A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
NCT ID: NCT05337553
Last Updated: 2025-08-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
269 participants
INTERVENTIONAL
2022-07-06
2026-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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taldefgrobep alfa
taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase.
taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week Open label Extension (OLE) phase.
taldefgrobep alfa
DB Phase: 35 mg/50 mg weekly subcutaneous injection
taldefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Placebo
Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase.
Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for 48-week OLE phase.
Placebo
DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection
taldefgrobep alfa
Extension Phase: 35 mg/50 mg weekly subcutaneous injection
Interventions
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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
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
4 Years
21 Years
ALL
No
Sponsors
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Biohaven Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Lindsey Lair, MD
Role: STUDY_DIRECTOR
Biohaven Pharmaceuticals, Inc.
Locations
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Phoenix Children's
Phoenix, Arizona, United States
UCSD & Rady Children's
La Jolla, California, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
UCSF Benioff Children's Hospital, Medical Center
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
UF Health, Shands Hospital
Gainesville, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Indiana University -Riley Research
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Boston Children's Hospital - Harvard
Boston, Massachusetts, United States
BSHS Office of Research
Grand Rapids, Michigan, United States
Washington University in St. Louis
St Louis, Missouri, United States
Columbia University Medical Center
New York, New York, United States
Stony Brook University Hospital
Stony Brook, New York, United States
Duke University Medicine
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Penn State College of Medicine
Hershey, Pennsylvania, United States
CHOP Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
UT Pediatric Neurosciences/Dell Children's Medical Center
Austin, Texas, United States
Neurology Rare Disease Center
Denton, Texas, United States
Cook Children's Hospital
Fort Worth, Texas, United States
University of Virginia Children's Hospital
Charlottesville, Virginia, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
MultiCare Institute of Research and Innovation
Tacoma, Washington, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
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
IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT
Pavia, , Italy
Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience
Roma, , Italy
University Medical Center Utrecht
Utrecht, , Netherlands
University Clinical Centre in Gdansk - Dept. of Developmental Neurology
Gdansk, , Poland
Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic
Poznan, , Poland
The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology
Warsaw, , Poland
T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept.
Wroclaw, , Poland
Donostia University Hospital
Donostia / San Sebastian, , Spain
Hospital Sant Joan de Déu
Esplugues de Llobregat, , Spain
Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit
Málaga, , Spain
La Fe University and Polytechnic Hospital
Valencia, , Spain
Royal Hospital For Children
Glasgow, Scotland, United Kingdom
John Radcliffe Hospital
Oxford, , United Kingdom
Countries
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Other Identifiers
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BHV2000-301
Identifier Type: -
Identifier Source: org_study_id
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