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Taking people in ItalyPhase 2

Tests apitegromab in young children with spinal muscular atrophy

Tests apitegromab in children under 2 with spinal muscular atrophy, a muscle disease.

Key facts

Who runs it
Scholar Rock Inc.
Where
Milan and Rome, ItalySee the 3 sitesAlso in 4 other countries, 8 sites
Registry title
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Status
Recruiting
Phase
Phase 2
People taking part
30 people, planned
Started
17 Dec 2025
Expected to end
2 Oct 2031
Type of study
Interventional
Record updated
29 October 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for babies and young children under 2 years old who have spinal muscular atrophy (SMA) and are already on an approved SMN therapy.

Age
Under 2 years old when consent is given
Sex
All sexes
Disease stage
Confirmed 5q autosomal recessive SMA with SMN2 gene copies. Motor milestones are delayed for age because of SMA, or the CHOP-INTEND score is below 55.
Earlier treatment
Must have been treated with an approved SMN1-targeted therapy (onasemnogene abeparvovec-xioi), or be continuing an approved SMN2-targeted therapy (nusinersen or risdiplam).
Biomarker or test result
Confirmed SMN2 gene copy or copies.

Main reasons for not taking part

  • Nutrition not expected to stay stable, or a gastric feeding tube is medically needed for most feeds
  • Major bone or joint problems, such as severe scoliosis or contractures, or spine or hip surgery within 6 months before screening or planned during the study
  • Other physical limits, such as a cast for contractures, that would prevent motor function testing
  • A sibling already taking part in the study
  • Breastfeeding while the mother takes barbiturates or opioids

The registry criteria, word for word

Show all 12 criteriaShow less

Who can take part

  • 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)
  • 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)
  • Body weight for age is no less than third percentile based on the WHO Child Growth Standards at the Screening Visit
  • Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score <55

Who cannot take part

  • 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, as assessed by the Investigator and/or an Oral and Swallowing Abilities Tool score of 0
  • 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
  • Has a sibling participating in this study (ie, only 1 subject per immediate family is allowed to participate in the study)
  • Subject is breastfeeding while the mother is taking barbiturates and/or opioids

Where in Italy

Also in 4 other countries, 8 sites

  • Centro Clinico NemoMilan, ItalyTaking people
  • IRCCS Foundation Istituto Neurologico Carlo BestaMilan, ItalyTaking people
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCSRome, ItalyTaking people

About the trial

What the trial wants to find out

Evaluate the PK and PD of aepitegromab in subjects <2 years old with SMA.

Read full original descriptionShow less

Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment

Open-Label Extension Period Assess the safety and tolerability of apitegromab administered to subjects receiving an SMN therapy

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record

Print a page for my doctor

Data and sources

Registry record
Listed on the EU's official list of trials (CTIS).
Official title
A 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) (OPAL)
Plain-language title
Tests apitegromab in young children with spinal muscular atrophyGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 9 November 2026.
Record updated
29 October 2026