New drug aims to build muscle in SMA patients

NCT ID NCT05337553

Ongoing Disease control Sponsor: Biohaven Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

Summary

This study is testing whether a new drug called taldefgrobep alfa can improve muscle function and strength in people with spinal muscular atrophy (SMA). The drug is given as a shot and is designed to block a protein that limits muscle growth. It is being tested in 269 people who are already receiving standard SMA treatments, to see if adding it provides extra benefit.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Locations

BSHS Office of Research

Grand Rapids, Michigan, 49503, United States
Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience

Roma, 00165, Italy
Boston Children's Hospital - Harvard

Boston, Massachusetts, 02115, United States
CHOP Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Columbia University Medical Center

New York, New York, 10032, United States
Cook Children's Hospital

Fort Worth, Texas, 76104, United States
Donostia University Hospital

Donostia / San Sebastian, 20014, Spain
Dr. Von Haunersches Children'S Hospital - Lmu Munich

Munich, 80337, Germany
Duke University Medicine

Durham, North Carolina, 27705, United States
Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic

Poznan, 60-355, Poland
Hospital Sant Joan de Déu

Esplugues de Llobregat, 08950, Spain
IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT

Pavia, 27100, Italy
Indiana University -Riley Research

Indianapolis, Indiana, 46202, United States
Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital

Bologna, 40139, Italy
John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom
La Fe University and Polytechnic Hospital

Valencia, 46026, Spain
Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit

Málaga, 29011, Spain
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Motol University Hospital

Prague, 150 06, Czechia
MultiCare Institute of Research and Innovation

Tacoma, Washington, 98405, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemo-Brescia Clinical Center For Neuromuscular Diseases

Gussago, 25064, Italy
Neurology Rare Disease Center

Denton, Texas, 75208, United States
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Penn State College of Medicine

Hershey, Pennsylvania, 17033, United States
Phoenix Children's

Phoenix, Arizona, 85016, United States
Rare Disease Research

Atlanta, Georgia, 30329, United States
Royal Hospital For Children

Glasgow, Scotland, G51 4TF, United Kingdom
Stony Brook University Hospital

Stony Brook, New York, 11794, United States
T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept.

Wroclaw, 54-049, Poland
The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology

Warsaw, 04-730, Poland
UCSD & Rady Children's

La Jolla, California, 92037, United States
UCSF Benioff Children's Hospital, Medical Center

San Francisco, California, 94158, United States
UF Health, Shands Hospital

Gainesville, Florida, 32610, United States
UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
UT Pediatric Neurosciences/Dell Children's Medical Center

Austin, Texas, 78723, United States
University Clinical Centre in Gdansk - Dept. of Developmental Neurology

Gdansk, 80-925, Poland
University Hospital Antwerp

Edegem, 02650, Belgium
University Hospital Brno - Dept. of Pediatric Neurology

Brno, 625 00, Czechia
University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I

Essen, 45147, Germany
University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders

Freiburg im Breisgau, 79106, Germany
University Hospital Ghent

Ghent, 09000, Belgium
University Hospital Leuven

Leuven, 03000, Belgium
University Medical Center Utrecht

Utrecht, 3584 CX, Netherlands
University of Iowa

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center

Fairway, Kansas, 66205, United States
University of Virginia Children's Hospital

Charlottesville, Virginia, 22903, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States
Washington University in St. Louis

St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.

NEUROMUSCULAR DISEASES SMA SPINAL MUSCULAR ATROPHY