This study is in progress, not accepting new patients

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

a study on Spinal Muscular Atrophy Muscular Atrophy

Summary

Eligibility
for people ages 4-21 (full criteria)
Location
at UCSD UCSF
Dates
study started
completion around

Description

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.

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

Details

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.

Keywords

Spinal Muscular Atrophy, Neuromuscular Diseases, SMA, Muscular Atrophy, Atrophy, taldefgrobep alfa

Eligibility

You can join if…

Open to people ages 4-21

  • Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
  • Ambulant or Non-Ambulant
  • Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen, risdiplam, and/or a history of onasemnogene abeparvovec

You CAN'T join if...

  • Cannot have previously taken anti-myostatin therapies
  • Must weigh at least 15kg
  • Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)
  • History of Spinal Fusion within 6 months of Screening. MAGEC rod nonsurgical adjustments are allowed during the study
  • Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter

Locations

  • UCSD & Rady Children's
    La Jolla California 92037 United States
  • UCSF Benioff Children's Hospital, Medical Center
    San Francisco California 94158 United States
  • Children's Hospital of Los Angeles
    Los Angeles California 90027 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Biohaven Pharmaceuticals, Inc.
ID
NCT05337553
Phase
Phase 3 research study
Study Type
Interventional
Participants
About 269 people participating
Last Updated