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

Launched by SCHOLAR ROCK, INC. · Jun 23, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called apitegromab to see how it works and how safe it is in babies under 2 years old who have a genetic condition called spinal muscular atrophy (SMA). SMA affects the muscles and movement, causing delays in reaching motor milestones like sitting or crawling. The study focuses on babies who have a certain type of SMA and who have already started or are continuing treatment with approved SMA therapies. Researchers want to learn if adding apitegromab can help improve muscle function and overall health in these young children.

To take part, babies must be under 2 years old, born after at least 35 weeks of pregnancy with a birth weight over 2 kg, have a confirmed diagnosis of SMA, and show delayed motor skills related to SMA. Participants should have a certain level of muscle function based on a simple test (CHOP-INTEND score less than 55). During the study, families can expect their child to receive several doses of apitegromab while doctors closely monitor their muscle strength, safety, and how the medicine is processed in the body. Some children won’t be eligible, such as those with major feeding issues or severe physical problems that would make it hard to measure muscle function. This trial is not yet recruiting but aims to help understand if apitegromab can be a helpful addition to current SMA treatments for very young children.

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ALL

Eligibility criteria

• 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.