Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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.
More Scholar Rock, Inc. trials in France
Other trials for the same condition
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 France