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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01822249
First Posted: April 2, 2013
Last Update Posted: August 4, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc
  Purpose
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
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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 ]
    Measure of disease progression


Secondary Outcome Measures:
  • Oxidative Stress Biomarkers [ Time Frame: Change at six months from baseline ]
  • Head circumference [ Time Frame: Change at six months from baseline ]
  • Rett syndrome behavioral questionnaire [ 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 ]
  • Respiratory Disturbance Index (RDI) [ Time Frame: Change at six months from baseline ]
    RDI will be determined on polysomnography study


Enrollment: 24
Study Start Date: January 2013
Study Completion Date: January 2014
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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01822249


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

Responsible Party: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT01822249     History of Changes
Other Study ID Numbers: OPBGC&RS_12_003
First Submitted: March 25, 2013
First Posted: April 2, 2013
Last Update Posted: August 4, 2015
Last Verified: August 2015

Keywords provided by Edison Pharmaceuticals Inc:
Rett syndrome
EPI-743
Edison

Additional relevant MeSH terms:
Syndrome
Rett Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Ubiquinone
Micronutrients
Growth Substances
Physiological Effects of Drugs