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)
Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA — Up to 6 years Incidence of TEAEs and SAEs by severity
Trial sites (49)
Facility
City
Region
Status
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.
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.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.