Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
Public ClinicalTrials.gov record NCT04886518. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04886518
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 30 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 27, 2021
- Primary completion
- Oct 5, 2023
- Completion
- Oct 30, 2024
- Last update posted
- Sep 3, 2026
2021 – 2024
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCI Center for Clinical Research | Irvine | California | 92697 | — |
| University of Colorado School of Medicine | Aurora | Colorado | 80045 | — |
| University of South Florida | Tampa | Florida | 33612 | — |
| Rare Disease Research | Atlanta | Georgia | 30329 | — |
| Indiana University | Indianapolis | Indiana | 46202 | — |
| Kennedy Krieger Institute Center for Genetic Muscle Disorders | Baltimore | Maryland | 21205 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| University of Rochester Medical Center | Rochester | New York | 14642 | — |
| University of North Carolina | Chapel Hill | North Carolina | 27599 | — |
| Wake Forest | Winston-Salem | North Carolina | 27157 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04886518, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 3, 2026 · Synced Sep 7, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04886518 live on ClinicalTrials.gov.