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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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    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44365   clinical trials with a EudraCT protocol, of which   7389   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 4 of 5.
    « Previous 1  2  3  4  5  Next»
    EudraCT Number: 2018-002878-50 Sponsor Protocol Number: NN9535-4321 Start Date*: 2019-04-02
    Sponsor Name:Novo Nordisk A/S
    Full Title: Effect of semaglutide versus placebo on the progression of renal impairment in subjects with type 2 diabetes and chronic kidney disease
    Medical condition: Diabetes Mellitus, Type 2 Chronic kidney disease
    Disease: Version SOC Term Classification Code Term Level
    23.1 10038359 - Renal and urinary disorders 10064848 Chronic kidney disease PT
    21.1 100000004861 10045242 Type II diabetes mellitus LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) HU (Completed) NL (Completed) SK (Completed) BG (Completed) DE (Completed) ES (Ongoing) FR (Completed) PL (Completed) BE (Completed) GR (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2010-022709-16 Sponsor Protocol Number: AT1001-013 Start Date*: 2011-09-12
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male
    Trial protocol: BE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-001528-23 Sponsor Protocol Number: PB-102-F50 Start Date*: 2017-10-18
    Sponsor Name:Protalix Ltd.
    Full Title: A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 week...
    Medical condition: Fabry disease (α-galactosidase A deficiency)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) ES (Completed) CZ (Completed) DK (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-003529-17 Sponsor Protocol Number: ARCADIA Start Date*: 2008-11-17
    Sponsor Name:IST. DI RICERCHE FARMACOLOG. M. NEGRI
    Full Title: A PROSPECTIVE, RANDOMIZED, OPEN LABEL, BLINDED END-POINT (PROBE) TRIAL TO EVALUATE WHETHER, AT COMPARABLE BLOOD PRESSURE CONTROL, ACE INHIBITOR THERAPY MORE EFFECTIVELY THAN NON RAS INHIBITOR THERA...
    Medical condition: End stage renal disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10038359 - Renal and urinary disorders 10029151 Nephropathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2017-004878-34 Sponsor Protocol Number: ATR-101-202 Start Date*: 2018-09-18
    Sponsor Name:Millendo Therapeutics, Inc.
    Full Title: A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
    Medical condition: Classic Congenital Adrenal Hyperplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10010323 Congenital adrenal hyperplasia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2007-002840-21 Sponsor Protocol Number: HGT-GCB-039 Start Date*: 2007-10-30
    Sponsor Name:Shire Human Genetic Therapies Inc
    Full Title: A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gauc...
    Medical condition: Type I Gaucher disease Enfermedad de Gaucher tipo 1
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-007158-36 Sponsor Protocol Number: GAU-CL-202X Start Date*: 2009-02-25
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease.
    Medical condition: Type I Gaucher Disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2007-002423-33 Sponsor Protocol Number: BVT.BSSL-020 Start Date*: 2007-10-30
    Sponsor Name:BIOVITRUM AB
    Full Title: A prospective, randomised, double-blind crossover study comparing 0.15 g/L rhBSSL added to infant formula versus placebo during one week of treatment in preterm infants born before week 32 of gesta...
    Medical condition: Enzyme replacement therapy in pre-term infants. Reduced fad uptake capabilities due to prematurity of pre-term infants
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013317 Lipid metabolism disorders HLGT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2010-023192-26 Sponsor Protocol Number: MVC-001-MiTox Start Date*: 2011-04-20
    Sponsor Name:mib Dienstleistungsgesellschaft mbH
    Full Title: EFFECT OF SWITCHING FROM NRTIS TO MARAVIROC ON NRTI-ASSOCIATED MITCHONDRIAL TOXICITY AND IMMUNE ACTIVATION
    Medical condition: Increases in inflammatory biomarkers have been associated with various non-AIDS complications such as cardiovascular events. Maraviroc has no known toxic effects on mitochondria and has been shown ...
