Rett Syndrome
- ClinicalTrials.gov ID
- NCT02023424
- Acronym / sponsor ID
- SHEBA-12-9855-BBZ-CTIL
- Conditions
- Rett Syndrome
- Keywords
- BDNF, GLATIRAMER ACETATE, RETT SYNDROME, COPAXONE, Bruria Ben-Zeev
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Sheba Medical CenterOther government
- Study start
- Jan 2014
- Primary completion
- Sep 2014 (estimated)
- Study completion
- Feb 2015 (estimated)
- First posted
- 30 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- To test the hypothesis that 6 months treatment with glatiramer acetate (GA) improves respiratory dysfunction.
- To evaluate the effect of GA treatment on general behaviour communication, hand stereotyping, feeding, sleep and other autonomic symptoms: gastrointestinal and cardiac.
- 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:
- Improvement in the scoring of breath holds and hyperventilation, as measured with non-invasive respiratory inductance plethysmography (NoxT3 device) and parents' diaries.
- 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.
- Decrease in seizure frequency as reported in parents' diary.
- Improvement in sleep schedule as recorded in a sleep diary.
- 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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Copaxone | Experimental | Drug: Glatiramer Acetate (Copaxone®)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). |
Who may qualify
- Ages
- 6 to 15 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Females, age 6-15 years (inclusive).
- Patients whose parents or legal custodians have provided written informed consent to participate in the study.
- A diagnosis of RTT (classical or variant), defined according to the internationally agreed 2010 RetSearch criteria [4].
- Evidence of a genetically defined pathological change in the MECP2 gene (point mutation or deletion)
- Patients with known epileptiform activity as recorded on EEG.
Key exclusion criteria (7 listed)
- 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.
- 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).
- Intractable seizures that started during the last 6 months prior to beginning of the study.
- Known hypersensitivity to glatiramer or mannitol.
- Participation in another clinical study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Glatiramer Acetate (Copaxone®)GA is a potent inducer of Th2-cells and modulates these immune cells to secrete high levels of neurotrophic factors, particularly BDNF. The induced cells cross the blood brain barrier (BBB), accumulate in the CNS and express BDNF and other regulatory substances in situ. | Drug | Copaxone |
Primary outcome
Improvement of epileptiform activity as recorded in a 24-hours EEG.
Time frame: 6 months
5 secondary outcomes
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Feb 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bruria Ben Zeev, prof. principal investigator
Sheba Medical Center
Andreea Nissenkorn, Dr. principal investigator
Sheba Medical Center
Irit Avisar, R.N M.A study director
Sheba Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Glatiramer Acetate (Copaxone®) in people with Rett Syndrome, run by Sheba Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact BRURIA BEN ZEEV, MD (+972 3 5302687, Bruria.BenZeev@sheba.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement of epileptiform activity as recorded in a 24-hours EEG. (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Feb 2014.