Trial record 1 of 24 for:    Rett Syndrome
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Phase 2 Study of EPI-743 for Treatment of Rett Syndrome

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc Identifier:
First received: March 25, 2013
Last updated: May 17, 2013
Last verified: May 2013

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.

Condition Intervention Phase
Rett Syndrome
Drug: EPI-743
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome

Resource links provided by NLM:

Further study details as provided by Edison Pharmaceuticals Inc:

Primary Outcome Measures:
  • Rett Syndrome Clinical Severity Sore [ Time Frame: Change at six months from baseline ] [ Designated as safety issue: No ]
    Measure of disease progression

Secondary Outcome Measures:
  • Oxidative Stress Biomarkers [ Time Frame: Change at six months from baseline ] [ Designated as safety issue: No ]
  • Head circumference [ Time Frame: Change at six months from baseline ] [ Designated as safety issue: No ]
  • Rett syndrome behavioral questionnaire [ Time Frame: Change at six months from baseline ] [ Designated as safety issue: No ]
  • PedsQL [ Time Frame: Change at six months from baseline ] [ Designated as safety issue: No ]
  • Number of Drug-related adverse and serious adverse events [ Time Frame: Six months ] [ Designated as safety issue: Yes ]
  • Respiratory Disturbance Index (RDI) [ Time Frame: Change at six months from baseline ] [ Designated as safety issue: No ]
    RDI will be determined on polysomnography study

Enrollment: 24
Study Start Date: March 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: EPI-743 15 mg/kg
Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily
Drug: EPI-743
Placebo Comparator: 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


Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Please refer to this study by its identifier: NCT01822249

University of Siena
Siena, Italy
Sponsors and Collaborators
Edison Pharmaceuticals Inc
  More Information

No publications provided

Responsible Party: Edison Pharmaceuticals Inc Identifier: NCT01822249     History of Changes
Other Study ID Numbers: OPBGC&RS_12_003
Study First Received: March 25, 2013
Last Updated: May 17, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Edison Pharmaceuticals Inc:
Rett syndrome

Additional relevant MeSH terms:
Rett Syndrome
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn processed this record on April 17, 2014