A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
NCT ID: NCT04488133
Last Updated: 2025-10-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE4
46 participants
INTERVENTIONAL
2021-01-04
2025-10-09
Brief Summary
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The main goal of the study is to learn about the effect nusinersen has on muscle and movement ability (motor function). The main question researchers want to answer is:
\- What score do participants have on the HINE Section 2 Motor Milestones test after treatment?
The Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones is an assessment that tests movements in different positions. This includes grasping, kicking, head control, rolling, sitting, crawling, standing, and walking.
Researchers will use a group of tests to study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing.
Researchers will also learn more about the safety of nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests.
The study will be done as follows:
* Participants will be screened to check if they can join the study.
* Each participant will receive 4 initial doses of 12 mg of nusinersen on Days 1, 15, 29, and 64 of the Treatment Period. Then, they will receive 12 mg doses once every 4 months.
* The total number of doses of nusinersen will be 9.
* Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back.
* The treatment period will last for up to 95 weeks (close to 2 years).
* There will be a follow-up safety period that lasts about 4 months.
* In total, participants will have up to 14 study visits. Participants will stay in the study for up to 115 weeks.
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Nusinersen 12 mg
Participants will receive Nusinersen 12 milligrams (mg) via intrathecal (IT) injection as loading doses on Days 1, 15, 29, and 64 followed by maintenance doses, every 4 months, on Days 183, 302, 421, 540 and 659.
Nusinersen
Administered as specified in the treatment arm.
Interventions
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Nusinersen
Administered as specified in the treatment arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Genetic documentation of 5q SMA homozygous gene survival motor neuron 1 (SMN1) deletion or mutation, or compound heterozygous mutation
* 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 Criteria for Subgroups A and B:
* \<300 days of age at the time of first Nusinersen dose
* SMN2 copy number of 2
Additional Criteria for Subgroup A:
* SMA symptom onset ≤4 months (120 days) of age
* Must have received intravenous (IV) onasemnogene abeparvovec at \>6 weeks to ≤6 months (43 days to 180 days) of age
* Must have received IV onasemnogene abeparvovec after SMA symptom onset
Additional Criteria for Subgroup B:
* Must have received IV onasemnogene abeparvovec at ≤6 weeks (42 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 Criteria 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.
2 Months
36 Months
ALL
No
Sponsors
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Biogen
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States
Stanford Neuromuscular Research
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Oregon Health and Science University (OHSU)
Portland, Oregon, United States
Children's Hospital Philadelphia - Neurology
Philadelphia, Pennsylvania, United States
University of Utah
Salt Lake City, Utah, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, , Germany
Schneider Children's Medical Center
Petah Tikva, , Israel
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Milan, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, , Italy
Hospital Sant Joan de Déu
Esplugues Del Llobregat, Barcelona, Spain
Hospital Universitario La paz
Madrid, , Spain
Countries
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References
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Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD. Open-label phase 4 trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. J Clin Invest. 2025 Sep 16:e193956. doi: 10.1172/JCI193956. Online ahead of print.
Related Links
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SMA Europe
CureSMA
Muscular Dystrophy Association
Child Neurology Foundation
Biogen Trial Link
Other Identifiers
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2020-003492-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-505640-18
Identifier Type: OTHER
Identifier Source: secondary_id
232SM404
Identifier Type: -
Identifier Source: org_study_id
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