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Clinical trials for SMN1

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   44341   clinical trials with a EudraCT protocol, of which   7370   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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    18 result(s) found for: SMN1. Displaying page 1 of 1.
    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: 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: 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: 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: 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: 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: 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: 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: 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: 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)
    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: 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: View results
    EudraCT Number: 2020-005995-37 Sponsor Protocol Number: COAV101A12306 Start Date*: 2021-08-25
    Sponsor Name:Novartis Pharma AG
    Full Title: A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric pa...
    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: FR (Completed) DE (Completed) BE (Completed) PT (Completed) IT (Completed)
    Trial results: View results
    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: 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: 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)
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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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