Apitegromab for infants under 2 with spinal muscular atrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a double-blind, Phase 2, multiple-dose trial testing apitegromab in infants under 2 years with 5q autosomal recessive spinal muscular atrophy who have delayed motor milestones or a CHOP-INTEND score under 55. Participants must be receiving or have received approved SMN-targeted therapy (for example, onasemnogene abeparvovec, nusinersen, or risdiplam) and will be monitored for pharmacokinetics, pharmacodynamics, safety, tolerability, and motor outcomes. The study compares repeated doses of apitegromab against control while continuing background SMA therapy to see if additional benefit on motor function can be achieved. Outcomes will include PK/PD measures and changes in standardized motor function scores over the treatment period.

Why it matters

Who can join: the full rules

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.

Where it's running

• Phoenix Children's Hospital
  Phoenix, Arizona, United States Recruiting
• Children's Hospital of Orange County (CHOC)
  Orange, California, United States Recruiting
• Stanford Neuroscience Health Center (SNHC)
  Palo Alto, California, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• Children's Healthcare of Atlanta
  Atlanta, Georgia, United States Recruiting
• University of Iowa
  Iowa City, Iowa, United States Recruiting

Study contacts

• Study coordinator: Scholar Rock
• Email: MedicalInformation@scholarrock.com
• Phone: 857-259-3860

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Registry for patients with Spinal Muscular Atrophy in the UK

  This study is collecting information from people with Spinal Muscular Atrophy to better understand their experiences and improve future treatments.

  Recruiting Newcastle University · Newcastle upon Tyne · NCT04292574

• Pregnancy-time treatment of fetal SMA: a registry of mother-child outcomes

  This registry collects information on pregnancies where a fetus diagnosed with spinal muscular atrophy was treated with a disease‑modifying therapy in the womb to see how the pregnancy and baby fare after birth.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT07805278

• Intrathecal Zolgensma in spinal muscular atrophy patients in Japan

  This study tests the safety and real‑world effectiveness of intrathecal onasemnogene abeparvovec in SMA patients in Japan.

  Recruiting at 4 sites Novartis · Toyoake, Aichi-ken and 3 other locations · NCT07767890

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• Spinal muscular atrophy patients get a single intrathecal gene therapy dose to see how it works in real-world U.S. settings

  This study tests whether a one-time intrathecal dose of ITVISMA® (onasemnogene abeparvovec-brve) is safe and effective for SMA patients in real-world U.S. clinical practice.

  Recruiting at 6 sites Phase 4 Novartis · Little Rock, Arkansas and 5 other locations · NCT07448610

• Salanersen (BIIB115) for improving movement in people 15–60 with spinal muscular atrophy who are treatment‑naïve or previously on risdiplam

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  Recruiting at 3 sites Phase 3 Biogen · Palo Alto, California and 2 other locations · NCT07444476

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.