Recruiting

OPALApitegromab Treatment Study for Spinal Muscular Atrophy (SMA) in Children Under 2

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Study Aim

This study is an interventional phase 2 trial to treat spinal muscular atrophy in children under 2 with apitegromab. The main goals are to evaluate the levels of apitegromab and myostatin in the body, and to assess improvements in motor function after 48 weeks of treatment.

What is being tested

Apitegromab

+ Nusinersen

+ Risdiplam

Drug
Who is being recruted

Central Nervous System Diseases+12

+ Muscular Atrophy, Spinal

+ Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Until 2 Years
+10 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: September 2025
See protocol details

Summary

Principal SponsorScholar Rock, Inc.
Study ContactScholar Rock
Last updated: February 11, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: September 15, 2025

Actual date on which the first participant was enrolled.

This study is focused on evaluating a new treatment called apitegromab for young children under 2 years old who have Spinal Muscular Atrophy (SMA), a genetic disorder that affects muscle strength and movement. The study is particularly interested in children who are not reaching movement milestones typical for their age, a common issue in SMA. By exploring how this treatment works in the body, its effectiveness, and any potential side effects, the study aims to find a safe and beneficial option for managing SMA in these young patients, potentially improving their quality of life. Participants in the study will receive multiple doses of the treatment, but neither the participants nor the researchers will know who is receiving apitegromab or a placebo, which helps ensure unbiased results. The treatment's effects will be monitored by measuring various indicators of muscle function and overall health. This includes observing any changes in motor skills and checking for any adverse reactions to the treatment. The study's structure helps to ensure that the findings are reliable and can provide insights into the safety and effectiveness of apitegromab for treating this condition in very young children.

Official TitleA 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)
NCT07047144
Principal SponsorScholar Rock, Inc.
Study ContactScholar Rock
Last updated: February 11, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

52 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Until 2 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Central Nervous System DiseasesMuscular Atrophy, SpinalCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsNeuromuscular DiseasesSigns and SymptomsSpinal Cord DiseasesPathological Conditions, Signs and SymptomsSpinal Muscular Atrophies of ChildhoodMotor Neuron DiseaseNeurodegenerative DiseasesHeredodegenerative Disorders, Nervous SystemNeuromuscular ManifestationsGenetic Diseases, Inborn

Criteria

7 inclusion criteria required to participate
Is <2 years old at the time of the informed consent
Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
Has confirmed diagnosis of 5q autosomal recessive SMA
Has confirmed presence of SMN2 gene copy(ies)
Show More Criteria
3 exclusion criteria prevent from participating
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
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
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 Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

2 intervention groups are designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
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).

Group II

Experimental
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).

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 14 locations

Recruiting

Phoenix Children's Hospital

Phoenix, United StatesOpen Phoenix Children's Hospital in Google Maps
Recruiting

Stanford Neuroscience Health Center (SNHC)

Palo Alto, United States
Recruiting

Children's Hospital Colorado

Aurora, United States
Recruiting

University of Iowa

Iowa City, United States
Recruiting
14 Study Centers