drugset / Trial / NCT02927080
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
RandomizedParallel-groupQuadruple-blindTreatment
Summary
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy (FSHD) to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.
Timeline
- Start
- 2016-11
- Primary completion
- 2019-09-17
- Completion
- 2019-10-09
Outcome
Met primary endpoint
Stopped: “Study was discontinued as it did not achieve functional secondary endpoints.”
registry p = 0.0138; Mean Difference (Net) 9.54 (90% CI 3.17 to 15.92) NCT02927080 ↗
registry p <0.0001; Mean Difference (Net) 16.39 (90% CI 9.75 to 23.02) NCT02927080 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | EFMITERMANT ALFA | Protein / enzyme biologic | 150 mg | Intramuscular |
| Subject | EFMITERMANT ALFA | Protein / enzyme biologic | 200 mg | Intramuscular |
| Subject | EFMITERMANT ALFA | Protein / enzyme biologic | 240 mg | Intramuscular |