A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

ClinicalTrials.gov ID: NCT07047144

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.

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ClinicalTrials.gov public records Last synced Sep 6, 2026, 1:10 PM EDT

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
Lead sponsor: Scholar Rock, Inc.; Industry
Enrollment: 52 participants

Conditions and interventions

Conditions

Interventions

Drug

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 14, 2025
Primary completion: Oct 31, 2028
Completion: Feb 28, 2029
Last update posted: Apr 30, 2026

2025 – 2029

United States locations

U.S. sites: 14
U.S. states: 10
U.S. cities: 14

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
Atrium Health Wake Forest Baptist	Winston-Salem	North Carolina	27157	Recruiting
The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	19104	Recruiting
Le Bonheur Children's Hospital	Memphis	Tennessee	38103	Not yet 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 Apr 30, 2026 · Synced Sep 6, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT07047144 live on ClinicalTrials.gov.

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