Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Summary

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C). The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).

Timeline

Start

2020-03-26

Primary completion

2024-02-21

Completion

2024-05-30

Publications

• Finkel RS, Crawford TO, Mercuri E, Sumner CJ, Garcia Romero MDM, Day JW, Montes J, Sun P, Tichler B, Paradis AD, Boesch E, Inra J, Littauer R, Sohn J, Monine M, Gambino G, Foster R, Farewell R, Fradette S. High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial. Nat Med. 2026 Mar;32(3):1095-1104. doi: 10.1038/s41591-025-04193-6. Epub 2026 Feb 3.
• Wang X, Zheng Y, Lu J, Li X, Chen S, Xue Y, Hu S, Peng G, Zhang F, Zhao X, Liu Y, Fan Y, Zhou H, Liang C, Liu L, He L, Zhang J, Shi W, Tian J, Han M, Xu K. Musculoskeletal deformities in children with spinal muscular atrophy: a multicenter cross-sectional study with longitudinal follow-up. Ann Phys Rehabil Med. 2026 Apr;69(3):102080. doi: 10.1016/j.rehab.2025.102080. Epub 2026 Jan 8.
• Finkel RS, Ryan MM, Pascual Pascual SI, Day JW, Mercuri E, De Vivo DC, Foster R, Montes J, Gurgel-Giannetti J, MacCannell D, Berger Z. Scientific rationale for a higher dose of nusinersen. Ann Clin Transl Neurol. 2022 Jun;9(6):819-829. doi: 10.1002/acn3.51562. Epub 2022 May 13.

Drugs

Indications