Clinical Trials

Risdiplam has been evaluated across numerous clinical trials primarily targeting spinal muscular atrophy (SMA)—including SMA Types 2 and 3—and assessing pharmacokinetics in healthy volunteers. Spanning Phase 1 through Phase 4 developmental stages, including combined Phase 2/3 protocols, these investigations encompass completed, actively recruiting, and post-marketing studies sponsored by industry leaders and academic medical centers. Collectively, these trials establish the safety, pharmacokinetics, and therapeutic efficacy of oral risdiplam in managing neuromuscular manifestations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07047144 RECRUITING
Spinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type 3; Neuromuscular Manifestations; Anti-myostatin
Scholar Rock, Inc.
 2025-09-15 PHASE2
NCT07444476 RECRUITING
Spinal Muscular Atrophy
Biogen
 2026-04-03 PHASE3
NCT05861986 RECRUITING
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2024-05-30 PHASE4
NCT05861999 RECRUITING
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2024-08-14 PHASE4
NCT05067790 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy
Biogen
 2022-01-21 PHASE3
NCT05232929 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy
Genentech, Inc.
 2022-03-29 PHASE4
NCT05115110 ACTIVE_NOT_RECRUITING
Spinal Muscular Atrophy (SMA)
Hoffmann-La Roche
 2022-06-02 PHASE2; PHASE3
NCT05808764 COMPLETED
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2024-04-26 PHASE2
NCT05522361 UNKNOWN
Spinal Muscular Atrophy
Clinic for Special Children
 2022-11-06 PHASE4
NCT03032172 COMPLETED
Spinal Muscular Atrophy
Hoffmann-La Roche
 2017-03-03 PHASE2
NCT04888702 UNKNOWN
Spinal Muscular Atrophy
Centre Hospitalier Universitaire de Liege
 2018-08-08
NCT05156320 COMPLETED
Spinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA; Neuromuscular Diseases; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Manifestations; Anti-myostatin
Scholar Rock, Inc.
 2022-04-14 PHASE3
NCT05386680 COMPLETED
Spinal Muscular Atrophy
Novartis Pharmaceuticals
 2023-01-12 PHASE3
NCT05861999 Recruiting
Muscular Atrophy Spinal
Hoffmann-La Roche
 2024-05-31 Phase 4
NCT05861986 Recruiting
Muscular Atrophy Spinal
Hoffmann-La Roche
 2024-04-29 Phase 4
NCT05808764 Not yet recruiting
Muscular Atrophy Spinal
Hoffmann-La Roche
 2024-04-15 Phase 2
NCT05769465 Recruiting
Spinal Muscular Atrophy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
 2023-04-01 --
NCT03779334 ACTIVE_NOT_RECRUITING
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2019-08-07 PHASE2
NCT04718181 COMPLETED
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2021-02-01 PHASE1
NCT05115110 Recruiting
Spinal Muscular Atrophy (SMA)
Hoffmann-La Roche
 2022-06-02 Phase 2|Phase 3
NCT04718181 Completed
Muscular Atrophy Spinal
Hoffmann-La Roche
 2021-02-01 Phase 1
NCT03920865 COMPLETED
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2019-05-16 PHASE1
NCT02913482 COMPLETED
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2016-12-23 PHASE2
NCT03988907 COMPLETED
Spinal Muscular Atrophy
Hoffmann-La Roche
 2019-06-18 PHASE1
NCT02908685 COMPLETED
Muscular Atrophy, Spinal
Hoffmann-La Roche
 2016-10-19 PHASE2
NCT03040635 COMPLETED
Healthy Volunteers
Hoffmann-La Roche
 2017-03-22 PHASE1
NCT03036501 COMPLETED
Healthy
Hoffmann-La Roche
 2017-01-03 PHASE1
NCT02633709 COMPLETED
Spinal Muscular Atrophy
Hoffmann-La Roche
 2016-01-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-05-01)

Check the Risdiplam product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Risdiplam functions as an SMN2 pre-mRNA splicing modifier that directly targets and promotes the correct splicing of SMN2 RNA transcripts. This enhancement boosts the production of functional survival motor neuron protein throughout central nervous system and peripheral tissues, thereby directly addressing motor neuron degradation in patients with spinal muscular atrophy.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.