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

NCT ID: NCT01822249

Last Updated: 2018-07-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-01-31
• Study Completion Date: 2014-01-31

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 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.

Conditions

Rett Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

EPI-743 15 mg/kg

Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily

Group Type: ACTIVE_COMPARATOR

EPI-743

Intervention Type: DRUG

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Interventions

EPI-743

Intervention Type: DRUG

Placebo

Intervention Type: DRUG

Eligibility Criteria

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

• Maximum Eligible Age: 18 Years
• Eligible Sex: FEMALE
• Accepts Healthy Volunteers: No

Sponsors

Edison Pharmaceuticals Inc

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of Siena

Siena, , Italy

Countries

Italy

Other Identifiers

OPBGC&RS_12_003

Identifier Type: -

Identifier Source: org_study_id