Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
Public ClinicalTrials.gov record NCT03943290. 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
An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients With Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX previously enrolled in Study A083-03. This study will be conducted in two Parts: Part 1, which is a loading phase of 6 months' duration, and Part 2, the maintenance phase, which will last up to 24 months.
Study identification
- NCT ID
- NCT03943290
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Industry
- Enrollment
- 62 participants
Conditions and interventions
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 10, 2019 (Actual)
- Primary completion
- Mar 11, 2020 (Actual)
- Completion
- Mar 11, 2020 (Actual)
- Last update posted
- Sep 26, 2022
2019 – 2020
United States locations
- U.S. sites
- 19
- U.S. states
- 16
- U.S. cities
- 19
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California-Irvine | Orange | California | 92697 | — |
| University of California Davis Medical Center | Sacramento | California | 95817 | — |
| University of Colorado | Aurora | Colorado | 80045 | — |
| University of Florida | Gainesville | Florida | 32611 | — |
| Indiana University | Indianapolis | Indiana | 46202 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Brigham & Women's Hospital | Boston | Massachusetts | 02115 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Columbia University | New York | New York | 10027 | — |
| University of Rochester School of Medicine | Rochester | New York | 14642 | — |
| Carolinas Healthcare System Neurosciences Institute | Charlotte | North Carolina | 28207 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
| Virginia Commonwealth University | Richmond | Virginia | 23284 | — |
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 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03943290, 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 26, 2022 · Synced Oct 20, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03943290 live on ClinicalTrials.gov.