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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01822249
• Date of registration: 25/03/2013
• Prospective Registration: No
• Primary sponsor: Edison Pharmaceuticals Inc
• Public title: Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
• Scientific title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
• Date of first enrolment: January 2013
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01822249
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Italy

Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: 18 Years
Gender: Female

Health Condition(s) or Problem(s) studied

Rett Syndrome

Intervention(s)

Drug: Placebo

Drug: EPI-743

Primary Outcome(s)

Rett Syndrome Clinical Severity Sore [Time Frame: Change at six months from baseline]

Secondary Outcome(s)

Head circumference [Time Frame: Change at six months from baseline]

PedsQL [Time Frame: Change at six months from baseline]

Number of Drug-related adverse and serious adverse events [Time Frame: Six months]

Oxidative Stress Biomarkers [Time Frame: Change at six months from baseline]

Rett syndrome behavioral questionnaire [Time Frame: Change at six months from baseline]

Respiratory Disturbance Index (RDI) [Time Frame: Change at six months from baseline]

Secondary ID(s)

OPBGC&RS_12_003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors