Acceleron starts Phase II trial of ACE-083 to treat Charcot-Marie-Tooth disease

Acceleron Pharma Inc. (NASDAQ:XLRN) began a Phase II trial of ACE-083 to treat Charcot-Marie-Tooth disease. The trial’s primary endpoints are safety for the first part and change from baseline in muscle volume for the second part; secondary endpoints include change from baseline in intramuscular fat tissue, muscle strength, walk/run time, walk distance, balance and fall risk, clinical examination score and quality of life.

In the open-label first part, up to 18 patients will receive 150, 200, or 250 mg intramuscular ACE-083 every 3 weeks for 3 months. The double-blind, placebo-controlled part will enroll 24 patients to receive 3 months of treatment with the optimal dose from the first part...