A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT07047144. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT07047144
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 52 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Up to 2 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 15, 2025 (Actual)
- Primary completion
- Nov 2028 (Estimated)
- Completion
- Mar 2029 (Estimated)
- Last update posted
- Oct 2, 2026
2025 – 2029
United States locations
- U.S. sites
- 12
- U.S. states
- 8
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | Recruiting |
| Children's Hospital of Orange County (CHOC) | Orange | California | 92868 | Recruiting |
| Stanford Neuroscience Health Center (SNHC) | Palo Alto | California | 94304 | Recruiting |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | Recruiting |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | Recruiting |
| University of Iowa | Iowa City | Iowa | 52242 | Recruiting |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan | 49503 | Recruiting |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75207 | Recruiting |
| Neurology Rare Disease Center | Flower Mound | Texas | 75028 | Recruiting |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | Recruiting |
| Texas Children's Hospital (TCH)-Clinical Care Center (CCC) | Houston | Texas | 77030 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 11 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07047144, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 2, 2026 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT07047144 live on ClinicalTrials.gov.