Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.

• Conditions: Rett Syndrome; MedDRA version: 14.1Level: HLGTClassification code 10021605Term: Inborn errors of metabolismSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2012-005021-76-IT
• Lead Sponsor: EDISON PHARMACEUTICALS INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-12-11
• Last Update Posted: 16 December 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Female
• Target Recruitment: Not specified

Inclusion Criteria

1.Diagnosis of Rett syndrome Stage 1-2 2.Abnormal disease biomarkers 3.Confirmed MeCP2 mutation 4.Patient or patient’s guardian able to consent and comply with protocol requirements 5.Abstention from use of Coenzyne Q10, vitamin E and Idebenone two weeks prior enrollment of treatment with EPI-743
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Any condition, which in the opinion of the investigator could compromise the subject’s safety or adherence to treatment with EPI-743 2.Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil) 3.Clinically significant allergy or hypersensitivity to VitaminE 4.Lack of confirmation of MeCP2 mutation 5.Clinical history of bleeding or abnormal baseline PT/PTT 6.Diagnosis of any other concurrent inborn error of metabolism 7.Hepatic insufficiency with LFTs greater than 3 times upper limit of normal 8.Renal insufficiency requiring dialysis 9.End stage cardiac failure 10.Fat malabsorption syndromes precluding drug absorption

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified