An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
Public ClinicalTrials.gov record NCT04535609. 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 Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of 24 Weeks Treatment With REN001 in Patients With Primary Mitochondrial Myopathy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study designed to investigate the efficacy and safety of REN001 administered once daily over a 24-week period to patients with PMM.
Study identification
- NCT ID
- NCT04535609
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 213 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- 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
- May 21, 2021 (Actual)
- Primary completion
- Sep 12, 2023 (Actual)
- Completion
- Oct 5, 2023 (Actual)
- Last update posted
- May 8, 2024
2021 – 2023
United States locations
- U.S. sites
- 9
- U.S. states
- 7
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, San Diego | La Jolla | California | 92093 | — |
| Myology Institute, University of Florida | Gainesville | Florida | 32608 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Columbia University Irving Medical Center | New York | New York | 10032 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| University of Texas SouthWestern Medical Center | Dallas | Texas | 75390 | — |
| Centre for the Treatment of Pediatric Neurodegenerative Disease, University of Texas McGovern Medical School | Houston | Texas | 77025 | — |
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 32 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04535609, 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 May 8, 2024 · Synced Nov 2, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04535609 live on ClinicalTrials.gov.