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NCT07047144 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, sponsored by Scholar Rock.

Recruiting
Registry status
Phase 2
Development phase
52
Enrollment target
14
Study locations

NCT07047144 is a Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3 that is actively recruiting participants, run by Scholar Rock. The registered enrollment target is 52 participants, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (51% lower). The trial reports 14 study locations across 7 states.

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The verdict

NCT07047144, a Phase 2 study of Spinal Muscular Atrophy and Spinal Muscular Atrophy Type 3, is actively recruiting participants, sponsored by Scholar Rock.

RECRUITING
Registry status
Phase 2
Development phase
52 participants
Enrollment target
14
Study locations

Study Summary

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.

Conditions Studied

Spinal Muscular Atrophy Spinal Muscular Atrophy Type 3 Neuromuscular Manifestations SMA Spinal Muscular Atrophy Type 2 Anti-myostatin

Interventions

  • DRUG Risdiplam
  • DRUG Apitegromab
  • DRUG Nusinersen

Study Locations (14)

Other

  • UZ Leuven - Campus Gasthuisberg - Leuven
  • CHR Citadelle - Liège
  • Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta - Milan
  • Centro Clinico NeMO Milano - Fondazione Serena Onlus - Milan
  • Hospital Sant Joan de Déu Barcelona - Barcelona
  • Hospital Universitari i Politecnico La Fecnic La Fe - Valencia

Texas

  • University of Texas Southwestern Medical Center - Dallas
  • Neurology Rare Disease Center - Flower Mound
  • Cook Children's Medical Center - Fort Worth

Arizona

  • Phoenix Children's Hospital - Phoenix

California

  • Stanford Neuroscience Health Center (SNHC) - Palo Alto

Colorado

  • Children's Hospital Colorado - Aurora

Iowa

  • University of Iowa - Iowa City

North Carolina

  • Atrium Health Wake Forest Baptist - Winston-Salem

Trial Details

FieldValue
Enrollment Target 52 participants
Start Date 2025-09-15
Est. Completion 2029-03
Phase Phase 2

Sponsor

Scholar Rock

7 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT07047144

The ClinicalTrials.gov registry entry for NCT07047144 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 52 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 107-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (51% lower). The listed sponsor is Scholar Rock, which has 7 total studies on file at ClinicalTrials.gov.

The record links to 6 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 3 interventions - of which Risdiplam is the first listed.

NCT07047144 reports 14 study locations spanning 7 distinct geographic areas - top geographies include Other, Texas, Arizona.

Frequently Asked Questions

What is clinical trial NCT07047144 about?

NCT07047144 is a clinical study titled "A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy". 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...

What is the current status of trial NCT07047144?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 52 participants. The study started on 2025-09-15. Estimated completion is 2029-03.

What conditions does trial NCT07047144 study?

This clinical trial studies the following conditions: Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, SMA, Spinal Muscular Atrophy Type 2.

What interventions are being tested in trial NCT07047144?

The interventions under investigation include: Risdiplam (DRUG), Apitegromab (DRUG), Nusinersen (DRUG).

Who is sponsoring clinical trial NCT07047144?

This trial is sponsored by Scholar Rock, which has 7 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT07047144 being conducted?

This trial has 14 study locations across Arizona, California, Colorado, Iowa, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.