Status:
COMPLETED
Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
Lead Sponsor:
Edison Pharmaceuticals Inc
Conditions:
Rett Syndrome
Eligibility:
FEMALE
Up to 18 years
Phase:
PHASE2
Brief Summary
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2014
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT01822249
Start Date
January 1 2013
End Date
January 1 2014
Last Update
July 26 2018
Active Locations (1)
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1
University of Siena
Siena, Italy