- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01822249
Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
July 23, 2018 updated by: Edison Pharmaceuticals Inc
A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children.
Rett syndrome is characterized by significant elevation in blood markers of oxidative stress.
EPI-743 is a novel therapeutic with demonstrated efficacy and safety in the treatment of disorders characterized by oxidative stress.
The purpose of this study is to examine the safety and efficacy of EPI-743 in a population of children with Rett syndrome.
Study Overview
Study Type
Interventional
Enrollment (Actual)
24
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Siena, Italy
- University of Siena
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 18 years (Child, Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Female
Description
Inclusion Criteria:
- Diagnosis of Rett syndrome with disease stage 1-2
- Abnormality of at least two disease biomarker levels
- Confirmed MeCP2 mutation
- Patient or patient's guardian able to consent and comply with protocol requirements
- Abstention from use of Coenzyme Q10, vitamin E and Idebenone two weeks prior to enrollment into the study
Exclusion Criteria:
- Any condition, which in the opinion of the investigator could compromise the subject's safety or adherence to treatment with EPI-743.
- Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil).
- Clinically significant allergy or hypersensitivity to Vitamin E
- Lack of confirmation of MeCP2 mutation
- Clinical history of bleeding or abnormal baseline PT/PTT
- Diagnosis of any other concurrent inborn error of metabolism
- Hepatic insufficiency with LFTs greater than 3 times upper limit of normal
- Renal insufficiency requiring dialysis
- End stage cardiac failure
- Fat malabsorption syndromes precluding drug absorption
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: EPI-743 15 mg/kg
Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily
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Placebo Comparator: Placebo
Subjects in this arm will receive placebo at a volume equivalent to the volume of EPI-743 they would receive if in active group based on their weight
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Rett Syndrome Clinical Severity Sore
Time Frame: Change at six months from baseline
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Measure of disease progression
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Change at six months from baseline
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Oxidative Stress Biomarkers
Time Frame: Change at six months from baseline
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Change at six months from baseline
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Head circumference
Time Frame: Change at six months from baseline
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Change at six months from baseline
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Rett syndrome behavioral questionnaire
Time Frame: Change at six months from baseline
|
Change at six months from baseline
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PedsQL
Time Frame: Change at six months from baseline
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Change at six months from baseline
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Number of Drug-related adverse and serious adverse events
Time Frame: Six months
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Six months
|
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Respiratory Disturbance Index (RDI)
Time Frame: Change at six months from baseline
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RDI will be determined on polysomnography study
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Change at six months from baseline
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
January 1, 2013
Primary Completion (Actual)
December 1, 2013
Study Completion (Actual)
January 1, 2014
Study Registration Dates
First Submitted
March 25, 2013
First Submitted That Met QC Criteria
March 27, 2013
First Posted (Estimate)
April 2, 2013
Study Record Updates
Last Update Posted (Actual)
July 26, 2018
Last Update Submitted That Met QC Criteria
July 23, 2018
Last Verified
July 1, 2018
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- OPBGC&RS_12_003
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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