Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
NCT ID: NCT05626855
Last Updated: 2025-05-25
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
238 participants
INTERVENTIONAL
2023-04-17
2029-05-02
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Treatment Period
Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
2 Years
ALL
No
Sponsors
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Scholar Rock, Inc.
INDUSTRY
Responsible Party
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Locations
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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
Countries
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Other Identifiers
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SRK-015-004
Identifier Type: -
Identifier Source: org_study_id
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