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Clinical trials for Pyruvate kinase deficiency

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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    7 result(s) found for: Pyruvate kinase deficiency. Displaying page 1 of 1.
    EudraCT Number: 2021-003265-36 Sponsor Protocol Number: AG348-C-022 Start Date*: 2022-05-10
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly T...
    Medical condition: Pyruvate Kinase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) ES (Ongoing) CZ (Trial now transitioned) DK (Trial now transitioned) IT (Prematurely Ended) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-003333-11 Sponsor Protocol Number: AG348-C-023 Start Date*: 2022-04-25
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regular...
    Medical condition: Pyruvate Kinase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-003459-39 Sponsor Protocol Number: AG348-C-011 Start Date*: 2019-04-10
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects with Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies
    Medical condition: Pyruvate Kinase Deficiency Haemolytic anaemia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed) GB (GB - no longer in EU/EEA) ES (Ongoing) FR (Completed) IE (Completed) NL (Completed) DE (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-000484-13 Sponsor Protocol Number: AG-348-C-003 Start Date*: 2015-07-10
    Sponsor Name:Agios Pharmaceuticals Inc
    Full Title: A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients with Pyruvate Kinase Deficiency
    Medical condition: Pyruvate Kinase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) NL (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-003803-22 Sponsor Protocol Number: AG348-C-007 Start Date*: 2018-06-27
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: An Open-Label Study to Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency
    Medical condition: Pyruvate Kinase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed) DK (Completed) GB (Completed) IE (Completed) IT (Completed) ES (Prematurely Ended) CZ (Completed)
    Trial results: View results
    EudraCT Number: 2019-001656-19 Sponsor Protocol Number: RP-L301-0119 Start Date*: 2019-09-19
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon O...
    Medical condition: Pyruvate kinase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-003823-31 Sponsor Protocol Number: AG348-C-006 Start Date*: 2019-04-08
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Subjects With Pyruvate Kinase Deficiency
    Medical condition: Pyruvate Kinase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) FR (Completed) PT (Completed) ES (Completed) DK (Completed) NL (Completed) CZ (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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