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Clinical trials for SMN2

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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    28 result(s) found for: SMN2. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2017-004087-35 Sponsor Protocol Number: AVXS-101-CL-304 Start Date*: 2018-12-21
    Sponsor Name:AveXis, Inc.
    Full Title: A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2
    Medical condition: Spinal Muscular Atrophy
    Disease:
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) IT (Completed) GB (GB - no longer in EU/EEA) BE (Completed) NL (Prematurely Ended)
    Trial results: View results
    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
    EudraCT Number: 2020-000095-38 Sponsor Protocol Number: AVXS-101-CL-303 Start Date*: 2020-03-18
    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: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Newborns, Infants and toddlers, 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: 2014-002246-41 Sponsor Protocol Number: BP29420 Start Date*: 2014-10-23
    Sponsor Name:F. Hoffmann-La Roche Ltd.
    Full Title: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF T...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    17.0 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) ES (Completed)
    Trial results: View results
    EudraCT Number: 2020-003678-28 Sponsor Protocol Number: AVXS-101-CL-102 Start Date*: 2020-09-15
    Sponsor Name:AveXis, Inc.
    Full Title: Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non- Ambulatory Patients with Spinal Muscular Atrophy
    Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    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: 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: 2020-001235-27 Sponsor Protocol Number: AVXS-101-CL-101 Start Date*: 2020-04-06
    Sponsor Name:AveXis, Inc.
    Full Title: Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101
    Medical condition: Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    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: 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: 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: 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
    EudraCT Number: 2019-002611-26 Sponsor Protocol Number: AVXS-101-LT-002 Start Date*: 2020-03-30
    Sponsor Name:AveXis, Inc.
    Full Title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
    Medical condition: Spinal Muscular Atrophy
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) BE (Trial now transitioned) ES (Prematurely Ended) FR (Trial now transitioned) IT (Trial now transitioned)
    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 (Completed) GB (GB - no longer in EU/EEA) 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: 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: 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: 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)
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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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