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A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy — RESILIENT  

Neuromuscular Diseases Clinical Trial

Official title:
A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension

Clinical Trial Summary

This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.

Clinical Trial Description

Myostatin is a negative regulator of muscle growth. Blocking myostatin activity has been shown to increase muscle size and function. Taldefgrobep alfa directly blocks myostatin activity and was well tolerated in other clinical studies. In combination with medications that increase the amount of SMN protein in the body, taldefgrobep alfa has the potential to further improve motor function and clinical measures for people living with SMA. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05337553
Study type Interventional
Source Biohaven Pharmaceuticals, Inc.
Contact
Status Active, not recruiting
Phase Phase 3
Start date July 6, 2022
Completion date January 2025
View Details
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