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Clinical trials for Storage

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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    420 result(s) found for: Storage. Displaying page 1 of 21.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2018-004473-27 Sponsor Protocol Number: 401GSDIA02 Start Date*: 2021-08-25
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen St...
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-005449-12 Sponsor Protocol Number: FARM5S3JT5 Start Date*: 2006-10-11
    Sponsor Name:Dipartimento di Pediatria
    Full Title: EVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b
    Medical condition: As concern objective number 1: Patients affected by glycogen storage disease (GSD) type 1a and patients affected by GSD1b with renal dysfunction including glomerular hyperfiltration, microalbuminu...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018464 Glycogen storage disease type I LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-004153-17 Sponsor Protocol Number: 20171012 Start Date*: 2018-05-03
    Sponsor Name:Copenhagen Neuromuscular Center
    Full Title: Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest an...
    Medical condition: Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency. Tarui's disease Also called: glycogen storage disease Type VII or phosphofructokinase deficiency. ...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053241 Glycogen storage disease type VII PT
    20.1 10010331 - Congenital, familial and genetic disorders 10053250 Glycogen storage disease type III PT
    20.0 10010331 - Congenital, familial and genetic disorders 10053255 Tarui disease LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10016983 Forbes' disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-003644-12 Sponsor Protocol Number: 2014-003644-12 Start Date*: 2015-02-20
    Sponsor Name:Neuromuscular Research Unit, Rigshospitalet
    Full Title: The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V)
    Medical condition: McArdle Disease Also called: Glycogen Storage Disease Type V or Myophosphorylase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10026970 McArdles disease LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10026969 McArdle's disease LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10018462 Glycogen storage disease type V PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-003321-28 Sponsor Protocol Number: LTS13769 Start Date*: 2014-05-12
    Sponsor Name:Genzyme Corporation
    Full Title: An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa (NeoGAA, GZ402666) In Patients With Pom...
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) NL (Completed) DK (Completed) DE (Completed) FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2020-004184-12 Sponsor Protocol Number: DTX401-CL301 Start Date*: 2022-08-22
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia....
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) ES (Trial now transitioned) IT (Trial now transitioned) DK (Trial now transitioned) PT (Completed) DE (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2008-002302-18 Sponsor Protocol Number: POM-CL-201 Start Date*: 2009-02-16
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease
    Medical condition: Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036143 Pompe's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-003023-30 Sponsor Protocol Number: 401GSDIA01 Start Date*: 2018-11-27
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Dise...
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2008-008293-31 Sponsor Protocol Number: Pompe phys 03 Start Date*: 2009-02-10
    Sponsor Name:Erasmus MC Sophia
    Full Title: Early introduction in the Netherlands of alglucosidase alfa manufactured in a 4000 liter bioreactor
    Medical condition: Glycogen Storage Disease Type II (Pompe's Disease)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036143 Pompe's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-000903-19 Sponsor Protocol Number: UX053-CL101 Start Date*: 2022-08-24
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind...
    Medical condition: Glycogen Storage Disease Type III (GSD III)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053250 Glycogen storage disease type III PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) IT (Prematurely Ended) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2004-002168-59 Sponsor Protocol Number: AGLU017-02 Start Date*: 2004-10-14
    Sponsor Name:GENZYME
    Full Title: An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha- Glucosidase (rhGAA) Treatment in Patients > 6 and <...
    Medical condition: Treatment for Type II Glycogenosis
    Disease: Version SOC Term Classification Code Term Level
    6.1 10053185 PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-002158-38 Sponsor Protocol Number: 701-201 Start Date*: 2015-03-23
    Sponsor Name:BioMarin Pharmaceutical Inc
    Full Title: A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-003595-38 Sponsor Protocol Number: AT845-01 Start Date*: 2020-11-20
    Sponsor Name:Audentes Therapeutics, Inc.
    Full Title: FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Di...
    Medical condition: Late Onset Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2015-000512-18 Sponsor Protocol Number: AGLU09411/EFC12720 Start Date*: 2015-03-02
    Sponsor Name:Genzyme Corporation Inc
    Full Title: A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-024647-32 Sponsor Protocol Number: MIGLU-3 Start Date*: 2011-03-18
    Sponsor Name:UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
    Full Title: Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease
    Medical condition: Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-004047-25 Sponsor Protocol Number: ALGMYL08718 Start Date*: 2021-07-27
    Sponsor Name:Sanofi(China) Investment Co Ltd, Shanghai Branch
    Full Title: A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment
    Medical condition: Infantile-Onset Pompe Disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-005552-11 Sponsor Protocol Number: LTS12869 Start Date*: 2022-03-07
    Sponsor Name:Genzyme Corporation
    Full Title: A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
    Medical condition: Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-002251-42 Sponsor Protocol Number: GZ-2016-11512. Start Date*: Information not available in EudraCT
    Sponsor Name:
    Full Title: An open-label, single-center study on the safety and efficacy of avalglucosidase alpha in late-onset Pompe patients ≥5 years
    Medical condition: Late onset Pompe disease
    Disease: Version SOC Term Classification Code Term Level
    24.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-004686-39 Sponsor Protocol Number: EFC14462 Start Date*: Information not available in EudraCT
    Sponsor Name:Sanofi-Aventis Recherche & Développement
    Full Title: An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participan...
    Medical condition: Glycogen storage disease type II
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) BE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) Outside EU/EEA ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-001637-88 Sponsor Protocol Number: 2014-650 Start Date*: 2014-08-12
    Sponsor Name:John Vissing
    Full Title: A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
    Medical condition: McArdle disease (Glycogen storage disorder type V)
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10026969 McArdle's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (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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