The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
238
Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength.
Phoenix Childrens Hospital
Phoenix, Arizona, United States
UCSD Altman Clinical and Translational Research
La Jolla, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford Neuroscience Health Center
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA
Incidence of TEAEs and SAEs by severity
Time frame: Up to 6 years
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Hammersmith Functional Motor Scale Expanded (HFMSE) total score at prespecified time points (excludes ambulatory patients)
Time frame: Up to 6 years
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Revised Upper Limb Module (RULM) total score at prespecified time points (excludes ambulatory patients)
Time frame: Up to 6 years
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Number of World Health Organization (WHO) motor development milestones attained at prespecified time points (excludes ambulatory patients)
Time frame: Up to 6 years
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Revised Hammersmith Scale (RHS) total score
Time frame: Up to 6 years
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Results for 6-Minute Walk Test
Time frame: Up to 6 years
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
30-Second Sit-to-Stand
Time frame: Up to 6 years
Further evaluate the immunogenicity of apitegromab
Presence or absence of antidrug antibody (ADA) against apitegromab in serum from blood samples
Time frame: Up to 6 years
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Nemours Biomedical Research
Orlando, Florida, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Johns HopkinsHospital
Baltimore, Maryland, United States
...and 39 more locations