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Clinical trials for Zolgensma

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   44336   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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    9 result(s) found for: Zolgensma. Displaying page 1 of 1.
    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: 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: 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: 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: 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: 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)
    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 (Completed) GR (Completed)
    Trial results: (No results available)
    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
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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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