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Clinical trials for nusinersen and spinal muscular atrophy

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    The EU Clinical Trials Register currently displays   43882   clinical trials with a EudraCT protocol, of which   7296   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).

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    19 result(s) found for: nusinersen and spinal muscular atrophy. Displaying page 1 of 1.
    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 (Ongoing) ES (Prematurely Ended) DE (Ongoing) PL (Completed) HU (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-003492-18 Sponsor Protocol Number: 232SM404 Start Date*: 2021-06-07
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
    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: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Ongoing) IT (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004708-32 Sponsor Protocol Number: 232SM302 Start Date*: Information not available in EudraCT
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
    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: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: EE (Completed) ES (Ongoing) HU (Ongoing) DE (Ongoing) PL (Completed) IT (Ongoing) NL (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    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) DE (Completed) BE (Ongoing) NL (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-004600-22 Sponsor Protocol Number: SMA-001 Start Date*: 2018-04-04
    Sponsor Name:CATALYST PHARMACEUTICALS INC.
    Full Title: A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3
    Medical condition: Spinal Muscular Atrophy (SMA) Type 3
    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: IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-001870-16 Sponsor Protocol Number: ISIS396443-CS11 Start Date*: 2016-03-08
    Sponsor Name:Biogen Idec Research Limited
    Full Title: An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
    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: DE (Completed) GB (GB - no longer in EU/EEA) SE (Completed) ES (Ongoing) FR (Completed) BE (Completed) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2017-000327-27 Sponsor Protocol Number: ISIS396443-CS2 Start Date*: 2017-01-26
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-003657-33 Sponsor Protocol Number: 232SM202 Start Date*: 2015-09-21
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects w...
    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: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-002663-10 Sponsor Protocol Number: 232SM203 Start Date*: 2020-03-27
    Sponsor Name:Biogen Idec Research Limited
    Full Title: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
    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: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: LV (Completed) IE (Completed) HU (Ongoing) PL (Ongoing) ES (Ongoing) GB (GB - no longer in EU/EEA) DE (Completed) FR (Ongoing) GR (Ongoing) IT (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-005007-40 Sponsor Protocol Number: S62874 Start Date*: 2020-02-04
    Sponsor Name:University Hospitals Leuven
    Full Title: Natural history study in adult patients with SMA types 2-3-4 and Role of neurodegenerative and neuro-inflammatory biomarkers in SMA adults treated with nusinersen.
    Medical condition: Adult patients with spinal muscular atrophy (SMA) type 2, type 3, or type 4
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2018-004383-65 Sponsor Protocol Number: SRK-015-002 Start Date*: 2019-10-30
    Sponsor Name:Scholar Rock, Inc.
    Full Title: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy
    Medical condition: Later Onset Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10079415 Spinal muscular atrophy type III LLT
    20.0 100000004850 10079416 Spinal muscular atrophy type II LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed) ES (Ongoing) DE (Completed) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-002098-12 Sponsor Protocol Number: 232SM201 Start Date*: 2015-05-12
    Sponsor Name:Biogen Idec Research Limited
    Full Title: An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptom...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing) GB (GB - no longer in EU/EEA) DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-005314-34 Sponsor Protocol Number: SRK-015-003 Start Date*: Information not available in EudraCT
    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 (Ongoing) DE (Ongoing) FR (Ongoing) ES (Ongoing) NL (Ongoing) IT (Ongoing) PL (Ongoing)
    Trial results: (No results available)
    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 (Ongoing) GR (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 (Ongoing) 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: 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 (Ongoing) DE (Ongoing) BE (Ongoing) IT (Ongoing) PL (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    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 (Ongoing) PL (Ongoing) DE (Ongoing) BE (Ongoing) NL (Ongoing) ES (Ongoing) PT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-000621-12 Sponsor Protocol Number: ISIS396443-CS3A Start Date*: 2017-02-15
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-002053-19 Sponsor Protocol Number: CLMI070X2201 Start Date*: 2014-12-16
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10051203 Spinal muscular atrophy congenital LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) IT (Completed) DE (Completed) BE (Completed) NL (Ongoing) CZ (Completed) PL (Completed) HU (Completed) BG (Completed)
    Trial results: View results
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