NCT05337553

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

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 269 (actual)
• Sponsor: Biohaven Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 4 Years – 21 Years
• Healthy volunteers: Not accepted

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 and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.

Detailed 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.

Conditions

• Spinal Muscular Atrophy
• Neuromuscular Diseases
• SMA

Interventions

Type	Name	Description
DRUG	taldefgrobep alfa	DB Phase: 35 mg/50 mg weekly subcutaneous injection
DRUG	Placebo	DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection
DRUG	taldefgrobep alfa	Extension Phase: 35 mg/50 mg weekly subcutaneous injection

Timeline

• Start date: 2022-07-06
• Primary completion: 2024-09-25
• Completion: 2026-06-01
• First posted: 2022-04-20
• Last updated: 2026-02-04

Locations

53 sites across 9 countries: United States, Belgium, Czechia, Germany, Italy, Netherlands, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study