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

Study Identifier:
BHV2000-301
ClinicalTrials.gov Identifier:
NCT05337553
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Biohaven Pharmaceuticals, Inc.
Active, not recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: taldefgrobep alfa
  • Drug: Placebo
Date
Jul 2022 - Sep 2024
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 4 - 21 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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
Exclusion Criteria
  • 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

Protocol 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.

Trial Locations

Location
Status
Location
Phoenix Children's
Phoenix, Arizona, United States, 85016
Status
N/A
Location
UCSD & Rady Children's
La Jolla, California, United States, 92037
Status
N/A
Location
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Status
N/A
Location
UCSF Benioff Children's Hospital, Medical Center
San Francisco, California, United States, 94158
Status
N/A
Location
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Status
N/A
Location
UF Health, Shands Hospital
Gainesville, Florida, United States, 32610
Status
N/A
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