Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
Study ID: STU-2021-1167
Summary
The purpose of this trial is to assess the safety and efficacy of apitegromab compared with placebo for improving motor function in patients with later-onset SMa who are receiving background therapy with nusinersen or risdiplam. Trial details include: Trial Duration: 76 weeks Treatment Duration: 52 weeks Visit Frequency: every 4 weeks
Main efficacy Population x efficacy and additional objectives/endpoints: 1. assess the efficacy of apitegromab compared with placebo using the HFMSe in patients 2 through 12 years old 2. assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in patients 2 through 12 years old 3. assess the efficacy of apitegromab compared with placebo by measuring changes in upper limb function using the Revised upper Limb Module (RuLM) in patients 2 through 12 years old 4. assess the efficacy of apitegromab compared with placebo by measuring changes in number of World Health organization (WHo) motor development milestones in patients 2 through 12 years old 5. Further assess the efficacy of apitegromab compared with placebo by evaluating changes in additional motor function outcome measures and changes in HFMSe at other prespecified time points in patients 2 through 12 years old 6. assess the efficacy of apitegromab compared [See protocol for complete text]
