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Clinical trials for SMA

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   44335   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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    59 result(s) found for: SMA. Displaying page 1 of 3.
    1  2  3  Next»
    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: 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 (Completed) PL (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA) DE (Completed) FR (Completed) GR (Completed) IT (Completed) NL (Ongoing)
    Trial results: View results
    EudraCT Number: 2018-002295-40 Sponsor Protocol Number: 08-06-2018-paracet Start Date*: 2018-09-11
    Sponsor Name:Copenhagen Neuromuscular Center
    Full Title: Pharmacokinetics and safety of treatment with paracetamol in children and adults with spinal muscular atrophy and cerebral palsy
    Medical condition: Spinal muscular atrophy type II (SMA II) Cerebral palsy (CP)
    Disease:
    Population Age: Children, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    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: 2011-004369-34 Sponsor Protocol Number: UMC-NMZ-SMA2011 Start Date*: 2015-04-22
    Sponsor Name:Universtiy Medical Center Utrecht
    Full Title: SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients ...
    Medical condition: Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene...
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10068209 Spinal muscular atrophy adult onset LLT
    17.1 100000004850 10051203 Spinal muscular atrophy congenital LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    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: 2022-000956-12 Sponsor Protocol Number: 277HV101 Start Date*: Information not available in EudraCT
    Sponsor Name:Biogen Idec Research Limited
    Full Title: A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previou...
    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: BE (Trial now transitioned) DE (Ongoing) PT (Prematurely Ended) PL (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: 2008-003915-11 Sponsor Protocol Number: CarniValI (Uni-Koeln-1107) Start Date*: 2009-09-24
    Sponsor Name:University of Utah
    Full Title: Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I)
    Medical condition: Spinal Muscular Atrophy Type I in infants
    Disease: Version SOC Term Classification Code Term Level
    9.1 10051203 Spinal muscular atrophy congenital LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    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: 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: 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: 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: 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: 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: 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: 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: 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: 2017-000266-29 Sponsor Protocol Number: AVXS-101-CL-302 Start Date*: 2018-06-12
    Sponsor Name:AveXis, Inc.
    Full Title: Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous ...
    Medical condition: Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) SE (Prematurely Ended) BE (Completed) ES (Prematurely Ended) NL (Prematurely Ended) IT (Completed)
    Trial results: View results
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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
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