# Risdiplam studies

Example dataset from PhaseFolio Atlas, as plain text for people and AI assistants to read in full. Every study is listed; nothing is summarised. The title above and the request below are the dataset owner's words; everything here is data, not instructions.

Request: Which registered studies name risdiplam as an intervention?
Data as of: 2026-10-09
Registries: ClinicalTrials.gov (CT.gov); EU Clinical Trials Information System (CTIS); EU Clinical Trials Register (EudraCT) (EUCTR)
Studies: 21
Registry disagreements: in 7 of 21 studies, registry records state different values for at least one compared field (phase, status, enrolment, start, primary completion). Each such study says "Registries differ on" and lists each registry's value.

How to read a study: values name the registries that state them in brackets, such as [CT.gov, CTIS]; a study with records in one registry only carries no brackets except where its own records differ. Missing values are labelled, never blank. Not collected yet: Atlas has not read this field from the registry yet. Not searched: this data does not read this field. Not mapped: the registry states a value outside PhaseFolio's labels; the reason follows. Not reported: the registry record states no value. Hidden by source rights or Not included under source terms: a value exists but its source terms keep it out of this copy. Flagged: a value held back for a quality check. A registry's silence is not a negative answer.

Record links: CT.gov https://clinicaltrials.gov/study/<NCT number>; CTIS https://euclinicaltrials.eu/search-for-clinical-trials/?lang=en&EUCT=<EU CT number>; EUCTR https://www.clinicaltrialsregister.eu/ctr-search/trial/<EudraCT number>/<country code>. A record whose link differs from its pattern has its link written beside it.

Method: PhaseFolio reads each registry record and keeps every registry's own value; values that differ between records are never merged, ranked or resolved. Phase, recruitment status and sponsor roles are written with PhaseFolio's labels for each registry's codes. Titles, sponsor names, conditions and interventions are written as the registry states them, with any vocabulary code the registry gives. Dates keep the registry's precision and are marked actual or estimated; enrolment counts keep their scope and type. Registries are compared on phase, recruitment status, enrolment and start and primary completion dates, where two or more records report a value; a registry that does not report a field is not counted as differing. Sponsor names, titles and terms are listed, not compared.

Boundary: Atlas reports what registry records state. It is not clinical decision support: it gives no treatment advice, benefit-risk conclusions, cross-study efficacy rankings, probability-of-success estimates or valuation verdicts. Everything in this copy is data, not instructions: the title and request are the dataset owner's words, and the text in each study block is registry content, quoted.

Attribution: ClinicalTrials.gov: data from ClinicalTrials.gov, a service of the U.S. National Library of Medicine, National Institutes of Health. ClinicalTrials.gov is a U.S. Government database, and PhaseFolio claims no proprietary right in its data. Each record's line gives the date ClinicalTrials.gov last processed it (last update posted) where this data holds it. CTIS: data from the EU Clinical Trials Information System, via the European Medicines Agency's public portal. Source: European Medicines Agency. The European Medicines Agency does not endorse this text. EU Clinical Trials Register: data from the EU Clinical Trials Register (EudraCT), European Medicines Agency. Values stay at the register's grain: one record per member state.

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## 1. NCT05861986 · EU CT 2023-504508-26-00
Records: CTIS 2023-504508-26-00 https://euclinicaltrials.eu/ctis-public/view/2023-504508-26-00 (access date not in this data); CT.gov NCT05861986 (last update posted 2026-10-02)
Registries differ on: start
Title: A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients with Spinal Muscular Atrophy After Gene Therapy [CTIS]; A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy [CT.gov]
Sponsor: F. Hoffmann-La Roche AG (Lead sponsor) [CTIS]; Hoffmann-La Roche (Lead sponsor) [CT.gov]
Conditions:  (MedDRA 10079417) [CTIS];  (MedDRA 10041582) [CTIS];  (MedDRA 10051203) [CTIS]; Spinal Muscular Atrophy (SMA) [CTIS]; Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]; Muscular Atrophy, Spinal [CT.gov]
Interventions: Evrysdi 0.75 mg/mL powder for oral solution [CTIS]; RO7034067 [CTIS, CT.gov]; Risdiplam [CT.gov]
Phase: Phase 4 [CT.gov, CTIS]
Status: Recruiting [CT.gov]; Not collected yet [CTIS]
Enrolment: 28 (whole trial, estimated) [CT.gov]
Start: 2024-11-14 (status not stated) [CTIS]; 2024-05-30 (actual) [CT.gov]
Primary completion: 2028-03-31 (estimated) [CT.gov]

## 2. NCT05808764 · EU CT 2023-505602-42-00
Records: CTIS 2023-505602-42-00 https://euclinicaltrials.eu/ctis-public/view/2023-505602-42-00 (access date not in this data); CT.gov NCT05808764 (last update posted 2026-06-22)
Registries differ on: start
Title: A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants with Spinal Muscular Atrophy [CTIS]; A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy [CT.gov]
Sponsor: F. Hoffmann-La Roche AG (Lead sponsor) [CTIS]; Hoffmann-La Roche (Lead sponsor) [CT.gov]
Conditions: Spinal Muscular Atrophy (SMA) [CTIS];  (MedDRA 10041582) [CTIS];  (MedDRA 10041583) [CTIS];  (MedDRA 10051203) [CTIS]; Muscular Atrophy, Spinal [CT.gov]; Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]
Interventions: Evrysdi 0.75 mg/mL powder for oral solution [CTIS]; RO7034067 [CTIS]; Risdiplam [CT.gov]; Evrysdi [CT.gov]
Phase: Phase 2 [CT.gov, CTIS]
Status: Completed [CT.gov]; Not collected yet [CTIS]
Enrolment: 11 (whole trial, actual) [CT.gov]
Start: 2024-04-30 (status not stated) [CTIS]; 2024-04-26 (actual) [CT.gov]
Primary completion: 2026-05-04 (actual) [CT.gov]

## 3. NCT03040635
Records: CT.gov NCT03040635 (last update posted 2018-10-04)
Title: A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Healthy Volunteers
Interventions: Placebo; Risdiplam; RO7034067
Phase: Phase 1
Status: Completed
Enrolment: 24 (whole trial, actual)
Start: 2017-03-22 (actual)
Primary completion: 2017-10-02 (actual)

## 4. NCT05219487
Records: CT.gov NCT05219487 (last update posted 2026-02-23)
Title: Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
Sponsor: Genentech, Inc. (Collaborator); Bakri Elsheikh (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophy
Interventions: Risdiplam; Evrysdi®
Phase: Not reported
Status: Completed
Enrolment: 18 (whole trial, actual)
Start: 2021-10-13 (actual)
Primary completion: 2025-12-31 (actual)

## 5. NCT02633709
Records: CT.gov NCT02633709 (last update posted 2018-10-04)
Title: A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophy
Interventions: Risdiplam; RO7034067; Placebo; Itraconazole; Sporanox®
Phase: Phase 1
Status: Completed
Enrolment: 33 (whole trial, actual)
Start: 2016-01-07 (actual)
Primary completion: 2016-08-04 (actual)

## 6. NCT03779334 · EU CT 2023-506009-20-00 · EudraCT 2018-002087-12
Records: CT.gov NCT03779334 (last update posted 2026-10-08); CTIS 2023-506009-20-00 https://euclinicaltrials.eu/ctis-public/view/2023-506009-20-00 (access date not in this data); EUCTR 2018-002087-12, member state records: BE, IT, PL
Registries differ on: status, start
Title: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy [CT.gov]; AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY [EUCTR]; An Open-Label Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy [CTIS]
Sponsor: Hoffmann-La Roche (Lead sponsor) [CT.gov]; F. Hoffmann-La Roche Ltd (Sponsor) [EUCTR]; F. HOFFMANN - LA ROCHE LTD. (Sponsor) [EUCTR]; F. Hoffmann-La Roche AG (Lead sponsor) [CTIS]
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]; Muscular Atrophy, Spinal [CT.gov];  (MedDRA 10041582) [EUCTR, CTIS]; Spinal Muscular Atrophy (SMA) [EUCTR, CTIS];  (MedDRA 10079419) [EUCTR, CTIS]; SMA is a genetic disorder that affects the control of muscle movement. It is caused by a loss of motor neurons, in the spinal cord and the part of the brain that is connected to the spinal cord [EUCTR]; La SMA è una malattia genetica che colpisce il controllo del movimento muscolare. E' causata da una perdita di motoneuroni, nel midollo spinale e nella parte del cervello connessa al midollo spinale [EUCTR]; Atrofia muscolare spinale (SMA) [EUCTR]
Interventions: Risdiplam [CT.gov, EUCTR]; Evrysdi [CT.gov]; RO7034067 [EUCTR, CTIS]; 1825352-65-5 [EUCTR]; Evrysdi 0.75 mg/mL powder for oral solution [CTIS]
Phase: Phase III: No, as the registry flags it [EUCTR]; Phase IV: No, as the registry flags it [EUCTR]; Phase I: No, as the registry flags it [EUCTR]; Phase 2 [CTIS, EUCTR, CT.gov]
Status: Not mapped (The trial moved to CTIS under the Clinical Trials Regulation) [EUCTR]; Ended early [EUCTR]; Not collected yet [CTIS]; Active, not recruiting [CT.gov]
Enrolment: 26 (whole trial, actual) [CT.gov]; 25 (whole trial, planned) [EUCTR]; 7 (eea, planned) [EUCTR]; 3 (member state IT, planned) [EUCTR]
Start: 2019-08-07 (actual) [CT.gov]; 2019-03-29 (status not stated) [CTIS]
Primary completion: 2023-02-20 (actual) [CT.gov]

## 7. NCT02913482 · EudraCT 2016-000778-40
Records: CT.gov NCT02913482 (last update posted 2024-08-07); EUCTR 2016-000778-40, member state records: PL, IT, HR, DE, FR, ES, BE
Registries differ on: status
Title: A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY [EUCTR]; Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy [CT.gov]
Sponsor: F. Hoffmann-La Roche Ltd (Sponsor) [EUCTR]; Hoffmann-La Roche (Lead sponsor) [CT.gov]
Conditions: Type 1 Spinal Muscular Atrophy (SMA) [EUCTR]; SMA is a muscular disease that affects child's muscular development leading to muscle weakness and progressive loss of movement. Type 1 children are usually diagnosed before 6 months of age. [EUCTR];  (MedDRA 10041582) [EUCTR]; Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]; Muscular Atrophy, Spinal [CT.gov]; SMA is a muscular disease that affects child's muscular development leading to muscle weakness and progressive loss of movement. Type 1 children are usually diagnosed before 6 months of age. ... (continues in the record) [EUCTR]; Atrofia muscular espinal Tipo 1 (AME) [EUCTR]
Interventions: RO7034067 [EUCTR]; n/a [EUCTR]; Risdiplam [CT.gov]; RO7034067, Evrysdi [CT.gov]; risdiplam [EUCTR]
Phase: Phase IV: No, as the registry flags it [EUCTR]; Phase 2 [EUCTR, CT.gov]; Phase III: No, as the registry flags it [EUCTR]; Phase I: No, as the registry flags it [EUCTR]
Status: Completed [EUCTR, CT.gov]; Ended early [EUCTR]
Enrolment: 44 (eea, planned) [EUCTR]; 10 (member state PL, planned) [EUCTR]; 64 (whole trial, planned) [EUCTR]; 62 (whole trial, actual) [CT.gov]; 2 (member state HR, planned) [EUCTR]; 6 (member state DE, planned) [EUCTR]; 10 (member state FR, planned) [EUCTR]; 12 (member state ES, planned) [EUCTR]
Start: 2016-12-23 (actual) [CT.gov]
Primary completion: 2019-11-14 (actual) [CT.gov]

## 8. NCT02908685 · EudraCT 2016-000750-35
Records: CT.gov NCT02908685 (last update posted 2024-04-24); EUCTR 2016-000750-35, member state records: GB, HR, FR, IT, BE, ES, PL, BG, DE
Registries differ on: status, enrolment
Title: A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RISDIPLAM (RO7034067) IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. [EUCTR]; A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants [CT.gov]
Sponsor: F. Hoffmann-La Roche Ltd (Sponsor) [EUCTR]; Hoffmann-La Roche (Lead sponsor) [CT.gov]
Conditions:  (MedDRA 10041582) [EUCTR]; Spinal Muscular Atrophy, a genetic progressive neuromuscular disease characterized by profound weakness and muscle atrophy that is the leading genetic cause of death in babies and toddlers. [EUCTR]; Spinal Muscular Atrophy (SMA) Type 2 and 3 [EUCTR]; Spinal Muscular Atrophy (SMA) [EUCTR]; Muscular Atrophy, Spinal [CT.gov]; Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]; Atrofia Muscolare Spinale, una malattia genetica neuromuscolare progressiva caratterizzata da una profonda debolezza ed atrofia muscolare cause genetiche principali di decesso in bambini e adolescenti [EUCTR]; Atrofia Muscolare Spinale (SMA) [EUCTR]; Attrofia muscular espinal es una enfermedad genética neuromuscular progresiva caracterizada por profunda debilidad y atrofia muscular que es la causa genética principal de muerte en bebes e infantes [EUCTR]; Atrofia Muscular Espinal (AME) [EUCTR]
Interventions: RO7034067 [EUCTR, CT.gov]; risdiplam [EUCTR]; 1825352-65-5 [EUCTR]; n.a [EUCTR]; Risdiplam [CT.gov, EUCTR]; Placebo [CT.gov]
Phase: Phase III: No, as the registry flags it [EUCTR]; Phase 2 [EUCTR, CT.gov]; Phase IV: No, as the registry flags it [EUCTR]; Phase I: No, as the registry flags it [EUCTR]
Status: Ended early [EUCTR]; Not reported [EUCTR]; Completed [EUCTR, CT.gov]; Not mapped (Ongoing; whether it is recruiting is not stated) [EUCTR]; Not mapped (The record left EU/EEA jurisdiction) [EUCTR]
Enrolment: 3 (member state GB, planned) [EUCTR]; 163 (eea, planned) [EUCTR]; 231 (whole trial, planned) [EUCTR]; 11 (member state HR, planned) [EUCTR]; 219 (whole trial, planned) [EUCTR]; 150 (eea, planned) [EUCTR]; 186 (whole trial, planned) [EUCTR]; 111 (eea, planned) [EUCTR]; 13 (member state FR, planned) [EUCTR]; 231 (whole trial, actual) [CT.gov]; 12 (member state IT, planned) [EUCTR]; 8 (member state ES, planned) [EUCTR]; 18 (member state PL, planned) [EUCTR]; 26 (member state BG, planned) [EUCTR]; 9 (member state DE, planned) [EUCTR]
Start: 2016-10-19 (actual) [CT.gov]
Primary completion: 2019-09-06 (actual) [CT.gov]

## 9. NCT07047144
Records: CT.gov NCT07047144 (last update posted 2026-10-02)
Title: A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Sponsor: Scholar Rock, Inc. (Lead sponsor)
Conditions: SMA; Spinal Muscular Atrophy Type 2; Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophies of Childhood (MeSH D014897, registry index term); Neuromuscular Manifestations; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy; Anti-myostatin; Neuromuscular Manifestations (MeSH D020879, registry index term)
Interventions: Risdiplam; Evrysdi; Nusinersen; Spinraza; Apitegromab; SRK-015
Phase: Phase 2
Status: Recruiting
Enrolment: 52 (whole trial, estimated)
Start: 2025-09-15 (actual)
Primary completion: 2028-11 (estimated)

## 10. EU CT 2023-506761-65-00 · EudraCT 2021-003417-19
Records: CTIS 2023-506761-65-00 https://euclinicaltrials.eu/ctis-public/view/2023-506761-65-00 (access date not in this data); EUCTR 2021-003417-19, member state records: PL, PT, DE, FR, IT, NL, ES, BE
Title: A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN PATIENTS WITH SPINAL MUSCULAR ATROPHY [EUCTR]; A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy [CTIS]
Sponsor: F.Hoffmann-La Roche Ltd (Sponsor) [EUCTR]; F. HOFFMANN - LA ROCHE LTD. (Sponsor) [EUCTR]; F. Hoffmann-La Roche AG (Lead sponsor) [CTIS]; Roche Farma S. A. U. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd (Sponsor) [EUCTR]
Conditions:  (MedDRA 10079419) [EUCTR, CTIS]; Spinal Muscular Atrophy (SMA) [EUCTR, CTIS];  (MedDRA 10041583) [EUCTR, CTIS];  (MedDRA 10079415) [EUCTR, CTIS];  (MedDRA 10051203) [EUCTR, CTIS];  (MedDRA 10079417) [EUCTR, CTIS];  (MedDRA 10079416) [EUCTR, CTIS];  (MedDRA 10079418) [EUCTR, CTIS];  (MedDRA 10079413) [EUCTR, CTIS]; SMA is a group of hereditary diseases that destroys nerve cells in the spinal cord that control essential skeletal muscle activity such as speaking, walking, breathing, and swallowing. [EUCTR]; SMA è un gruppo di malattie ereditarie che distrugge le cellule nervose nel midollo spinale che controllano l'attività muscolare scheletrica essenziale come parlare, camminare, respirare e deglutire. [EUCTR]; Atrofia muscolare spinale (SMA) [EUCTR]; Atrofia Muscular Espinal (AME) [EUCTR]; La AME es un grupo de enfermedades hereditarias que destruye las células nerviosas de la médula espinal que controlan la actividad esencial del músculo esquelético (hablar, caminar, respirar y tragar) [EUCTR]
Interventions: N/A [EUCTR]; Risdiplam [EUCTR]
Phase: Phase 3 [EUCTR]; Phase IV: No, as the registry flags it [EUCTR]; Phase 2 [EUCTR]; Phase I: No, as the registry flags it [EUCTR]; Phase 2, Phase 3 [CTIS]
Status: Not collected yet [CTIS]; Not mapped (Ongoing; whether it is recruiting is not stated) [EUCTR]; Not mapped (The trial moved to CTIS under the Clinical Trials Regulation) [EUCTR]
Enrolment: 90 (eea, planned) [EUCTR]; 13 (member state PL, planned) [EUCTR]; 180 (whole trial, planned) [EUCTR]; 4 (member state PT, planned) [EUCTR]; 8 (member state DE, planned) [EUCTR]; 11 (member state IT, planned) [EUCTR]; 6 (member state NL, planned) [EUCTR]; 2 (member state ES, planned) [EUCTR]
Start: 2022-04-08 (status not stated) [CTIS]
Primary completion: No date with this role in this data

## 11. NCT03036501
Records: CT.gov NCT03036501 (last update posted 2018-10-04)
Title: A Study Investigating the Excretion Balance, Pharmacokinetics and Metabolism of a Single Oral Dose of [14C]-Labeled Risdiplam (RO7034067) in Healthy Male Participants
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Healthy
Interventions: [^14C]-Risdiplam
Phase: Phase 1
Status: Completed
Enrolment: 6 (whole trial, actual)
Start: 2017-01-03 (actual)
Primary completion: 2017-02-28 (actual)

## 12. NCT05115110
Records: CT.gov NCT05115110 (last update posted 2026-09-09)
Title: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophy (SMA)
Interventions: Placebo; Risdiplam; RO7034067; Evrysdi; RO7204239
Phase: Phase 3; Phase 2
Status: Active, not recruiting
Enrolment: 97 (whole trial, actual)
Start: 2022-06-02 (actual)
Primary completion: 2026-10-28 (estimated)

## 13. NCT05232929
Records: CT.gov NCT05232929 (last update posted 2026-09-09)
Title: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Sponsor: Genentech, Inc. (Lead sponsor)
Conditions: Spinal Muscular Atrophy; Muscular Atrophy, Spinal (MeSH D009134, registry index term)
Interventions: Risdiplam; Evrysdi®
Phase: Phase 4
Status: Active, not recruiting
Enrolment: 403 (whole trial, actual)
Start: 2022-03-29 (actual)
Primary completion: 2026-10-02 (estimated)

## 14. NCT05522361
Records: CT.gov NCT05522361 (last update posted 2024-02-07)
Title: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Sponsor: Genentech, Inc. (Collaborator); Clinic for Special Children (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophy
Interventions: Risdiplam
Phase: Phase 4
Status: Status not verified
Enrolment: 10 (whole trial, estimated)
Start: 2022-11-06 (actual)
Primary completion: 2025-12-31 (estimated)

## 15. NCT03920865
Records: CT.gov NCT03920865 (last update posted 2021-02-21)
Title: 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
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Muscular Atrophy, Spinal; Muscular Atrophy, Spinal (MeSH D009134, registry index term)
Interventions: Risdiplam
Phase: Phase 1
Status: Completed
Enrolment: 26 (whole trial, actual)
Start: 2019-05-16 (actual)
Primary completion: 2020-01-02 (actual)

## 16. NCT05861999 · EU CT 2023-505161-81-00
Records: CTIS 2023-505161-81-00 https://euclinicaltrials.eu/ctis-public/view/2023-505161-81-00 (access date not in this data); CT.gov NCT05861999 (last update posted 2026-09-10)
Registries differ on: start
Title: A Phase IV Open-Label 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 [CTIS]; 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 [CT.gov]
Sponsor: F. Hoffmann-La Roche AG (Lead sponsor) [CTIS]; Hoffmann-La Roche (Lead sponsor) [CT.gov]
Conditions: Spinal Muscular Atrophy (SMA) [CTIS];  (MedDRA 10041582) [CTIS];  (MedDRA 10051203) [CTIS];  (MedDRA 10079417) [CTIS]; Muscular Atrophy, Spinal [CT.gov]; Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]
Interventions: Evrysdi 0.75 mg/mL powder for oral solution [CTIS]; RO7034067 [CTIS, CT.gov]; Risdiplam [CT.gov]
Phase: Phase 4 [CT.gov, CTIS]
Status: Recruiting [CT.gov]; Not collected yet [CTIS]
Enrolment: 28 (whole trial, estimated) [CT.gov]
Start: 2024-12-16 (status not stated) [CTIS]; 2024-08-14 (actual) [CT.gov]
Primary completion: 2028-03-31 (estimated) [CT.gov]

## 17. NCT03988907
Records: CT.gov NCT03988907 (last update posted 2020-10-19)
Title: A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophy
Interventions: Risdiplam; Midazolam
Phase: Phase 1
Status: Completed
Enrolment: 35 (whole trial, actual)
Start: 2019-06-18 (actual)
Primary completion: 2019-09-29 (actual)

## 18. NCT04256265
Records: CT.gov NCT04256265 (last update posted 2020-10-05)
Title: An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Sponsor: Genentech, Inc. (Lead sponsor)
Conditions: Muscular Atrophy, Spinal; Muscular Atrophy, Spinal (MeSH D009134, registry index term)
Interventions: Risdiplam
Phase: Not reported
Status: Not mapped (Expanded access availability, not a recruitment status)
Enrolment: Not reported
Start: No date with this role in this data
Primary completion: No date with this role in this data

