A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Summary
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.
Timeline
- Start
- 2016-10-19
- Primary completion
- 2019-09-06
- Completion
- 2023-10-02
Outcome
Met primary endpoint
registry analysis (superiority test); Part 2: Risdiplam vs Part 2: Placebo; p = 0.0156; Least Square Mean Difference 1.55 (95% CI 0.30 to 2.81) NCT02908685 ↗
registry LEAST_SQUARES_MEAN Part 2: Risdiplam: 1.36 (0.61 to 2.11) Scores on a Scale LEAST_SQUARES_MEAN Part 2: Placebo: -0.19 (-1.22 to 0.84) Scores on a Scale NCT02908685 ↗
release “Genentech’s Risdiplam Meets Primary Endpoint In Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy” gene.com ↗
release “Pivotal part 2 of the Sunfish study, risdiplam People aged 2-25 with type 2 or 3 SMA Primary endpoint met” roche.com ↗
release “SUNFISH trial in non-ambulatory children and adults demonstrated that change from baseline in total Motor Function Measure 32 (MFM-32) score was significantly greater at 12 months” ptcbio.com ↗
Publications
- Cleary Y, Kletzl H, Grimsey P, Heinig K, Ogungbenro K, Silber Baumann HE, Frey N, Aarons L, Galetin A, Gertz M. Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug-Drug Interactions in Children. Clin Pharmacokinet. 2023 Jun;62(6):891-904. doi: 10.1007/s40262-023-01241-7. Epub 2023 May 6.
- Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Risdiplam | Small molecule | 0.25 mg/kg | Oral |
| Subject | Risdiplam | Small molecule | 5 mg | Oral |