Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Summary

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

Timeline

Start

2017-07-06

Primary completion

2020-04-28

Completion

2020-04-28

Outcome

Missed primary endpoint

paper The phase 2/3 trial found that the effects of treatment did not meet the primary endpoint pre-specified futility analysis threshold PMID 38190001 ↗

paper The futility analysis demonstrated that taldefgrobep alfa did not result in functional change for boys with DMD. PMID 38190001 ↗

Publications

• Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy. Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Taldefgrobep Alfa	Protein / enzyme biologic	—	Subcutaneous

Indications