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Clinical trials for Risdiplam

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    10 result(s) found for: Risdiplam. Displaying page 1 of 1.
    EudraCT Number: 2021-003417-19 Sponsor Protocol Number: BN42644 Start Date*: 2022-01-03
    Sponsor Name:F. HOFFMANN - LA ROCHE LTD.
    Full 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 COMBI...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10079415 Spinal muscular atrophy type III LLT
    20.1 100000004850 10051203 Spinal muscular atrophy congenital LLT
    20.1 100000004850 10041583 Spinal muscular atrophy, unspecified LLT
    20.0 100000004850 10079413 Spinal muscular atrophy type I LLT
    20.0 100000004850 10079416 Spinal muscular atrophy type II LLT
    20.0 100000004850 10079417 Spinal muscular atrophy infantile onset LLT
    20.0 100000004850 10079419 Spinal muscular atrophy pre-symptomatic LLT
    20.0 100000004850 10079418 Spinal muscular atrophy later onset LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) PL (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) NL (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) PT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-002087-12 Sponsor Protocol Number: BN40703 Start Date*: 2018-12-07
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10079419 Spinal muscular atrophy pre-symptomatic LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) PL (Trial now transitioned) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-001294-23 Sponsor Protocol Number: 232SM303 Start Date*: Information not available in EudraCT
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam
    Medical condition: Muscular Atrophy, Spinal
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed) IT (Trial now transitioned) ES (Prematurely Ended) DE (Ongoing) PL (Completed) HU (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-000750-35 Sponsor Protocol Number: BP39055 Start Date*: 2018-03-02
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 ...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) GB (GB - no longer in EU/EEA) IT (Completed) BE (Completed) DE (Completed) FR (Ongoing) PL (Completed) HR (Completed) BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-006709-31 Sponsor Protocol Number: COAV101B12302 Start Date*: 2022-11-03
    Sponsor Name:Novartis Farmacéutica, S.A.
    Full Title: Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 1014 vector genomes) to participants 2 to 12 yea...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Completed) DE (Completed) BE (Completed) NL (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-004184-39 Sponsor Protocol Number: BP39054 Start Date*: 2017-09-05
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Trial now transitioned) DE (Completed) FR (Trial now transitioned) NL (Trial now transitioned) BE (Trial now transitioned) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-005314-34 Sponsor Protocol Number: SRK-015-003 Start Date*: 2022-06-01
    Sponsor Name:Scholar Rock, Inc.
    Full Title: Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or ...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10041583 Spinal muscular atrophy, unspecified LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed) DE (Completed) FR (Completed) ES (Ongoing) NL (Completed) IT (Completed) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-000193-25 Sponsor Protocol Number: BHV2000-301 Start Date*: 2022-11-04
    Sponsor Name:Biohaven Pharmaceuticals, Inc
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-L...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) CZ (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-000778-40 Sponsor Protocol Number: BP39056 Start Date*: 2017-01-05
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full 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
    Medical condition: Type 1 Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) ES (Prematurely Ended) IT (Completed) BE (Completed) FR (Completed) PL (Completed) HR (Completed)
    Trial results: View results
    EudraCT Number: 2021-003474-31 Sponsor Protocol Number: COAV101B12301 Start Date*: 2023-06-01
    Sponsor Name:Novartis Pharma AG
    Full Title: A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 1...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) GR (Completed)
    Trial results: (No results available)
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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