Recruiting
Phase 2

Apitegromab

Sponsor:

Scholar Rock, Inc.

Code:

NCT07047144

Conditions

Spinal Muscular Atrophy

SMA

Spinal Muscular Atrophy Type 2

Spinal Muscular Atrophy Type 3

Neuromuscular Manifestations

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Interventions

Apitegromab

Nusinersen

Risdiplam

Study Details

Brief 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

Spinal Muscular Atrophy

SMA

Spinal Muscular Atrophy Type 2

Spinal Muscular Atrophy Type 3

Neuromuscular Manifestations

Study ID

NCT07047144

Start date

Sep 15, 2025

Status verified date

Apr, 2026

Completion date

Mar, 2029

Anticipated

Primary completion date

Nov, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Is <2 years old at the time of the informed consent
2. Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
3. Has confirmed diagnosis of 5q autosomal recessive SMA
4. Has confirmed presence of SMN2 gene copy(ies)
5. Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)
6. Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit
7. Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score <55

Exclusion Criteria:

1. Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route
2. Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study
3. Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.

Study Design

Enrollment

52 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Apitegromab low dose + SMN Therapy

Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period.

Patients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam).

experimental: Apitegromab high dose + SMN Therapy

Patients who are less than 2 years of age with SMA will receive apitegromab every 4 weeks by intravenous (IV) infusion during the 48-week Treatment Period.

Patients must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam).

Interventions

Apitegromab

Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 will be administered every 4 weeks by intravenous (IV) infusion.

Nusinersen

Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.

Risdiplam

Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.

Primary outcome measure

  • Evaluate the PK of apitegromab in subjects <2 years old with SMA [ Time Frame: 52 Weeks ]
  • Evaluate the PD of apitegromab in subjects <2 years old with SMA [ Time Frame: 52 Weeks ]
  • Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment [ Time Frame: 48 Weeks ]

Central Contacts and Locations

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Contacts

Children's Hospital of Orange County (CHOC)

Recruiting

Orange, California, United States, 92868

Contacts

Stanford Neuroscience Health Center (SNHC)

Recruiting

Palo Alto, California, United States, 94304

Contacts

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Children's Healthcare of Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Helen DeVos Children's Hospital at Spectrum Health

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

Atrium Health Wake Forest Baptist

Recruiting

Winston-Salem, North Carolina, United States, 27157

Contacts

The Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75207

Contacts

Neurology Rare Disease Center

Recruiting

Flower Mound, Texas, United States, 75028

Contacts

Cook Children's Medical Center

Recruiting

Fort Worth, Texas, United States, 76104

Contacts

Texas Children's Hospital (TCH)-Clinical Care Center (CCC)

Recruiting

Houston, Texas, United States, 77030

Contacts

More Information

Sponsor

Scholar Rock, Inc.

Last update posted

May 1, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Scholar Rock, Inc. on 2026-05-01.