11 completed, 3 active not recruiting, 3 recruiting, 1 approved for marketing. 7 Phase 2, 5 Phase 1, 4 Phase 4, 1 Phase 3.
By phase and status
Follow Risdiplam: new trials by email Literature Review of Risdiplam trials Research Report on Risdiplam
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Apitegromab, Nusinersen, Risdiplam - Spinal Muscular Atrophy, SMA | Phase 2 | Recruiting | Scholar Rock, Inc. | 52 | 2025-09-15 | 2026-10-02 |
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Risdiplam - Muscular Atrophy, Spinal | Phase 4 | Active not recruiting | Hoffmann-La Roche | 19 | 2024-08-14 | 2026-10-22 |
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Risdiplam - Muscular Atrophy, Spinal | Phase 4 | Recruiting | Hoffmann-La Roche | 28 | 2024-05-30 | 2026-10-02 |
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Risdiplam - Muscular Atrophy, Spinal | Phase 2 | Completed | Hoffmann-La Roche | 11 | 2024-04-26 | 2026-06-22 |
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Risdiplam - Spinal Muscular Atrophy | Phase 4 | Unknown | Clinic for Special Children | 10 | 2022-11-06 | 2024-02-07 |
Adult SMA Research and Clinical Hub
Risdiplam, Nusinersen Injectable Product - Spinal Muscular Atrophy (SMA) | | Recruiting | Newcastle-upon-Tyne Hospitals NHS Trust | 600 | 2022-07-29 | 2025-05-18 |
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
RO7204239, Placebo, Risdiplam - Spinal Muscular Atrophy (SMA) | Phase 2 / Phase 3 | Active not recruiting | Hoffmann-La Roche | 97 | 2022-06-02 | 2026-09-09 |
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Risdiplam - Spinal Muscular Atrophy | Phase 4 | Completed | Genentech, Inc. | 403 | 2022-03-29 | 2026-10-22 |
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
Risdiplam - Spinal Muscular Atrophy | | Completed | Bakri Elsheikh | 18 | 2021-10-13 | 2026-02-23 |
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
risdiplam, omeprazole - Muscular Atrophy, Spinal | Phase 1 | Completed | Hoffmann-La Roche | 131 | 2021-02-01 | 2023-03-13 |
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Risdiplam - Muscular Atrophy, Spinal | Phase 2 | Active not recruiting | Hoffmann-La Roche | 26 | 2019-08-07 | 2026-10-08 |
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
Risdiplam, Midazolam - Spinal Muscular Atrophy | Phase 1 | Completed | Hoffmann-La Roche | 35 | 2019-06-18 | 2020-10-19 |
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
Risdiplam - Muscular Atrophy, Spinal | Phase 1 | Completed | Hoffmann-La Roche | 26 | 2019-05-16 | 2021-02-21 |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants
Risdiplam, Placebo - Healthy Volunteers | Phase 1 | Completed | Hoffmann-La Roche | 24 | 2017-03-22 | 2018-10-04 |
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
Risdiplam - Spinal Muscular Atrophy | Phase 2 | Completed | Hoffmann-La Roche | 174 | 2017-03-03 | 2025-10-01 |
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Risdiplam - Muscular Atrophy, Spinal | Phase 2 | Completed | Hoffmann-La Roche | 62 | 2016-12-23 | 2024-08-07 |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Placebo, Risdiplam - Muscular Atrophy, Spinal | Phase 2 | Completed | Hoffmann-La Roche | 231 | 2016-10-19 | 2024-04-24 |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
Itraconazole, Placebo, Risdiplam - Spinal Muscular Atrophy | Phase 1 | Completed | Hoffmann-La Roche | 33 | 2016-01-07 | 2018-10-04 |
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Risdiplam - Muscular Atrophy, Spinal | | Approved for marketing | Genentech, Inc. | | | 2020-10-05 |
Conditions studied
Muscular Atrophy, Spinal (18) · Neuromuscular Manifestations (1) · Spinal Muscular Atrophies of Childhood (1)
Sponsors
Hoffmann-La Roche (13) · Genentech, Inc. (2) · Bakri Elsheikh (1) · Clinic for Special Children (1) · Newcastle-upon-Tyne Hospitals NHS Trust (1) · Scholar Rock, Inc. (1)
Countries
United States (17) · Poland (8) · Belgium (7) · United Kingdom (7) · Italy (6) · Germany (5) · Netherlands (5) · France (4) · Spain (4) · Brazil (3) · Canada (3) · Croatia (3) · Japan (3) · Russia (3) · Australia (2)
Top investigators
Bakri Elsheikh, MBBS (1) · Prof Chiara Marini-Bettolo, MD, PhD (1)
Results publications
PMID 37148485, PMID 33626251, PMID 34320287, PMID 34942136, PMID 35567422, PMID 35906608, PMID 36244364, PMID 38705943, PMID 40802943, PMID 41830075, PMID 42385757, PMID 42413222
NIH funding
2023 (6), 2016 (3), 2015 (1), 2018 (1)