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)
Stopped early · Phase 2
Conditions studied: Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
In brief
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.
Key facts
- Study ID
- NCT03943290
- Run by
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- People needed
- 62
- Starts
- 2019-05-10
- Expected to finish
- 2020-03-11
- Last updated by the study team
- 2022-09-26
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California-Irvine — Orange, California, United States
- University of California Davis Medical Center — Sacramento, California, United States
- University of Colorado — Aurora, Colorado, United States
- University of Florida — Gainesville, Florida, United States
- Indiana University — Indianapolis, Indiana, United States
- University of Iowa — Iowa City, Iowa, United States
- University of Kansas Medical Center — Kansas City, Kansas, United States
- Brigham & Women's Hospital — Boston, Massachusetts, United States
- University of Minnesota — Minneapolis, Minnesota, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Hackensack University Medical Center — Hackensack, New Jersey, United States
- Columbia University — New York, New York, United States
- University of Rochester School of Medicine — Rochester, New York, United States
- Carolinas Healthcare System Neurosciences Institute — Charlotte, North Carolina, United States
- Duke University Medical Center — Durham, North Carolina, United States
- Oregon Health & Science University — Portland, Oregon, United States
- University of Pennsylvania — Philadelphia, Pennsylvania, United States
- University of Utah — Salt Lake City, Utah, United States
- Virginia Commonwealth University — Richmond, Virginia, United States
- University of Calgary — Calgary, Alberta, Canada
- London Health Sciences Centre — London, Ontario, Canada
- Montreal Neurological Institute & Hospital — Montreal, Quebec, Canada
- Hospital de la Santa Creu i Sant Pau — Barcelona, Spain
- Hospital Universitario Vall d'Hebrón — Barcelona, Spain
Full record on ClinicalTrials.gov
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