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NCT02758626 · ClinicalTrials.gov registry record · Phase 2
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
A Phase 2 study, sponsored by NYU Langone Health.
- Completed
- Registry status
- Phase 2
- Development phase
- 15
- Enrollment target
NCT02758626: Completed Phase 2 study, sponsored by NYU Langone Health.
NCT02758626 is a Phase 2 study that has completed, run by NYU Langone Health. The registered enrollment target is 15 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (89% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02758626, a Phase 2 study, has completed, sponsored by NYU Langone Health.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 15 participants
- Enrollment target
Study 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.
Primary Outcome
Convulsive seizure frequency per 28 days is defined as total number of convulsive seizures reported during the period divided by number of days during the period seizures were assessed, multiplied by 28. Percent change from Baseline will be defined as the frequency of seizures per 28 days during the Ataluren Treatment Period minus frequency of seizures per 28 days at Baseline, divided by the frequency of seizures per 28 days at Baseline, multiplied by 100. Negative percent change from Baseline i
Interventions
- DRUG Placebo
- DRUG ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2016-11 |
| Est. Completion | 2021-02-27 |
| Phase | Phase 2 |
What the finished NCT02758626 record still lists
NCT02758626 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (89% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT02758626 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02758626 about?
NCT02758626 is a clinical study titled "Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome". 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 wil...
What is the current status of trial NCT02758626?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 15 participants. The study started on 2016-11. Estimated completion is 2021-02-27.
What interventions are being tested in trial NCT02758626?
The interventions under investigation include: Placebo (DRUG), ataluren (DRUG).
Who is sponsoring clinical trial NCT02758626?
This trial is sponsored by NYU Langone Health, which has 1,164 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02758626's enrollment target sits among peer trials
15 1919th of 2000 higher than 60 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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