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

RecruitingPhase 2Ages any - 2 YearsAllScholar Rock, Inc.
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: 1. Is \<2 years old at the time of the informed consent 2. Had a gestational age of โ‰ฅ35 weeks and gestational body weight โ‰ฅ2.0 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.
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What it tests

  • Apitegromab
  • Nusinersen
  • Risdiplam
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Where

23 sites
Phoenix Children's Hospital
Phoenix, United States
Children's Hospital of Orange County (CHOC)
Orange, United States
Stanford Neuroscience Health Center (SNHC)
Palo Alto, United States
Children's Hospital Colorado
Aurora, United States
Children's Healthcare of Atlanta
Atlanta, United States
University of Iowa
Iowa City, United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, United States
The Children's Hospital of Philadelphia
Philadelphia, United States

โ€ฆand 15 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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