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Clinical Trials in the UK / NCT05626855
Active, not recruiting Phase 3

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

NCT05626855 · tracked via the Priya Life Science UK tracker
Sponsor
Scholar Rock, Inc.
Phase
Phase 3
Started
2023-04-17
Last updated
2026-09-01

Condition(s) studied

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

Investigational drug(s) / intervention(s)

Apitegromab

Apitegromab: Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength.

Study summary

The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.

Eligibility

Sex
ALL
Min age
2 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Patients have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or the Phase 3 SAPPHIRE (Study SRK-015-003) trial. (For TOPAZ, completed is defined as completion of Visit EC14 in Extension Period C or participating in TOPAZ at the time the trial is ended. For SAPPHIRE, completed is defined as completion of Visit 14 or participating in SAPPHIRE at the time the trial is ended) * Estimated life expectancy \>2 years from the Baseline Visit (Day 1) * Able to receive study drug infusions and provide blood samples through the use of a peripheral IV or a long-term IV access device that the patient has placed for reasons independent from the trial * Able to adhere to the requirements of the protocol, including travel to the trial site and completing all trial procedures and trial visits * Females of childbearing potential must have a negative pregnancy test at the Baseline Visit and agree to use at least 1 highly effective method of contraception throughout the trial and for 20 weeks after the last dose of apitegromab Exclusion Criteria: * Patient permanently discontinued study treatment during the feeder trial (i.e., TOPAZ or SAPPHIRE) * Nutritional status that was not stable over the past 6 months and is not anticipated to be stable throughout the trial or medical necessity for a gastric/nasogastric feeding tube, where the majority of feeds are given by this route, as assessed by the Investigator * Patient is currently enrolled in any investigational drug trial other than TOPAZ or SAPPHIRE * Prior history of severe hypersensitivity reaction or intolerance to SMN-targeted therapies * Prior history of severe hypersensitivity reaction or intolerance to apitegromab * Use of chronic daytime noninvasive ventilatory support for \>16 hours daily in the 2 weeks before dosing, or anticipated to regularly receive such daytime ventilator support chronically throughout the trial * Any acute or comorbid condition interfering with the well-being of the patient at the patient's last visit in TOPAZ or SAPPHIRE, (including active systemic infection, the need for acute treatment, or inpatient observation due to any reason). After resolution of the condition, the patient can be enrolled in the trial if they meet all the other eligibility criteria. * Pregnant or breastfeeding * Any other condition or clinically significant laboratory result or ECG value that, in the opinion of the Investigator, may compromise safety or compliance, would preclude the patient from successful completion of the trial, or interfere with the interpretation of the results

Primary outcome measure(s)

Trial sites (49)

FacilityCityRegionStatus
Phoenix Childrens Hospital Phoenix Arizona
UCSD Altman Clinical and Translational Research La Jolla California
Children's Hospital Los Angeles Los Angeles California
Stanford Neuroscience Health Center Palo Alto California
Children's Hospital Colorado Aurora Colorado
Nemours Biomedical Research Orlando Florida
Lurie Children's Hospital of Chicago Chicago Illinois
University of Iowa Iowa City Iowa
University of Kansas Medical Center Fairway Kansas
Johns Hopkins Hospital Baltimore Maryland
Boston Children's Hospital Boston Massachusetts
Helen DeVos Children's Hospital Grand Rapids Michigan
Gillette Children's Specialty Healthcare Saint Paul Minnesota
Washington University Medical Campus St Louis Missouri
Columbia University Medical Center New York New York
Wake Forest University School of Medicine Winston-Salem North Carolina
Nationwide Children's Hospital Columbus Ohio
Oregon Health & Science University Portland Oregon
The Children's Hospital of Philadelphia Philadelphia Pennsylvania
St. Jude Children's Research Hospital Memphis Tennessee
University of Texas Southwestern - Pediatric Neurology Dallas Texas
University of Utah Salt Lake City Utah
Children's Specialty Group PLLC (Children's Hospital of The King's Daughters) Newport News Virginia
Seattle Children's Hospital Seattle Washington
University of Wisconsin Hospital Madison Wisconsin
UZ Gent Ghent Belgium
UZ Leuven Leuven Belgium
CHR Citadelle Liège Belgium
CHRU de Lille - Hpital Jeanne de Flandre Lille France
Hopital Trousseau - I-Motion Paris France
CHU Toulouse Hopital des Enfants Toulouse France
Universitätskinderklinik Bonn, Abteilung für Neuropädiatrie und SPZ Bonn Germany
Universitatsklinikum Essen Essen Germany
Universitatsklinikum Freiburg Freiburg im Breisgau Germany
Klinikum der Universitat Munchen, Dr. von Haunersches Kinderspital, Abteilung fur Kinderneurologie und Entwicklungsneurologie Munchen, Bayern Germany
Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative Genova Italy
UOC NEUROLOGIA E MALATTIE NEUROMUSCOLARI A.O.U Policlinico G. Martino Messina Italy
Carlo Besta Neurological Research Institute Milan Italy
NeuroMuscular Omnicentre Milan Italy
Fondazione Policlinico Universitario A. Gemelli Roma Italy

+ 9 more sites — see the full list on the official registry below.

More Scholar Rock, Inc. trials in the UK

Other trials for the same condition

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 NCT05626855 on ClinicalTrials.gov ↗ ← All trials in the UK