Rett Syndrome Clinical Trial
Official title:
A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
Verified date | July 2018 |
Source | Edison Pharmaceuticals Inc |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 24 |
Est. completion date | January 2014 |
Est. primary completion date | December 2013 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | N/A to 18 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
Italy | University of Siena | Siena |
Lead Sponsor | Collaborator |
---|---|
Edison Pharmaceuticals Inc |
Italy,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Rett Syndrome Clinical Severity Sore | Measure of disease progression | Change at six months from baseline | |
Secondary | Oxidative Stress Biomarkers | Change at six months from baseline | ||
Secondary | Head circumference | Change at six months from baseline | ||
Secondary | Rett syndrome behavioral questionnaire | Change at six months from baseline | ||
Secondary | PedsQL | Change at six months from baseline | ||
Secondary | Number of Drug-related adverse and serious adverse events | Six months | ||
Secondary | Respiratory Disturbance Index (RDI) | RDI will be determined on polysomnography study | Change at six months from baseline |
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