In:
Journal of Neurology, Neurosurgery & Psychiatry, BMJ, Vol. 93, No. 6 ( 2022-06), p. A87.1-A87
Abstract:
SUNFISH ( NCT02908685 ) is a multicentre, two-part, randomised (2:1, risdiplam:placebo), placebo-con- trolled, double-blind study in patients, aged 2–25 years, with Type 2 or Type 3 spinal muscular atrophy (SMA). SUNFISH investigates the safety and efficacy of risdiplam, an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier. SUNFISH is comprised of two parts: Part 1 (n=51) is a dose-selection study assessing the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam doses in patients with Type 2/3 SMA; confirmatory Part 2 (n=180) assesses the safety and efficacy of the risdiplam dose selected from Part 1 compared with placebo in patients with Type 2 and non-ambulant Type 3 SMA. The primary objective of Part 2 is to evaluate the efficacy of risdiplam compared with placebo in terms of motor function as assessed by the change from baseline in the 32-item Motor Function Measure total score at Month 12. In SUNFISH Part 1, no drug-related safety findings led to withdrawals from the study following 1 year of treatment with risdiplam (data-cut: 28th June 2019). Here we report data from Part 2 of the SUNFISH study including baseline demographics, safety and efficacy data in participants who have received treatment with risdiplam or placebo for 12 months. laurent.servais@paediatrics.ox.ac.uk
Type of Medium:
Online Resource
ISSN:
0022-3050
,
1468-330X
DOI:
10.1136/jnnp-2022-ABN.282
Language:
English
Publisher:
BMJ
Publication Date:
2022
detail.hit.zdb_id:
1480429-3
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