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NCT04488133 · ClinicalTrials.gov registry record · Phase 4
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)
A Phase 4 study, sponsored by Biogen.
- Completed
- Registry status
- Phase 4
- Development phase
- 46
- Enrollment target
NCT04488133 is a Phase 4 study that has completed, run by Biogen. The registered enrollment target is 46 participants.
The verdict
NCT04488133, a Phase 4 study, has completed, sponsored by Biogen.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 46 participants
- Enrollment target
Study Summary
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.
Interventions
- DRUG Nusinersen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 46 participants |
| Start Date | 2021-01-04 |
| Est. Completion | 2025-10-09 |
| Phase | Phase 4 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04488133
The ClinicalTrials.gov registry entry for NCT04488133 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 46 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Biogen, which has 334 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Nusinersen is the first listed.
NCT04488133 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04488133 about?
NCT04488133 is a clinical study titled "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)". 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 ...
What is the current status of trial NCT04488133?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 46 participants. The study started on 2021-01-04. Estimated completion is 2025-10-09.
What interventions are being tested in trial NCT04488133?
The interventions under investigation include: Nusinersen (DRUG).
Who is sponsoring clinical trial NCT04488133?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
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