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NCT04886518 · ClinicalTrials.gov registry record · Phase 2
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
A Phase 2 study, sponsored by Harmony Biosciences Management.
- Completed
- Registry status
- Phase 2
- Development phase
- 30
- Enrollment target
NCT04886518: Completed Phase 2 study, sponsored by Harmony Biosciences Management.
NCT04886518 is a Phase 2 study that has completed, run by Harmony Biosciences Management. The registered enrollment target is 30 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (77% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04886518, a Phase 2 study, has completed, sponsored by Harmony Biosciences Management.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 30 participants
- Enrollment target
Study Summary
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years. The secondary objectives of this study are to assess the impact of pitolisant on fatigue, cognitive function and the burden of disease along with assessing the long-term safety and effectiveness of pitolisant in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
Primary Outcome
The score of the DSS ranges from 0 to 15. A decrease in the DSS score represents an improvement in EDS.
Interventions
- DRUG Placebo oral tablet
- DRUG Pitolisant Oral Tablet
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2021-06-28 |
| Est. Completion | 2024-10-31 |
| Phase | Phase 2 |
What the finished NCT04886518 record still lists
NCT04886518 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (77% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo oral tablet is the first listed.
NCT04886518 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04886518 about?
NCT04886518 is a clinical study titled "Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1". The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years. The secondary objectives of this study are to assess the impact of pitolisant...
What is the current status of trial NCT04886518?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 30 participants. The study started on 2021-06-28. Estimated completion is 2024-10-31.
What interventions are being tested in trial NCT04886518?
The interventions under investigation include: Placebo oral tablet (DRUG), Pitolisant Oral Tablet (DRUG).
Who is sponsoring clinical trial NCT04886518?
This trial is sponsored by Harmony Biosciences Management, which has 10 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04886518's enrollment target sits among peer trials
30 1582nd of 2000 higher than 332 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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