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

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Rett syndrome
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Brief Title

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

Official Title

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

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.

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Rett Syndrome Clinical Severity Sore

Secondary Outcome

 Oxidative Stress Biomarkers

Condition

Rett Syndrome

Intervention

EPI-743

Study Arms / Comparison Groups

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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

24

Start Date

January 2013

Completion Date

January 2014

Primary Completion Date

December 2013

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

Gender

Female

Ages

N/A - 18 Years

Accepts Healthy Volunteers

No

Contacts

, ,

Location Countries

Italy

Location Countries

Italy

Administrative Informations

NCT ID

NCT01822249

Organization ID

OPBGC&RS_12_003

Responsible Party

Sponsor

Study Sponsor

Edison Pharmaceuticals Inc

Study Sponsor

, ,

Verification Date

July 2018