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

Rett Syndrome Clinical Trial

Official title:

A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01822249
• Other study ID #: OPBGC&RS_12_003
• Status: Completed
• Phase: Phase 2
• Start date: January 2013
• Est. completion date: January 2014

Study information

• Verified date: July 2018
• Source: Edison Pharmaceuticals Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: January 2014
• Est. primary completion date: December 2013
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Rett Syndrome
• Syndrome

Intervention

Drug:
• EPI-743

• Placebo

Locations

Country	Name	City	State
Italy	University of Siena	Siena

Sponsors (1)

Lead Sponsor	Collaborator
Edison Pharmaceuticals Inc

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rett Syndrome Clinical Severity Sore	Measure of disease progression	Change at six months from baseline
Secondary	Oxidative Stress Biomarkers		Change at six months from baseline
Secondary	Head circumference		Change at six months from baseline
Secondary	Rett syndrome behavioral questionnaire		Change at six months from baseline
Secondary	PedsQL		Change at six months from baseline
Secondary	Number of Drug-related adverse and serious adverse events		Six months
Secondary	Respiratory Disturbance Index (RDI)	RDI will be determined on polysomnography study	Change at six months from baseline