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

  • End date
    Sep 4, 2024
  • participants needed
  • sponsor
Updated on 7 October 2022
Primary Contact
Children's Specialty Healthcare (8.9 mi away) Contact
+19 other location
gene deletion


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.

The secondary objectives of this study are to evaluate the safety and tolerability; and clinical outcomes following treatment with nusinersen in participants with SMA who previously received onasemnogene abeparvovec.

Condition Muscular Atrophy, Spinal
Treatment Nusinersen
Clinical Study IdentifierNCT04488133
Last Modified on7 October 2022


Yes No Not Sure

Inclusion Criteria

Genetic documentation of 5q SMA homozygous gene survival motor neuron 1 (SMN1) deletion or mutation, or compound heterozygous mutation
Must have previously received onasemnogene abeparvovec
Participants with suboptimal clinical status per the Investigator

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
Note: Other protocol defined Inclusion/Exclusion criteria may apply
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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