Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
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ClinicalTrials.gov Identifier: NCT02758626 |
Recruitment Status :
Active, not recruiting
First Posted : May 2, 2016
Last Update Posted : October 23, 2020
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Sponsor:
NYU Langone Health
Collaborator:
PTC Therapeutics
Information provided by (Responsible Party):
NYU Langone Health
Tracking Information | ||||
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First Submitted Date ICMJE | April 4, 2016 | |||
First Posted Date ICMJE | May 2, 2016 | |||
Last Update Posted Date | October 23, 2020 | |||
Study Start Date ICMJE | November 2016 | |||
Estimated Primary Completion Date | January 2021 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE | Same as current | |||
Change History | ||||
Current Secondary Outcome Measures ICMJE |
Seizure frequency from baseline after ataluren treatment in patients w/ CDKL5 or Dravet from daily seizure diary. [ Time Frame: Baseline, 3 months, 6 months, and 1 year ] | |||
Original Secondary Outcome Measures ICMJE | Same as current | |||
Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Same as current | |||
Descriptive Information | ||||
Brief Title ICMJE | Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome | |||
Official Title ICMJE | A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet Syndrome | |||
Brief Summary | This is a phase 2, crossover study of Ataluren for the treatment of nonsense mutation Dravet syndrome or cyclin-dependent kinase-like 5 (CDKL5) deficiency, resulting in drug-resistant epilepsy. Patients will receive 12 weeks of ataluren or placebo during each treatment period. Treatment Period 1 will be followed by a 4-week Washout Period. Based on ataluren PK and pharmacodynamic data, the 4-week washout period is deemed an appropriate length of time to eliminate any ataluren drug effects. Following the Washout Period, patients will crossover to receive the opposite treatment during Treatment Period 2 as follows: Patients receiving ataluren during Treatment Period 1 will receive placebo during Treatment Period 2. Patients receiving placebo during Treatment Period 1 will receive ataluren during Treatment Period 2. | |||
Detailed Description | Investigators will try to characterize the safety profile of ataluren in patients with CDKL5 or Dravet syndrome resulting from a nonsense mutation and evaluate changes in convulsive and/or drop seizure frequency from Baseline following ataluren treatment in patients with CDKL5 or Dravet syndrome resulting from a nonsense mutation. Investigators will measure changes in minor seizure types (absence, myoclonic, complex partial/focal dyscognitive) following ataluren treatment in patients with CDKL5 or Dravet syndrome resulting from a nonsense mutation and changes from Baseline in cognitive, motor, and behavioral function as well as QOL following ataluren treatment in patients with CDKL5 or Dravet syndrome resulting from a nonsense mutation. | |||
Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 2 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Crossover Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Epilepsy | |||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Active, not recruiting | |||
Actual Enrollment ICMJE |
16 | |||
Original Estimated Enrollment ICMJE | Same as current | |||
Estimated Study Completion Date ICMJE | March 2021 | |||
Estimated Primary Completion Date | January 2021 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 2 Years to 12 Years (Child) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT02758626 | |||
Other Study ID Numbers ICMJE | 15-00426 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE |
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Responsible Party | NYU Langone Health | |||
Study Sponsor ICMJE | NYU Langone Health | |||
Collaborators ICMJE | PTC Therapeutics | |||
Investigators ICMJE |
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PRS Account | NYU Langone Health | |||
Verification Date | October 2020 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |