Phase 2 Study of EPI-743 for Treatment of Rett Syndrome

Edison Pharmaceuticals

Status and phase

Completed

Phase 2

Conditions

Rett Syndrome

Treatments

Drug: EPI-743

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT01822249

OPBGC&RS_12_003

Details and patient eligibility

About

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.

Enrollment

24 patients

Sex

Female

Ages

Under 18 years old

Volunteers

No Healthy Volunteers

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