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Clinical trials for Enzyme replacement therapy

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
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  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
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    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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    89 result(s) found for: Enzyme replacement therapy. Displaying page 1 of 5.
    1  2  3  4  5  Next»
    EudraCT Number: 2017-004370-34 Sponsor Protocol Number: POM-003 Start Date*: 2018-04-09
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy
    Medical condition: Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) HU (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-005491-20 Sponsor Protocol Number: 20151205 Start Date*: 2016-08-29
    Sponsor Name:Actavis Hungary Gyógyszermarketing és Kereskedelmi Korlátolt Felelősségű Társaság
    Full Title: The effect of pancreatic enzyme replacement therapy on glucose metabolism, nutritional parameters, incretin response and beta-cell function in patients with diabetes mellitus and exocrine pancreati...
    Medical condition: To study the patients with diabetes mellitus (type 1, 2 and 3c) and exocrine pancreatic insufficiency.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-004940-31 Sponsor Protocol Number: 18122019 Start Date*: 2020-03-24
    Sponsor Name:Terese Katzenstein
    Full Title: Effect of timing of enzyme replacement therapy (Creon®) at meals on fat malabsorption in adult patients with cystic fibrosis - a cross-over trial
    Medical condition: GI pains and fat malabsorption in cystic fibrosis patients with pancreas insufficiency
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004856 10025479 Malabsorption syndrome LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-002750-31 Sponsor Protocol Number: LAL-CL02 Start Date*: 2013-05-29
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency
    Medical condition: Lysosomal Acid Lipase Deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004915 10024579 Lysosomal storage disorders HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) IT (Completed) ES (Completed) CZ (Completed) PL (Completed) GR (Completed) HR (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2015-000104-26 Sponsor Protocol Number: UX003-CL203 Start Date*: 2015-07-23
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA ES (Prematurely Ended) PT (Completed)
    Trial results: View results
    EudraCT Number: 2006-005216-27 Sponsor Protocol Number: 78993095 Start Date*: 2007-03-26
    Sponsor Name:HUS, Hospital for Children and Adolescents
    Full Title: Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord c...
    Medical condition: Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respir...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10056887 Mucopolysaccharidosis IH/S LLT
    Population Age: Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    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: 2020-005032-30 Sponsor Protocol Number: FLT201-01 Start Date*: 2023-03-13
    Sponsor Name:Freeline Therapeutics Limited
    Full Title: A Phase 1/2, open-label, safety, tolerability and efficacy study of FLT201 in adult patients with Gaucher disease Type 1 (Galileo-1)
    Medical condition: Type 1 Gaucher Disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10010331 - Congenital, familial and genetic disorders 10075697 Gaucher's disease type I PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-003427-38 Sponsor Protocol Number: HGT-GCB-068 Start Date*: 2016-04-07
    Sponsor Name:Shire Human Genetic Therapies
    Full Title: A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease
    Medical condition: Type 3 Gaucher disease
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10075697 Gaucher's disease type I PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-001875-32 Sponsor Protocol Number: UX003-CL202 Start Date*: 2015-11-12
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA PT (Completed)
    Trial results: View results
    EudraCT Number: 2008-005825-12 Sponsor Protocol Number: PB-06-002 Start Date*: 2009-06-04
    Sponsor Name:Protalix Biotherapeutics
    Full Title: Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD)...
