A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy (NCT07047144)
A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Phoenix Children's Hospital — Phoenix, Arizona
Age range: Not specified to 2 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (25 Sites)
- Phoenix Children's Hospital — Phoenix, Arizona
- Children's Hospital of Orange County (CHOC) — Orange, California
- Stanford Neuroscience Health Center (SNHC) — Palo Alto, California
- Children's Hospital Colorado — Aurora, Colorado
- Children's Healthcare of Atlanta — Atlanta, Georgia
- University of Iowa — Iowa City, Iowa
- Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan
- Atrium Health Wake Forest Baptist — Winston-Salem, North Carolina
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Le Bonheur Children's Hospital — Memphis, Tennessee
- University of Texas Southwestern Medical Center — Dallas, Texas
- Neurology Rare Disease Center — Flower Mound, Texas
- Cook Children's Medical Center — Fort Worth, Texas
- Texas Children's Hospital (TCH)-Clinical Care Center (CCC) — Houston, Texas
- UZ Gent — Ghent, East-Flanders
- UZ Leuven - Campus Gasthuisberg — Leuven,
- CHR Citadelle — Liège,
- Hopital Trousseau - I-Motion — Paris,
- Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta — Milan,
- Centro Clinico NeMO Milano - Fondazione Serena Onlus — Milan,
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome,
- Universitair Medisch Centrum Utrecht — Utrecht,
- Hospital Sant Joan de Déu Barcelona — Barcelona,
- Hospital Universitari i Politecnico La Fecnic La Fe — Valencia,
- Leeds General Infirmary Children's Research Department — Leeds, West Yorkshire
📞 Contact the Study Coordinator
Phone: (857) 259-3860
Email: MedicalInformation@scholarrock.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Spinal Muscular Atrophy
- Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — Phoenix, Arizona
- Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb — Pittsburgh, Pennsylvania
- A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) — Phoenix, Arizona
- Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — Phoenix, Arizona
- Spinraza in Adult Spinal Muscular Atrophy — Phoenix, Arizona
- Early Check: Expanded Screening in Newborns — Research Triangle Park, North Carolina
Explore by condition: All Spinal Muscular Atrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.