    Disease: Version SOC Term Classification Code Term Level
    13.1 10027433 - Metabolism and nutrition disorders 10053961 Mitochondrial toxicity PT
    13.1 10021881 - Infections and infestations 10008919 Chronic HIV infection LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-003676-37 Sponsor Protocol Number: PB-06-006 Start Date*: 2011-11-15
    Sponsor Name:Protalix Biotherapeutics
    Full Title: A Multicenter Extension Study of Taliglucerase alfa in Pediatric Subjects with Gaucher Disease
    Medical condition: Gaucher disease
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10018048 Gaucher's disease PT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2011-003685-32 Sponsor Protocol Number: PB-06-007 Start Date*: 2012-01-27
    Sponsor Name:Protalix Biotherapeutics
    Full Title: A Multicenter Extension Study of Taliglucerase alfa in Adult Subjects with Gaucher Disease
    Medical condition: Gaucher disease
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10018048 Gaucher's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2023-000624-11 Sponsor Protocol Number: LPS16583 Start Date*: 2023-06-21
    Sponsor Name:Genzyme Europe B.V.
    Full Title: A Phase 4, open label, safety and efficacy study of Fabrazyme® (agalsidase beta) as enzyme replacement therapy in Chinese participants with Fabry Disease
    Medical condition: Fabry’s disease
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2023-001027-16 Sponsor Protocol Number: LPS16578 Start Date*: 2023-11-22
    Sponsor Name:Genzyme Europe B. V
    Full Title: A Phase 4, single-arm, open-label safety and efficacy study of Aldurazyme® (laronidase) as enzyme replacement therapy in participants with Mucopolysaccharidosis I (MPS I) in China
    Medical condition: Mucopolysaccharidosis I
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-005402-10 Sponsor Protocol Number: EFC17215 Start Date*: 2022-03-23
    Sponsor Name:Sanofi-aventis recherche & développement
    Full Title: A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher...
    Medical condition: Gaucher's disease type III
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10075699 Gaucher's disease type III PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) HU (Completed) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2009-010576-21 Sponsor Protocol Number: CA180-274 Start Date*: 2009-08-26
    Sponsor Name:Bristol Myers Squibb International Corporation
    Full Title: Randomized phase II of CCNU versus CCNU-dasatinib in patients with recurrent glioblastoma
    Medical condition: recurrent glioblastoma
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018336 Glioblastoma LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed) DE (Prematurely Ended) IT (Prematurely Ended) GB (Completed) BE (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2020-003947-27 Sponsor Protocol Number: TAK-981-1002 Start Date*: 2021-07-04
    Sponsor Name:Takeda Development Center Americas, Inc.
    Full Title: An Open Label, Dose-Escalation, Phase 1/2 Study to Evaluate the Safety, Tolerability, Preliminary Efficacy, and Pharmacokinetics of TAK-981 in Adult Patients With Advanced or Metastatic Solid Tumor...
    Medical condition: Advanced or Metastatic Solid Tumors or Relapsed/Refractory Hematologic Malignancies
    Disease: Version SOC Term Classification Code Term Level
    27.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10066481 Hematological malignancy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PL (Completed) ES (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2007-005097-31 Sponsor Protocol Number: CA182-025 Start Date*: 2008-08-19
    Sponsor Name:Bristol Myers Squibb International Corporation
    Full Title: A Blinded, Randomized, Phase 1/2 Study of Brivanib Alaninate vs. Placebo in Combination with Erbitux and Irinotecan in K-Ras wild type Subjects with Metastatic Colorectal Cancer. Revised Protocol...
    Medical condition: Patients with advanced metastatic colorectal cancer (MCRC)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10061451 Colorectal cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) DK (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2008-001965-27 Sponsor Protocol Number: HGT-GCB-044 Start Date*: 2008-10-29
    Sponsor Name:Shire Human Genetic Therapies, Inc.
    Full Title: An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease
    Medical condition: Type I Gaucher disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2020-003200-14 Sponsor Protocol Number: JR-141-GS31 Start Date*: 2022-05-16
    Sponsor Name:JCR Pharmaceuticals Co., Ltd.
    Full Title: A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients.
    Medical condition: Mucopolysaccharidosis type II
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-021986-60 Sponsor Protocol Number: GFT505-210-5 Start Date*: 2010-12-07
    Sponsor Name:GENFIT
    Full Title: A Pilot study to evaluate the Efficacy and Safety of GFT505 (80mg) orally administered once daily for 12 weeks in patients with Type 2 Diabetes mellitus. A Multicentre, Randomised, Double Blind, Pl...
    Medical condition: Patients with Type 2 Diabetes Mellitus.
    Disease: Version SOC Term Classification Code Term Level
    12.1 10045242 Type II diabetes mellitus LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: LV (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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