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ACTIVE NOT RECRUITING

NCT05626855

PHASE3

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

Sponsor: Scholar Rock, Inc.

View on ClinicalTrials.gov

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.

Official title: An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

Key Details

Gender

All

Age Range

2 Years - Any

Study Type

INTERVENTIONAL

Enrollment

238

Start Date

2023-04-17

Completion Date

2029-05-02

Last Updated

2025-05-25

Healthy Volunteers

Conditions

Spinal Muscular Atrophy Spinal Muscular Atrophy Type 3 Spinal Muscular Atrophy Type 2 SMA Neuromuscular Diseases Muscular Atrophy Atrophy Muscular Atrophy, Spinal Neuromuscular Manifestations Anti-myostatin

Interventions

DRUG

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.

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

Locations (49)

Phoenix Childrens Hospital

Phoenix, Arizona, United States

UCSD Altman Clinical and Translational Research

La Jolla, California, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

Stanford Neuroscience Health Center

Palo Alto, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Biomedical Research

Orlando, Florida, United States

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

University of Iowa

Iowa City, Iowa, United States

University of Kansas Medical Center

Fairway, Kansas, United States

Johns HopkinsHospital

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, United States

Washington University Medical Campus

St Louis, Missouri, United States

Columbia University Medical Center

New York, New York, United States

Wake Forest University School of Medicine

Winston-Salem, North Carolina, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

University of Texas Southwestern - Pediatric Neurology

Dallas, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Children's Specialty Group PLLC (Children's Hospital of The King's Daughters)

Newport News, Virginia, United States

Seattle Children's Hospital

Seattle, Washington, United States

University of Wisconsin Hospital

Madison, Wisconsin, United States

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

UMC Utrecht

Utrecht, Netherlands

Uniwersyteckie Centrum Kliniczne

Gdansk, Poland

Uniwersytecki Szpital Kliniczny w Poznaniu, Oddział Kliniczny Neurologii Dzieci i Młodzieży

Poznan, Poland

Instytut Pomnik - Centrum Zdrowia Dziecka

Warsaw, Poland

Hospital Sant Joan de Deau

Barcelona, Spain

Hospital Universitari i Politecnic La Fe

Valencia, Spain

Leeds Children's Hospital Clinical Research

Leeds, United Kingdom

Great Ormond Street Hospital for Children

London, United Kingdom

University of Oxford

Oxford, United Kingdom