Efmitermant alfa
- Generic Name
- Efmitermant alfa
- Drug Type
- Biotech
- CAS Number
- 1644543-31-6
- Unique Ingredient Identifier
- 566QNS333V
- Background
Efmitermant alfa, a locally-acting GDF/activin ligand trap, is under investigation in clinical trial NCT03124459 (Study of ACE-083 in Patients With Charcot-marie-tooth Disease).
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)
- Conditions
- Facioscapulohumeral Muscular DystrophyCharcot-Marie-Tooth Disease
- Interventions
- First Posted Date
- 2019-05-09
- Last Posted Date
- 2022-09-26
- Lead Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Target Recruit Count
- 62
- Registration Number
- NCT03943290
- Locations
- 🇺🇸
University of Florida, Gainesville, Florida, United States
🇪🇸Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
🇪🇸Hospital Universitario Vall d'Hebrón, Barcelona, Spain
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
- First Posted Date
- 2017-04-21
- Last Posted Date
- 2022-09-26
- Lead Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Target Recruit Count
- 63
- Registration Number
- NCT03124459
- Locations
- 🇺🇸
University of Vermont, Burlington, Vermont, United States
🇺🇸University of Colorado, Aurora, Colorado, United States
🇺🇸University of California-Irvine, Orange, California, United States
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Interventions
- Drug: ACE-083 or placebo
- First Posted Date
- 2016-10-06
- Last Posted Date
- 2022-09-26
- Lead Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Target Recruit Count
- 95
- Registration Number
- NCT02927080
- Locations
- 🇺🇸
University of California Los Angeles Medical Center, Los Angeles, California, United States
🇺🇸Indiana University School of Medicine, Indianapolis, Indiana, United States
🇺🇸Johns Hopkins Hugo W. Moser Research Inst. at Kennedy Krieger Inc., Baltimore, Maryland, United States
Phase 1 Study of ACE-083 in Healthy Subjects
- First Posted Date
- 2014-10-06
- Last Posted Date
- 2022-09-23
- Lead Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Target Recruit Count
- 58
- Registration Number
- NCT02257489
- Locations
- 🇺🇸
Acceleron Investigative Site, Lincoln, Nebraska, United States
Drug Development Updates
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