A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)

Phase 1

• Conditions: Spinal Muscular Atrophy; MedDRA version: 20.0Level: SOCClassification code: 10010331Term: Congenital familial and genetic disorders Class: 21; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: CTIS2023-505640-18-00
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-23
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 46

Inclusion Criteria

Genetic documentation of 5q SMA homozygous gene survival motor neuron 1 (SMN1) deletion or mutation, or compound heterozygous mutation, Additional Criterion for Subgroup A: Must have received IV onasemnogene abeparvovec after SMA symptom onset, Additional Criterion for Subgroup B: Must have received IV onasemnogene abeparvovec at =6 weeks (42 days) of age, SMN2 copy number of =1, =36 months of age at the time of first Nusinersen dose, Must have previously received onasemnogene abeparvovec per the approved label or local/regional regulations =2 months prior to first Nusinersen dose, Must have suboptimal clinical status per the Investigator, Additional Criterion for Subgroups A and B: <300 days of age at the time of first Nusinersen dose, Additional Criterion for Subgroups A and B: SMN2 copy number of 2, Additional Criterion for Subgroup A: SMA symptom onset =4 months (120 days) of age, Additional Criterion for Subgroup A: Must have received intravenous (IV) onasemnogene abeparvovec at >6 weeks to =6 months (43 days to 180 days) of age

Exclusion Criteria

Prior exposure to Nusinersen, Ongoing severe or serious AEs related to onasemnogene abeparvovec, Treatment with an investigational drug, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to study; any prior or current treatment with any survival motor neuron 2 (SMN2)-directed splicing modifier; prior antisense oligonucleotide treatment or cell transplantation; gene therapy for the treatment of SMA other than onasemnogene abeparvovec. Note: treatment with onasemnogene abeparvovec as part of an investigational study is allowed, Additional Criterion for Subgroups A and B: Weight-for-age is below the third percentile, based on WHO Child Growth Standards at the time of receiving onasemnogene abeparvovec. Adjustments for the gestational weight of premature babies enrolled in Subgroups A and B are allowed provided IV onasemnogene abeparvovec was dosed per the approved label or per local/regional regulations. Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec.;Secondary Objective: The secondary objectives of this study are to evaluate the safety and tolerability; clinical outcomes and pharmacodynamics (PD) of nusinersen treatment in participants with SMA who previously received onasemnogene abeparvovec.;Primary end point(s): Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score [ Time Frame: Up to Day 778 ]