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Clinical trials for Progressive Muscular Atrophy

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
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    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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    129 result(s) found for: Progressive Muscular Atrophy. Displaying page 1 of 7.
    1  2  3  4  5  6  7  Next»
    EudraCT Number: 2006-006845-14 Sponsor Protocol Number: TRO19622 CL E Q 1115-1 Start Date*: 2007-03-20
    Sponsor Name:TROPHOS
    Full Title: Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10041582 Spinal muscular atrophy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2014-001947-18 Sponsor Protocol Number: ISIS396443-CS4 Start Date*: 2015-07-07
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscula...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) SE (Completed) ES (Completed) IT (Completed) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2013-004422-29 Sponsor Protocol Number: ISIS396443-CS3B Start Date*: 2014-11-13
    Sponsor Name:Isis Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Mus...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) GB (Completed) ES (Completed) SE (Completed) DE (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-001910-88 Sponsor Protocol Number: ATYR1940-C-004 Start Date*: 2015-12-17
    Sponsor Name:aTyr Pharma, Inc.
    Full Title: An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Musc...
    Medical condition: Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2021-006781-21 Sponsor Protocol Number: COAV101A12308 Start Date*: 2022-10-31
    Sponsor Name:Novartis Pharma AG
    Full Title: Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials
    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: BE (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) DE (Completed) DK (Trial now transitioned) ES (Ongoing) GR (Prematurely Ended) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000160-28 Sponsor Protocol Number: SMA-002 Start Date*: 2018-11-06
    Sponsor Name:CATALYST PHARMACEUTICALS INC.
    Full Title: Long term safety study 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 (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-003346-27 Sponsor Protocol Number: ATYR1940-C-003 Start Date*: Information not available in EudraCT
    Sponsor Name:ATYR PHARMA, INC.
    Full Title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Fa...
    Medical condition: Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: 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: 2022-000389-16 Sponsor Protocol Number: 1821-FSH-301 Start Date*: 2022-08-08
    Sponsor Name:Fulcrum Therapeutics
    Full Title: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy
    Medical condition: Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) NL (Trial now transitioned) DK (Trial now transitioned) ES (Ongoing) IT (Trial now transitioned)
    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: 2023-000864-67 Sponsor Protocol Number: COAV101A1IC01 Start Date*: 2024-02-02
    Sponsor Name:NOVARTIS BIOCIÊNCIAS S.A
    Full Title: A Phase IV Open-label, single-arm, single-dose, multicenter study to evaluate the saFEty, toLerability and effIcacy of gene replacement therapy with intravenous OAV101(AVXS101) in pediatric patien...
    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, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-020386-24 Sponsor Protocol Number: TRO19622CLEQ1275-1 Start Date*: Information not available in EudraCT
    Sponsor Name:TROPHOS SA
    Full Title: Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.
    Medical condition: Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years)
    Disease: Version SOC Term Classification Code Term Level
    12.1 10041582 Spinal muscular atrophy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing) NL (Completed) BE (Completed) DE (Completed) IT (Completed) GB (Completed)
    Trial results: View results
    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 (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-001088-32 Sponsor Protocol Number: SMA200701 Start Date*: 2008-07-29
    Sponsor Name:ISTITUTO NEUROLOGICO "CARLO BESTA"
    Full Title: A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY.
    Medical condition: Patients affected by SMA
    Disease: Version SOC Term Classification Code Term Level
    9.1 10041582 Spinal muscular atrophy LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    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 (Completed)
    Trial results: View results
    EudraCT Number: 2022-001771-14 Sponsor Protocol Number: SRK-015-004 Start Date*: 2023-05-08
    Sponsor Name:Scholar Rock, Inc.
    Full Title: An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigat...
    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: ES (Ongoing) NL (Ongoing) BE (Trial now transitioned) DE (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-000624-25 Sponsor Protocol Number: ATYR1940-C-006 Start Date*: 2016-06-22
    Sponsor Name:aTyr Pharma, Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy
    Medical condition: Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10064087 Facioscapulohumeral muscular dystrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) IT (Prematurely Ended)
    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 (Completed)
    Trial results: View results
    EudraCT Number: 2022-002301-24 Sponsor Protocol Number: NMD670-02-0001 Start Date*: Information not available in EudraCT
    Sponsor Name:NMD Pharma A/S
    Full Title: A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy
    Medical condition: Type 3 spinal muscular atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10079415 Spinal muscular atrophy type III LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) BE (Trial now transitioned) DK (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned)
    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) FR (Completed) DE (Completed) BE (Completed) NL (Completed) IT (Completed)
    Trial results: (No results available)
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