A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
Stopped early · Phase 2 · Has a placebo group
Conditions studied: Spinal Muscular Atrophy
In brief
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS 396443-CS4 (NCT02292537). The secondary objective of Part 1 of this study is to examine the pharmacokinetics (PK) of Nusinersen in participants with SMA. The primary objective of Part 2 of this study is to assess the long-term safety and tolerability of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments. The secondary objective of Part 2 of this study is to examine the PK of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments.
Key facts
- Study ID
- NCT02462759
- Run by
- Biogen
- People needed
- 21
- Starts
- 2015-08-19
- Expected to finish
- 2018-09-24
- Last updated by the study team
- 2021-02-17
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
Where it is running
- David Geffen School of Medicine at UCLA — Los Angeles, California, United States
- Connecticut Childrens Medical — Hartford, Connecticut, United States
- The Johns Hopkins Hospital — Baltimore, Maryland, United States
- Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
- The University of Texas Southwestern Medical Center — Dallas, Texas, United States
- Seattle Children's Research Institute — Seattle, Washington, United States
- LMU-Campus Innenstadt — München, Germany
Full record on ClinicalTrials.gov
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