Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

PHASE3CompletedINTERVENTIONAL
Enrollment

188

Participants

Timeline

Start Date

April 14, 2022

Primary Completion Date

December 18, 2024

Study Completion Date

December 18, 2024

Conditions
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2SMANeuromuscular DiseasesMuscular AtrophyAtrophyMuscular Atrophy, SpinalNeuromuscular ManifestationsAnti-myostatin
Interventions
DRUG

Apitegromab

Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 was administered every 4 weeks by intravenous (IV) infusion.

DRUG

Placebo

Placebo was administered every 4 weeks by intravenous (IV) infusion.

Trial Locations (50)

3000

UZ Leuven, Leuven

3584

Universitair Medisch Centrum Utrecht, Utrecht

4000

Chr de La Citadelle, Liège

9000

UZ Gent, Ghent

10032

Columbia University, SMA Clinical Research Center, New York

19104

Childrens Hospital of Philadelphia, Philadelphia

20133

Foundation I.R.C.C.S. Carlo Besta Neurological Institute, Milan

20162

ASST Grande Ospedale Metropolitano Niguarda, Milan

21287

The Johns Hopkins University, Baltimore

23507

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

27157

Wake Forest Baptist Medical Center, Winston-Salem

31059

CHU Toulouse - Hopital des Enfants, Toulouse

32827

Nemours Children's Hospital, Orlando

35233

Children's of Alabama, Birmingham

38105

St. Jude Children's Research Hospital, Memphis

43205

Nationwide Children's Hospital, Columbus

45147

Universitätsklinikum Essen, Essen

49503

Helen DeVos Children's Hospital, Grand Rapids

52242

University of Iowa, Iowa City

53127

Universitätsklinikum Bonn, Bonn

53705

University of Wisconsin School of Medicine and Public Health, Madison

55101

Gillette Children's Specialty Healthcare, Saint Paul

59037

CHU Lille - Hôpital Jeanne de Flandre, Lille

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

63110

Washington University School of Medicine in St. Louis, St Louis

66160

University of Kansas Medical Center, Kansas City

75012

Hôpital Armand Trousseau, I-Motion, Paris

75235

Children's Medical Center Dallas, Dallas

79106

Universitaetsklinikum Freiburg, Freiburg im Breisgau

80045

Children's Hospital Colorado, Aurora

80337

Dr. von Haunersches Kinderspital, Munich

84112

University of Utah, Salt Lake City

85016

Phoenix Children's Hospital, Phoenix

90027

Children's Hospital of Los Angeles, Los Angeles

92123

Rady's Children's Hospital/UCSD, San Diego

94304

Stanford University Medical Center, Palo Alto

97239

Oregon Health & Sciences University, Portland

98105

Seattle Children's Hospital, Seattle

98125

A.O.U Policlinico G. Martino, Messina

02115

Boston Children's Hospital, Boston

Unknown

IRCCS Istituto Giannina Gaslini, Genoa

Hospital Sant Joan de Déu, Barcelona

Hospital Universitario y Politécnico La Fe, Valencia

Great Ormond Street Hospital for Children NHS Foundation Trust, London

00168

Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore, Roma

80-001

Uniwersyteckie Centrum Kliniczne w Gdańsku, Gdansk

61-701

Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu, Poznan

04-736

Instytut Pomnik - Centrum Zdrowia Dziecka: CZD Warszawa, Warsaw

LS1 3EX

Leeds Teaching Hospitals NHS Trust, Leeds

OX3 0ER

University of Oxford, Oxford

All Listed Sponsors
lead

Scholar Rock, Inc.

INDUSTRY