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RECRUITINGPhase 2INTERVENTIONAL

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

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)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Is \<2 years old at the time of the willing to sign a consent form 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 Who Should NOT Join This Trial: 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

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.

Locations (20)

Phoenix Children's Hospital
Phoenix, Arizona, United States
Children's Hospital of Orange County (CHOC)
Orange, California, United States
Stanford Neuroscience Health Center (SNHC)
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Iowa
Iowa City, Iowa, United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Neurology Rare Disease Center
Flower Mound, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Texas Children's Hospital (TCH)-Clinical Care Center (CCC)
Houston, Texas, United States
UZ Gent
Ghent, East-Flanders, Belgium
UZ Leuven - Campus Gasthuisberg
Leuven, Belgium
CHR Citadelle
Liège, Belgium
Hopital Trousseau - I-Motion
Paris, France
Fondazione I.R.C.C.S. - Istituto Neurologico Carlo Besta
Milan, Italy
Centro Clinico NeMO Milano - Fondazione Serena Onlus
Milan, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy