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

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ClinicalTrials.gov Identifier: NCT01822249
Recruitment Status : Completed
First Posted : April 2, 2013
Last Update Posted : July 25, 2018
Sponsor:
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Tracking Information
First Submitted Date  ICMJE March 25, 2013
First Posted Date  ICMJE April 2, 2013
Last Update Posted Date July 25, 2018
Study Start Date  ICMJE January 2013
Actual Primary Completion Date December 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 27, 2013)
Rett Syndrome Clinical Severity Sore [ Time Frame: Change at six months from baseline ]
Measure of disease progression
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01822249 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 1, 2013)
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: March 27, 2013)
  • 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 ]
  • 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
Official Title  ICMJE 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Rett Syndrome
Intervention  ICMJE
  • Drug: EPI-743
  • Drug: Placebo
Study Arms  ICMJE
  • 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
    Intervention: 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
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 17, 2013)
24
Original Estimated Enrollment  ICMJE
 (submitted: March 27, 2013)
20
Actual Study Completion Date  ICMJE January 2014
Actual Primary Completion Date December 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE up to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01822249
Other Study ID Numbers  ICMJE OPBGC&RS_12_003
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Edison Pharmaceuticals Inc
Study Sponsor  ICMJE Edison Pharmaceuticals Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Edison Pharmaceuticals Inc
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP