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Recruiting NCT07047144

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

Phase II Interventional Spinal Muscular Atrophy SMA Spinal Muscular Atrophy Type 2 Spinal Muscular Atrophy Type 3

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Apitegromab, Nusinersen, Risdiplam.
Who it may be relevant to
Registry conditions: Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3. Basic parameters: up to 2 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Belgium, France, Italy, Netherlands +2
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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.

Interventions

  • Drug Apitegromab
    Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. SRK-015 will be administered every 4 weeks by intravenous (IV) infusion.
  • Drug Nusinersen
    Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.
  • Drug Risdiplam
    Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.

Primary outcome measures

  • Evaluate the PK of apitegromab in subjects <2 years old with SMA [Time frame: 52 Weeks]
  • Evaluate the PD of apitegromab in subjects <2 years old with SMA [Time frame: 52 Weeks]
  • Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment [Time frame: 48 Weeks]
Secondary outcome measures (1)
  • Assess the safety and tolerability of apitegromab administered to subjects receiving an SMN therapy [Time frame: 52 Weeks]

Eligibility criteria

Inclusion criteria

  • 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 1st 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

Exclusion criteria

  • 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
  • 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
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 14 centers
  • Phoenix Children's Hospital — Phoenix
  • Children's Hospital of Orange County (CHOC) — Orange
  • Stanford Neuroscience Health Center (SNHC) — Palo Alto
  • Children's Hospital Colorado — Aurora
  • Children's Healthcare of Atlanta — Atlanta
  • University of Iowa — Iowa City
  • Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids
  • Atrium Health Wake Forest Baptist — Winston-Salem
  • … and 6 more centers
Belgium · 3 centers
  • UZ Gent — Ghent
  • UZ Leuven - Campus Gasthuisberg — Leuven
  • CHR Citadelle — Liège
Italy · 3 centers
  • Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta — Milan
  • Centro Clinico NeMO Milano - Fondazione Serena Onlus — Milan
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome
Spain · 2 centers
  • Hospital Sant Joan de Déu Barcelona — Barcelona
  • Hospital Universitari i Politecnico La Fecnic La Fe — Valencia
France · 1 center
  • Hopital Trousseau - I-Motion — Paris
Netherlands · 1 center
  • Universitair Medisch Centrum Utrecht — Utrecht
United Kingdom · 1 center
  • Leeds General Infirmary Children's Research Department — Leeds

Identifiers

NCT: NCT07047144 · SRK-015-005

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