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

NCT05337553 · tracked via the Priya Life Science UK tracker

Sponsor

Biohaven Pharmaceuticals, Inc.

Phase

Phase 3

Started

2022-07-06

Last updated

2026-06-01

Condition(s) studied

Spinal Muscular AtrophyNeuromuscular DiseasesSMA

Investigational drug(s) / intervention(s)

taldefgrobep alfaPlacebotaldefgrobep alfa

taldefgrobep alfa: DB Phase: 35 mg/50 mg weekly subcutaneous injection

Placebo: DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection

taldefgrobep alfa: Extension Phase: 35 mg/50 mg weekly subcutaneous injection

Study 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.

Eligibility

Sex

ALL

Min age

4 Years

Max age

21 Years

Healthy volunteers

Key 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 Key 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

Primary outcome measure(s)

Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score — Baseline to Week 48
Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The scores from the 32 items are summed and transformed to a 0-100 scale, with higher scores reflecting higher levels of functional abilities.

Trial sites (53)

Facility	City	Region
Phoenix Children's	Phoenix	Arizona
UCSD & Rady Children's	La Jolla	California
Children's Hospital of Los Angeles	Los Angeles	California
UCSF Benioff Children's Hospital, Medical Center	San Francisco	California
Children's Hospital Colorado	Aurora	Colorado
UF Health, Shands Hospital	Gainesville	Florida
Rare Disease Research	Atlanta	Georgia
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
Indiana University -Riley Research	Indianapolis	Indiana
University of Iowa	Iowa City	Iowa
University of Kansas Medical Center	Fairway	Kansas
Boston Children's Hospital - Harvard	Boston	Massachusetts
BSHS Office of Research	Grand Rapids	Michigan
Washington University in St. Louis	St Louis	Missouri
Columbia University Medical Center	New York	New York
Stony Brook University Hospital	Stony Brook	New York
Duke University Medicine	Durham	North Carolina
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
Nationwide Children's Hospital	Columbus	Ohio
Penn State College of Medicine	Hershey	Pennsylvania
CHOP Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
Vanderbilt University Medical Center	Nashville	Tennessee
UT Pediatric Neurosciences/Dell Children's Medical Center	Austin	Texas
Neurology Rare Disease Center	Denton	Texas
Cook Children's Hospital	Fort Worth	Texas
University of Virginia Children's Hospital	Charlottesville	Virginia
Children's Hospital of The King's Daughters	Norfolk	Virginia
MultiCare Institute of Research and Innovation	Tacoma	Washington
Medical College of Wisconsin	Milwaukee	Wisconsin
University Hospital Antwerp	Edegem	Belgium
University Hospital Ghent	Ghent	Belgium
University Hospital Leuven	Leuven	Belgium
University Hospital Brno - Dept. of Pediatric Neurology	Brno	Czechia
Motol University Hospital	Prague	Czechia
University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I	Essen	Germany
University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders	Freiburg im Breisgau	Germany
Dr. Von Haunersches Children'S Hospital - Lmu Munich	Munich	Germany
Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital	Bologna	Italy
Nemo-Brescia Clinical Center For Neuromuscular Diseases	Gussago	Italy

+ 13 more sites — see the full list on the official registry below.

Other trials for the same condition

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi Active, not recruiting

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Active, not recruiting

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy Recruiting

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients Recruiting

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05337553 on ClinicalTrials.gov ↗ ← All trials in the UK