A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
NCT ID: NCT04729907
Last Updated: 2025-02-03
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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ACTIVE_NOT_RECRUITING
PHASE3
115 participants
INTERVENTIONAL
2021-04-19
2026-07-31
Brief Summary
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The main goal of the study is to learn about the long-term safety of nusinersen. The main questions researchers want to answer are:
* How many participants have adverse events and serious adverse events during the study?
* How do the results of electrocardiograms (ECGs), vital signs, and laboratory tests including blood and urine tests change after treatment?
* How many participants have a low platelet count after treatment?
* How many participants had a change in the time it took for their heart to recharge between beats after treatment?
* How does each participant's height and other measures of growth change after treatment?
* How much do the results of neurological exams that check movement, reflexes, and brain function change after treatment?
Researchers will also learn about the effect of nusinersen on mobility using various tests. They will study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing.
The 232SM302 study will be done as follows:
* Participants will be screened to check if they can join the study.
* Participants will receive their 1st dose of nusinersen in this study about 4 months after their final dose in the parent study.
* Each participant will receive nusinersen once every 4 months during the treatment period.
* 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 64 months (1921 days).
* There will be a follow-up safety period that lasts from 4 to 8 weeks.
* In total, participants will have up to 19 study visits. Participants will stay in the study for close to 6 years.
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Detailed Description
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The secondary objective of this study is to evaluate the long-term efficacy of nusinersen administered intrathecally at higher doses to participants with SMA who previously participated in study 232SM203 (NCT04089566).
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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BIIB058 28 mg (Prior Maintenance Dose 28 mg)
Participants who received maintenance dose of 28 milligrams (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.
Nusinersen
Administered as specified in the treatment arm
BIIB058 50/28 mg (Prior Maintenance Dose 12 mg)
Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.
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
Exclusion Criteria
* Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566)
2 Years
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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Stanford University Medical Center
Sacramento, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
The Johns Hopkins Hospital
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Children's Medical Center
Plano, Texas, United States
Hospital de Clínicas de Porto Alegre
Porto Alegre, , Brazil
Hospital das Clinicas - FMUSP
São Paulo, , Brazil
London Health Sciences Centre (LHSC) - Children's Hospital
London, Ontario, Canada
Hospital Luis Calvo Mackenna
Santiago, , Chile
Clinica Las Condes
Santiago, , Chile
Clinica MEDS La Dehesa
Santiago, , Chile
Peking University First Hospital
Beijing, Beijing Municipality, China
Guangzhou Woman and Children's Medical Center
Guangzhou, Guangdong, China
The Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Hospital Universitario San Ignacio
Bogotá, , Colombia
Tallinn Children's Hospital
Tallinn, , Estonia
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Universitaetsklinikum Giessen und Marburg GmbH
Giessen, Hesse, Germany
Fondazione Serena Onlus - Centro Clinico Nemo
Milan, , Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, , Italy
Kurume University Hospital
Kurume-shi, Fukuoka, Japan
Hyogo Medical University Hospital
Nishinomiya-shi, Hyōgo, Japan
Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo-To, Japan
Saint George University Hospital Medical Center
Beirut, , Lebanon
Instituto Nacional de Pediatria
Mexico City, Mexico City, Mexico
Hospital Infantil de Mexico Federico Gomez
Mexico City, Mexico City, Mexico
Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde
Guadalajara, , Mexico
Russian Children Neuromuscular Center of Veltischev
Moskva, , Russia
Regional Pediatric Clinical Hospital #1
Yekaterinburg, , Russia
King Fahad Specialist Hospital
Dammam, , Saudi Arabia
National Guard Health Affairs: King Abdulaziz Medical City
Jeddah, , Saudi Arabia
King Faisal Specialist Hospital & Research Center
Riyadh, , Saudi Arabia
Hospital Sant Joan de Deu
Esplugues Del Llobregat, Barcelona, Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Countries
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Related Links
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SMA Europe
CureSMA
Muscular Dystrophy Association
Other Identifiers
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2023-505637-27
Identifier Type: OTHER
Identifier Source: secondary_id
232SM302
Identifier Type: -
Identifier Source: org_study_id
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