Clinical Trial: Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy

Brief Summary: Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

Detailed Summary:

Part 1 (dose escalation, open-label) Part 1 will consist of up to 6 cohorts (A to F) of patients and will evaluate multiple ascending dose levels of ACE-083 in either the tibialis anterior (TA) or biceps brachii (BB) muscle. Patients in each cohort will be enrolled in a 4-week screening period before beginning treatment. A Safety Review Team (SRT) will meet to review data for each cohort when at least 4 patients within a cohort have completed their Day 43 visit prior to dose escalation.

Part 2 (randomized, double-blind, placebo-controlled) Prior to the initiation of Part 2, a review of safety and efficacy data from Part 1 will be conducted to determine whether cohorts for one or both muscles will be pursued in Part 2, as well as the recommended dose level for each muscle. A total of up to 40 new patients (20 patients per muscle) may be enrolled and randomized (3:2) to receive either ACE-083 (n=12) or placebo (n=8) unilaterally or bilaterally (if both sides are affected per inclusion criteria) to either the TA or BB muscles (but not both). The SRT will meet to review Part 2 patient safety data after the first 10 patients reach Day 43 in the study and approximately every 3 months thereafter.

Study duration for Parts 1 and 2 for each patient will be approximately 24 weeks, including a 4-week screening period, a 12-week treatment period, and an 8-week follow-up period after the last dose


Sponsor: Acceleron Pharma, Inc.

Current Primary Outcome:

  • Part 1: Safety and Tolerability (adverse events) [ Time Frame: From initiation of treatment (Study Day 1) to end of follow-up period (Study Day 141). ]
  • Part 2: Percent change from baseline in muscle volume of injected muscle by MRI [ Time Frame: From initiation of treatment (Study Day 1) to end of follow-up period (Study Day 141). ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Estimation of systemic exposure to ACE-083 by quantitative LC-MS assay of serum samples following local intramuscular administration [ Time Frame: From initiation of treatment (Study Day 1) to end of follow-up period (Study Day 141) ]
  • Percent change from baseline in strength of injected muscle by quantitative muscle testing [ Time Frame: From initiation of treatment (Study Day 1) to end of treatment visit (Study Day 106) ]
  • Percent change from baseline in function of injected muscle by motor function tests [ Time Frame: From initiation of treatment (Study Day 1) to end of treatment visit (Study Day 106) ]
  • Change from baseline in Health-Related-Quality-of-Life (HRQoL) by FSHD-Health Index questionnaire [ Time Frame: From initiation of treatment (Study Day 1) to end of treatment visit (Study Day 106) ]


Original Secondary Outcome: Same as current

Information By: Acceleron Pharma, Inc.

Dates:
Date Received: October 5, 2016
Date Started: November 2016
Date Completion: April 2019
Last Updated: May 16, 2017
Last Verified: May 2017