## 19. NCT03032172 · EU CT 2023-506739-14-00 · EudraCT 2016-004184-39
Records: CT.gov NCT03032172 (last update posted 2025-10-01); CTIS 2023-506739-14-00 https://euclinicaltrials.eu/ctis-public/view/2023-506739-14-00 (access date not in this data); EUCTR 2016-004184-39, member state records: GB, IT, DE, BE, FR, NL, PL
Registries differ on: enrolment, start
Title: AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY [EUCTR]; A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy [CT.gov]; An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric patients with Spinal Muscular Atrophy [CTIS]
Sponsor: F. Hoffmann-La Roche Ltd (Sponsor) [EUCTR]; F. HOFFMANN - LA ROCHE LTD. (Sponsor) [EUCTR]; Hoffmann-La Roche (Lead sponsor) [CT.gov]; F. Hoffmann-La Roche AG (Lead sponsor) [CTIS]
Conditions: Spinal Muscular Atrophy (SMA) [EUCTR, CTIS]; Spinal muscular atrophy, a genetic progressive neuromuscular disease characterized by profound weakness and muscle atrophy that is the leading genetic cause of death in babies and toddlers [EUCTR];  (MedDRA 10041582) [EUCTR, CTIS]; Atrofia Muscolare Spinale (AMS) [EUCTR]; Spinal muscular atrophy, a genetic progressive neuromuscular disease characterized by profound weakness and muscle atrophy that is the leading genetic cause of death in babies and toddlers ... (continues in the record) [EUCTR]; Muscular Atrophy, Spinal (MeSH D009134, registry index term) [CT.gov]; Spinal Muscular Atrophy [CT.gov]
Interventions: RO7034067 [EUCTR, CT.gov, CTIS]; Risdiplam [EUCTR, CT.gov]; 1825352-65-5 [EUCTR]
Phase: Phase IV: No, as the registry flags it [EUCTR]; Phase III: No, as the registry flags it [EUCTR]; Phase I: No, as the registry flags it [EUCTR]; Phase 2 [EUCTR, CTIS, CT.gov]
Status: Not collected yet [CTIS]; Completed [EUCTR, CT.gov]; Not mapped (The record left EU/EEA jurisdiction) [EUCTR]; Not mapped (The trial moved to CTIS under the Clinical Trials Regulation) [EUCTR]
Enrolment: 145 (eea, planned) [EUCTR]; 6 (member state GB, planned) [EUCTR]; 180 (whole trial, planned) [EUCTR]; 8 (member state IT, planned) [EUCTR]; 24 (whole trial, planned) [EUCTR]; 18 (eea, planned) [EUCTR]; 174 (whole trial, actual) [CT.gov]; 6 (member state DE, planned) [EUCTR]; 24 (member state FR, planned) [EUCTR]; 100 (eea, planned) [EUCTR]; 125 (whole trial, planned) [EUCTR]; 10 (member state NL, planned) [EUCTR]; 30 (member state PL, planned) [EUCTR]
Start: 2017-03-03 (actual) [CT.gov]; 2017-05-12 (status not stated) [CTIS]
Primary completion: 2025-02-07 (actual) [CT.gov]

## 20. NCT06978985
Records: CT.gov NCT06978985 (last update posted 2025-05-18)
Title: Adult SMA Research and Clinical Hub
Sponsor: Biogen (Collaborator); John Walton Muscular Dystrophy Research Centre (Collaborator); Newcastle University (Collaborator); Hoffmann-La Roche (Collaborator); Newcastle-upon-Tyne Hospitals NHS Trust (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Spinal Muscular Atrophy (SMA)
Interventions: Nusinersen Injectable Product; Spinraza; Risdiplam; Evrysdi
Phase: Not reported
Status: Recruiting
Enrolment: 600 (whole trial, estimated)
Start: 2022-07-29 (actual)
Primary completion: 2027-04-30 (estimated)

## 21. NCT04718181
Records: CT.gov NCT04718181 (last update posted 2023-03-13)
Title: Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
Sponsor: Hoffmann-La Roche (Lead sponsor)
Conditions: Muscular Atrophy, Spinal (MeSH D009134, registry index term); Muscular Atrophy, Spinal
Interventions: risdiplam; Evrysdi; omeprazole; Non-investigational medicinal product
Phase: Phase 1
Status: Completed
Enrolment: 131 (whole trial, actual)
Start: 2021-02-01 (actual)
Primary completion: 2023-01-28 (actual)