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)
Completed · Phase 4
Conditions studied: Muscular Atrophy, Spinal
In brief
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on children under the age of 3 who were previously treated with the gene therapy onasemnogene abeparvovec but are still facing health challenges related to their disease. 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.
Key facts
- Study ID
- NCT04488133
- Run by
- Biogen
- People needed
- 46
- Starts
- 2021-01-04
- Expected to finish
- 2025-10-09
- Last updated by the study team
- 2025-10-14
Who can join
Age: 0 and older, up to 3. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.