Risdiplam

DRUG19 trials

Sponsors

Hoffmann-La Roche, Genentech, Inc., Bakri Elsheikh, Clinic for Special Children, Newcastle-upon-Tyne Hospitals NHS Trust

Conditions

Anti-myostatinHealthy VolunteersMuscular Atrophy, SpinalNeuromuscular ManifestationsSMASpinal Muscular AtrophySpinal Muscular Atrophy (SMA)Spinal Muscular Atrophy Type 2

Phase 1

A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers

CompletedNCT02633709

Hoffmann-La RocheSpinal Muscular Atrophy

Start: 2016-01-07End: 2016-08-04Updated: 2018-10-04

A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants

CompletedNCT03040635

Hoffmann-La RocheHealthy Volunteers

Start: 2017-03-22End: 2017-10-02Updated: 2018-10-04

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

CompletedNCT03920865

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2019-05-16End: 2020-01-02Updated: 2021-02-21

A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants

CompletedNCT03988907

Hoffmann-La RocheSpinal Muscular Atrophy

Start: 2019-06-18End: 2019-09-29Updated: 2020-10-19

Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

CompletedNCT04718181

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2021-02-01End: 2023-01-28Updated: 2023-03-13

Phase 2

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

CompletedNCT02908685

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2016-10-19End: 2023-10-02Updated: 2024-04-24

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

CompletedNCT02913482

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2016-12-23End: 2023-12-22Updated: 2024-08-07

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

CompletedNCT03032172

Hoffmann-La RocheSpinal Muscular Atrophy

Start: 2017-03-03End: 2025-02-07Updated: 2025-10-01

A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, not recruitingNCT03779334

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2019-08-07End: 2027-02-28Updated: 2026-07-09

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

Active, not recruitingNCT05115110

Hoffmann-La RocheSpinal Muscular Atrophy (SMA)

Start: 2022-06-02End: 2026-10-28Updated: 2026-09-09

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

CompletedNCT05808764

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2024-04-26End: 2026-05-04Updated: 2026-06-22

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

RecruitingNCT07047144

Scholar Rock, Inc.Anti-myostatin, Neuromuscular Manifestations, SMA +3

Start: 2025-09-15End: 2029-03-01Target: 52Updated: 2026-09-14

Phase 4

Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Active, not recruitingNCT05232929

Genentech, Inc.Spinal Muscular Atrophy

Start: 2022-03-29End: 2026-10-02Updated: 2026-09-09

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, not recruitingNCT05522361

Clinic for Special ChildrenSpinal Muscular Atrophy

Start: 2022-11-06End: 2026-06-15Target: 10Updated: 2024-02-07

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

RecruitingNCT05861986

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2024-05-30End: 2029-03-31Target: 28Updated: 2026-09-03

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

RecruitingNCT05861999

Hoffmann-La RocheMuscular Atrophy, Spinal

Start: 2024-08-14End: 2029-03-31Target: 28Updated: 2026-09-10

Unknown Phase

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Genentech, Inc.Muscular Atrophy, Spinal

Updated: 2020-10-05

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

CompletedNCT05219487

Bakri ElsheikhSpinal Muscular Atrophy

Start: 2021-10-13End: 2025-12-31Updated: 2026-02-23

Adult SMA Research and Clinical Hub

RecruitingNCT06978985

Newcastle-upon-Tyne Hospitals NHS TrustSpinal Muscular Atrophy (SMA)

Start: 2022-07-29End: 2027-04-30Target: 600Updated: 2025-05-18

Related Papers

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Open Access CRIS of the University of Bern2026-09-01

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

2026-07-01

Adult SMA REACH: A UK clinical network and real-world data collection study for adults living with spinal muscular atrophy.

2026-03-13

Risdiplam in Presymptomatic Spinal Muscular Atrophy

New England Journal of Medicine2025-08-1312 citations

Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy.

2024-09-249 citations

JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam

Journal of Neurology2024-05-1123 citations

Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy.

2024-05-0511 citations

FIREFISH parts 1 and 2: 5-year efficacy and safety of risdiplam in type 1 SMA

Arquivos de Neuro-Psiquiatria2024-01-01

RAINBOWFISH: primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)

Arquivos de Neuro-Psiquiatria2024-01-01

Safety update: risdiplam clinical trial programme for spinal muscular atrophy (SMA)

2023-11-01

JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment- naïve patients with spinal muscular atrophy (SMA)

2023-11-01

SUNFISH: 4-year efficacy and safety data of risdiplam in types 2 and 3 SMA

2023-11-01

Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug-Drug Interactions in Children.

2023-05-068 citations

SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA) (S34.009)

Neurology2023-04-253 citations

FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA) (P7-9.009)

Neurology2023-04-251 citations

JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (P7-9.004)

Neurology2023-04-25

Risdiplam in non‐sitter patients aged 16 years and older with 5q spinal muscular atrophy

Muscle & Nerve2023-02-2336 citations

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

2023-02-1335 citations

Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA).

2023-02-0362 citations

SUNFISH parts 1 and 2: 4-year efficacy and safety data of risdiplam in types 2 and 3 SMA

2023-01-01

23 more papers not shown