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

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

269

Participants

Timeline

Start Date

July 6, 2022

Primary Completion Date

September 25, 2024

Study Completion Date

January 31, 2026

Conditions
Spinal Muscular AtrophyNeuromuscular DiseasesSMA
Interventions
DRUG

taldefgrobep alfa

DB Phase: 35 mg/50 mg weekly subcutaneous injection

DRUG

Placebo

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

DRUG

taldefgrobep alfa

Extension Phase: 35 mg/50 mg weekly subcutaneous injection

Trial Locations (53)

10032

Columbia University Medical Center, New York

11794

Stony Brook University Hospital, Stony Brook

15224

UPMC Children's Hospital of Pittsburgh, Pittsburgh

17033

Penn State College of Medicine, Hershey

19104

CHOP Children's Hospital of Philadelphia, Philadelphia

20014

Donostia University Hospital, Donostia / San Sebastian

22903

University of Virginia Children's Hospital, Charlottesville

23507

Children's Hospital of The King's Daughters, Norfolk

25064

Nemo-Brescia Clinical Center For Neuromuscular Diseases, Gussago

27100

IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT, Pavia

27705

Duke University Medicine, Durham

29011

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

30329

Rare Disease Research, Atlanta

32610

UF Health, Shands Hospital, Gainesville

37232

Vanderbilt University Medical Center, Nashville

40139

Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital, Bologna

43205

Nationwide Children's Hospital, Columbus

45147

University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I, Essen

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

46026

La Fe University and Polytechnic Hospital, Valencia

46202

Indiana University -Riley Research, Indianapolis

49503

BSHS Office of Research, Grand Rapids

52242

University of Iowa, Iowa City

53226

Medical College of Wisconsin, Milwaukee

60611

Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

63110

Washington University in St. Louis, St Louis

66205

University of Kansas Medical Center, Fairway

75208

Neurology Rare Disease Center, Denton

76104

Cook Children's Hospital, Fort Worth

78723

UT Pediatric Neurosciences/Dell Children's Medical Center, Austin

79106

University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders, Freiburg im Breisgau

80045

Children's Hospital Colorado, Aurora

80337

Dr. Von Haunersches Children'S Hospital - Lmu Munich, Munich

85016

Phoenix Children's, Phoenix

90027

Children's Hospital of Los Angeles, Los Angeles

92037

UCSD & Rady Children's, La Jolla

94158

UCSF Benioff Children's Hospital, Medical Center, San Francisco

98405

MultiCare Institute of Research and Innovation, Tacoma

02115

Boston Children's Hospital - Harvard, Boston

02650

University Hospital Antwerp, Edegem

09000

University Hospital Ghent, Ghent

03000

University Hospital Leuven, Leuven

625 00

University Hospital Brno - Dept. of Pediatric Neurology, Brno

150 06

Motol University Hospital, Prague

00165

Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience, Roma

3584 CX

University Medical Center Utrecht, Utrecht

80-925

University Clinical Centre in Gdansk - Dept. of Developmental Neurology, Gdansk

60-355

Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic, Poznan

04-730

The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology, Warsaw

54-049

T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept., Wroclaw

08950

Hospital Sant Joan de Déu, Esplugues de Llobregat

G51 4TF

Royal Hospital For Children, Glasgow

OX3 9DU

John Radcliffe Hospital, Oxford

Sponsors
All Listed Sponsors
lead

Biohaven Pharmaceuticals, Inc.

INDUSTRY