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

Primary Purpose

Rett Syndrome

Status
Completed
Phase
Phase 2
Locations
Italy
Study Type
Interventional
Intervention
EPI-743
Placebo
Sponsored by
Edison Pharmaceuticals Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Rett Syndrome focused on measuring Rett syndrome, EPI-743, Edison

Eligibility Criteria

undefined - 18 Years (Child, Adult)FemaleDoes not accept healthy volunteers

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

Sites / Locations

  • University of Siena

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

EPI-743 15 mg/kg

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

Rett Syndrome Clinical Severity Sore
Measure of disease progression

Secondary Outcome Measures

Oxidative Stress Biomarkers
Head circumference
Rett syndrome behavioral questionnaire
PedsQL
Number of Drug-related adverse and serious adverse events
Respiratory Disturbance Index (RDI)
RDI will be determined on polysomnography study

Full Information

First Posted
March 25, 2013
Last Updated
July 23, 2018
Sponsor
Edison Pharmaceuticals Inc
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1. Study Identification

Unique Protocol Identification Number
NCT01822249
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2018
Overall Recruitment Status
Completed
Study Start Date
January 2013 (undefined)
Primary Completion Date
December 2013 (Actual)
Study Completion Date
January 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Edison Pharmaceuticals Inc

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Rett Syndrome
Keywords
Rett syndrome, EPI-743, Edison

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
EPI-743 15 mg/kg
Arm Type
Active Comparator
Arm Description
Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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 Type
Drug
Intervention Name(s)
EPI-743
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Rett Syndrome Clinical Severity Sore
Description
Measure of disease progression
Time Frame
Change at six months from baseline
Secondary Outcome Measure Information:
Title
Oxidative Stress Biomarkers
Time Frame
Change at six months from baseline
Title
Head circumference
Time Frame
Change at six months from baseline
Title
Rett syndrome behavioral questionnaire
Time Frame
Change at six months from baseline
Title
PedsQL
Time Frame
Change at six months from baseline
Title
Number of Drug-related adverse and serious adverse events
Time Frame
Six months
Title
Respiratory Disturbance Index (RDI)
Description
RDI will be determined on polysomnography study
Time Frame
Change at six months from baseline

10. Eligibility

Sex
Female
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
University of Siena
City
Siena
Country
Italy

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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