    Medical condition: Enfermedad de Gaucher Gaucher Disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) GB (Completed) IT (Completed) DE (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-003798-82 Sponsor Protocol Number: R1578 Start Date*: 2014-01-29
    Sponsor Name:Hull & East Yorkshire Hospitals NHS Trust
    Full Title: Multi-centre Randomised Controlled Trial of Angiotensin Converting Enzyme inhibitor (ACEi) / Angiotensin Receptor Blocker (ARB) withdrawal in advanced renal disease; The STOP-ACEi Trial
    Medical condition: Progressive, advanced (stage 4 or 5) chronic kidney disease (CKD). NB. There was not an appropriate therapeutic area in the drop down menu in E1-1. Have selected 'Male diseases of the urinary and...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10038359 - Renal and urinary disorders 10064848 Chronic kidney disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2017-001227-45 Sponsor Protocol Number: PHT/2017/20 Start Date*: 2017-06-12
    Sponsor Name:Portsmouth Hospitals NHS Trust
    Full Title: Comparison of ambulatory glucose profile prior to and during pancreatic enzyme replacement therapy in patients with diabetes and pancreatic exocrine insufficiency: a single-arm phase IV trial
    Medical condition: Type 1 diabetes and type 2 diabetes mellitus Pancreatic exocrine insufficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 10027433 - Metabolism and nutrition disorders 10067585 Type 2 diabetes mellitus PT
    20.1 10027433 - Metabolism and nutrition disorders 10045228 Type I diabetes mellitus LLT
    20.0 100000004856 10033628 Pancreatic insufficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-002748-10 Sponsor Protocol Number: AD-02-013 Start Date*: 2013-12-12
    Sponsor Name:Fundación para la Investigación en Enfermedades del Aparato Digestivo
    Full Title: A double-blind, randomized, multicenter, placebo-controlled, parallel-group phase IV clinical trial to assess the efficacy of pancreatic enzyme replacement therapy (PERT) in patients with pancreati...
    Medical condition: Patients with pancreatic exocrine insufficiency (PEI) secondary to type 1 diabetes mellitus (DM)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-005310-35 Sponsor Protocol Number: AD-03-013 Start Date*: 2014-08-13
    Sponsor Name:Fundación para la Investigación en Enfermedades del Aparato Digestivo
    Full Title: A phase IV double-blind, randomized, placebo-controlled, multi-center study with pancreatic enzyme replacement therapy (PERT) in subjects suffering from pancreatic exocrine insufficiency (PEI) afte...
    Medical condition: Patients 2 weeks after discharge from the hospital after an attack of acute necrotizing pancreatitis
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-005874-24 Sponsor Protocol Number: PEI004/2021 Start Date*: 2022-07-01
    Sponsor Name:Juan Enrique Domínguez Muñoz
    Full Title: Open label randomized, multicentre, controlled trial of pancreatic enzyme replacement therapy (PERT) for pancreatic exocrine insufficiency (PEI) in patients with unresectable pancreatic cancer
    Medical condition: pancreatic exocrine insufficiency (PEI) in patients with unresectable pancreatic cancer.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-002158-35 Sponsor Protocol Number: IVA_01_ODI_HMPS_17_002 Start Date*: 2017-11-16
    Sponsor Name:Inventiva S.A.
    Full Title: A phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI.
    Medical condition: Mucopolysaccharidosis (MPS) type VI.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) FR (Completed) PT (Completed)
    Trial results: View results
    EudraCT Number: 2016-004141-90 Sponsor Protocol Number: 4.0 Start Date*: 2017-02-28
    Sponsor Name:Queen Mary University of London
    Full Title: Stopping Perioperative Angiotensin II Converting Enzyme inhibitors and/or receptor blockers in major non-cardiac surgery (SPACE): a phase II, explanatory, randomised controlled trial.
    Medical condition: Myocardial injury (as measured by plasma troponin) after major surgery.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10007541 - Cardiac disorders 10061024 Cardiac disorder PT
    20.0 10007541 - Cardiac disorders 10061024 Cardiac disorder PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2007-001163-30 Sponsor Protocol Number: ALID02307 Start Date*: 2015-05-11
    Sponsor Name:Genzyme, a Sanofi Company
    Full Title: A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase)
    Medical condition: Mucopolysaccharidosis I
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-001152-35 Sponsor Protocol Number: UX003-CL201 Start Date*: 2013-08-02
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (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
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