Date: 2017-08-01
Type of
information: Treatment of the first patient
phase: 2
Announcement: treatment of the first patient
Company: Acceleron Pharma (USA - CA)
Product: ACE 083
Action
mechanism:
- protein. ACE-083 is a therapeutic candidate based on the naturally-occurring protein, follistatin, that acts as a ligand trap for members in the transforming growth factor-beta superfamily involved in regulating muscle growth and strength. ACE-083 has been designed to have a concentrated effect along targeted muscles to maximize growth and strength selectively in the muscles into which the drug is administered.
- Acceleron is developing ACE-083 for diseases such as facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease in which improved muscle strength in target muscles may provide a clinical benefit and enhance quality of life.
Disease: Charcot-Marie-Tooth Disease
Therapeutic
area: Rare diseases - Neurological diseases - Genetic diseases
Country: USA
Trial
details:
- The two-part Phase 2 clinical trial is designed to evaluate ACE-083 in Charcot-Marie-Tooth disease patients with muscle weakness in the tibialis anterior, a muscle in the lower leg involved in foot dorsiflexion (raising the foot at the ankle). Part 1 is an open-label, dose-escalation study, with ACE-083 administered by injection into the tibialis anterior muscle once every 3 weeks in up to 18 patients to evaluate safety and increases in muscle volume over a 3-month treatment period. Part 2 is a randomized, double-blind, placebo-controlled study using the optimal dose level selected in Part 1. A total of 24 patients will be randomized in Part 2 to receive either placebo or ACE-083 and will be evaluated for increases in muscle volume, strength, function and safety over a 3-month treatment period. (NCT03124459)
Latest
news:
- • On August 1, 2017, Acceleron Pharma announced that the first patient has been treated in a Phase 2 clinical trial of ACE-083 for the treatment of patients with Charcot-Marie-Tooth disease.
- • On March 16, 2017, Acceleron Pharma announced that in the second half of 2017 it plans to initiate a Phase 2 clinical trial of ACE-083 for the treatment of patients with Charcot-Marie-Tooth disease.
Is
general: Yes
