Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Sep 3, 2026, 3:09 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–23 of 23 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Intervention: Risdiplam Clear all
Active, not recruiting Phase 3 Interventional

A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)

NCT05067790
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
15 Years to 50 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 2 Interventional Results available

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

NCT02908685
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Placebo, Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 25 Years
Enrollment
231 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
2
States / cities
Palo Alto, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 23, 2024 · Synced Sep 3, 2026, 3:09 PM EDT
Completed No phase listed Observational

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

NCT05219487
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Bakri Elsheikh
Other
Eligibility
18 Years to 70 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 22, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Not listed Phase 4 Interventional

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

NCT05522361
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Clinic for Special Children
Other
Eligibility
2 Years to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 3 Interventional

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320
View directory record Official record
Conditions
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
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

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

NCT03988907
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam, Midazolam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 55 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 18, 2020 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 2 Interventional Results available

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

NCT03032172
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
6 Months to 60 Years
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
4
States / cities
Palo Alto, California • Orlando, Florida • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2025 · Synced Sep 3, 2026, 3:09 PM EDT
Active, not recruiting Phase 2 Interventional Results available

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

NCT03779334
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
1 Day to 6 Weeks
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Recruiting Phase 3 Interventional

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

NCT07444476
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Salanersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
15 Years to 60 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed No phase listed Observational

Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy

NCT05518773
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced Sep 3, 2026, 3:09 PM EDT
Recruiting Phase 2 Interventional

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

NCT07047144
View directory record Official record
Conditions
Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Nusinersen, Risdiplam
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
Up to 2 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
14
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Recruiting Phase 4 Interventional

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

NCT05861986
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months to 24 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
11
States / cities
Little Rock, Arkansas • Aurora, Colorado • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

NCT04718181
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
risdiplam, omeprazole
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 55 Years
Enrollment
131 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
4
States / cities
Daytona Beach, Florida • Springfield, Missouri • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2023 · Synced Sep 3, 2026, 3:09 PM EDT
Recruiting Phase 4 Interventional

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

NCT05861999
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months to 24 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
10
States / cities
Little Rock, Arkansas • Madera, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

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

NCT03920865
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 70 Years
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
3
States / cities
Miami, Florida • Orlando, Florida • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Sep 3, 2026, 3:09 PM EDT
Active, not recruiting Phase 4 Interventional

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

NCT05232929
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
Not listed
Enrollment
403 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
37
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 31 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Approved For Marketing No phase listed Expanded access

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

NCT04256265
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
2 Months and older
U.S. locations
29
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2020 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 2 Interventional

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

NCT05808764
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
Up to 19 Days
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
3
States / cities
Chicago, Illinois • Ann Arbor, Michigan • Gordonville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

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

NCT03040635
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Risdiplam, Placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 60 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017
U.S. locations
1
States / cities
Garden Grove, California
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced Sep 3, 2026, 3:09 PM EDT
Completed No phase listed Observational

A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies

NCT07378943
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
4,114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 2 Interventional Results available

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

NCT02913482
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
1 Month to 7 Months
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
4
States / cities
Palo Alto, California • Chicago, Illinois • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Sep 3, 2026, 3:09 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

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

NCT05115110
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
RO7204239, Placebo, Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 25 Years
Enrollment
259 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
Orlando, Florida • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Sep 3, 2026, 3:09 PM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Sep 3, 2026, 3:09 PM EDT