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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
52
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.
Nusinersen is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered intrathecally per the prescribing information.
Risdiplam is a current standard-of-care SMN therapy that targets the SMN2 gene. It will be administered orally per the prescribing information.
Phoenix Children's Hospital
Phoenix, Arizona, United States
RECRUITINGStanford Neuroscience Health Center (SNHC)
Palo Alto, California, United States
RECRUITINGChildren's Hospital Colorado
Aurora, Colorado, United States
RECRUITINGUniversity of Iowa
Iowa City, Iowa, United States
RECRUITINGAtrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
RECRUITINGUniversity of Texas Southwestern Medical Center
Dallas, Texas, United States
RECRUITINGNeurology Rare Disease Center
Flower Mound, Texas, United States
RECRUITINGCook Children's Medical Center
Fort Worth, Texas, United States
RECRUITINGUZ Leuven - Campus Gasthuisberg
Leuven, Belgium
RECRUITINGCHR Citadelle
Liège, Belgium
RECRUITING...and 4 more locations
Evaluate the PK of apitegromab in subjects <2 years old with SMA
Apitegromab concentrations in serum
Time frame: 52 Weeks
Evaluate the PD of apitegromab in subjects <2 years old with SMA
Total latent myostatin concentrations in serum
Time frame: 52 Weeks
Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment
Change from baseline in the raw score of the Bayley Scale of Infant and Toddler Development, Fourth Edition - Gross Motor Subscale (BSID-4 GMS) at 48 weeks. BSID-4 GMS is a standardized assessment commonly used to evaluate development across 5 domains in infants and young children. It consists of 58 items, scored from 0 to 2 for each item, with higher scores indicating better gross motor development.
Time frame: 48 Weeks
Assess the safety and tolerability of apitegromab administered to subjects receiving an SMN therapy
Incidence of TEAEs and SAEs by severity
Time frame: 52 Weeks
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