Status:
ACTIVE_NOT_RECRUITING
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Lead Sponsor:
Biohaven Pharmaceuticals, Inc.
Conditions:
Spinal Muscular Atrophy
Neuromuscular Diseases
Eligibility:
All Genders
4-21 years
Phase:
PHASE3
Brief 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 o...
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 t...
Eligibility Criteria
Inclusion
- Key
- 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 and/or risdiplam and/or a history of onasemnogene abeparvovec
- Key
Exclusion
- 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
Key Trial Info
Start Date :
July 6 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2026
Estimated Enrollment :
269 Patients enrolled
Trial Details
Trial ID
NCT05337553
Start Date
July 6 2022
End Date
January 1 2026
Last Update
August 26 2025
Active Locations (53)
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1
Phoenix Children's
Phoenix, Arizona, United States, 85016
2
UCSD & Rady Children's
La Jolla, California, United States, 92037
3
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
4
UCSF Benioff Children's Hospital, Medical Center
San Francisco, California, United States, 94158