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 26, 2018
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Condition or disease | Intervention/treatment | Phase |
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Rett Syndrome | Drug: EPI-743 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 24 participants |
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 |
Study Start Date : | January 2013 |
Actual Primary Completion Date : | December 2013 |
Actual Study Completion Date : | January 2014 |
Arm | Intervention/treatment |
---|---|
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
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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
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Drug: Placebo |
- Rett Syndrome Clinical Severity Sore [ Time Frame: Change at six months from baseline ]Measure of disease progression
- 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | up to 18 Years (Child, Adult) |
Sexes 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
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
Italy | |
University of Siena | |
Siena, Italy |
Responsible Party: | Edison Pharmaceuticals Inc |
ClinicalTrials.gov Identifier: | NCT01822249 |
Other Study ID Numbers: |
OPBGC&RS_12_003 |
First Posted: | April 2, 2013 Key Record Dates |
Last Update Posted: | July 26, 2018 |
Last Verified: | July 2018 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Rett syndrome EPI-743 Edison |
Rett Syndrome 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 |