Condition: Spinal Muscular Atrophy · SMA · Spinal Muscular Atrophy Type 2 · Sponsor: Scholar Rock, Inc.
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.
This description comes directly from the study's public registry record.
Scholar Rock · 857-259-3860 · MedicalInformation@scholarrock.com
Always discuss trial participation with your own doctor first.
| Phoenix Children's Hospital | Phoenix, Arizona, United States | Recruiting |
| Children's Hospital of Orange County (CHOC) | Orange, California, United States | Recruiting |
| Stanford Neuroscience Health Center (SNHC) | Palo Alto, California, United States | Recruiting |
| Children's Hospital Colorado | Aurora, Colorado, United States | Recruiting |
| Children's Healthcare of Atlanta | Atlanta, Georgia, United States | Recruiting |
| University of Iowa | Iowa City, Iowa, United States | Recruiting |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids, Michigan, United States | Recruiting |
| Atrium Health Wake Forest Baptist | Winston-Salem, North Carolina, United States | Recruiting |
| The Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | Recruiting |
| Le Bonheur Children's Hospital | Memphis, Tennessee, United States | Not Yet Recruiting |
| University of Texas Southwestern Medical Center | Dallas, Texas, United States | Recruiting |
| Neurology Rare Disease Center | Flower Mound, Texas, United States | Recruiting |
+ 13 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT07047144