Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Protein 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
  • 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   44343   clinical trials with a EudraCT protocol, of which   7372   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    163 result(s) found for: Protein replacement therapy. Displaying page 1 of 9.
    1  2  3  4  5  6  7  8  9  Next»
    EudraCT Number: 2017-003573-34 Sponsor Protocol Number: 270-302 Start Date*: 2018-02-23
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemoph...
    Medical condition: Haemophilia A
    Disease:
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Prematurely Ended) FR (Completed) BE (Completed) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-003215-19 Sponsor Protocol Number: 270-301 Start Date*: 2017-11-25
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Res...
    Medical condition: Haemophilia A
    Disease:
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) ES (Ongoing) FR (Completed) BE (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-003652-52 Sponsor Protocol Number: 991 Start Date*: 2016-09-29
    Sponsor Name:Biotest AG
    Full Title: An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as repl...
    Medical condition: Replacement therapy in patients with primary immunodeficiency disease (PID)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10064859 Primary immunodeficiency syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-001271-23 Sponsor Protocol Number: C0371004 Start Date*: 2019-02-20
    Sponsor Name:Pfizer Inc.
    Full Title: AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF CURRENT FACTOR IX (FIX) OR FACTOR VIII (FVIII) PROPHYLAXIS REPLA...
    Medical condition: Moderately Severe To Severe Adult Hemophilia B Participants (FIX:C≤2%) Who Are Negative For Neutralizing Antibodies (NAb) To Adeno-Associated Virus Vector (AAV) Spark100. Moderately severe to sever...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    20.0 100000004850 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adults Gender: Male
    Trial protocol: SE (Completed) GB (GB - no longer in EU/EEA) IE (Completed) DE (Completed) FR (Completed) ES (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-000447-26 Sponsor Protocol Number: SPK-8011-301 Start Date*: 2019-10-23
    Sponsor Name:Spark Therapeutics, Inc
    Full Title: An Open-label, Non-investigational Product, Multi-center, Lead-in Study to Evaluate Prospective Bleeding and Infusion Data of Current FVIII Replacement Therapy in Adult Males with Hemophilia A
    Medical condition: Hemophilia A
    Disease:
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: FI (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-001383-18 Sponsor Protocol Number: Start Date*: 2015-11-30
    Sponsor Name:University of Oxford
    Full Title: An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1)
    Medical condition: Choroideremia - a currently incurable retinal degeneration caused by null mutations in the CHM gene encoding REP1 protein located on the X chromosome. The condition causes blindness in males by the...
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004853 10008791 Choroideremia LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2013-005579-42 Sponsor Protocol Number: CT-AMT-060-01 Start Date*: 2015-03-20
    Sponsor Name:uniQure biopharma B.V.
    Full Title: A phase I/II, open-label, uncontrolled, single-dose, dose-ascending, multi-centre trial investigating an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) ...
    Medical condition: Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018939 Haemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: DE (Completed) DK (Completed) NL (Completed)
    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: 2021-000930-32 Sponsor Protocol Number: SJ2021005 Start Date*: 2021-04-27
    Sponsor Name:AZ Sint-Jan Brugge-Oostende AV
    Full Title: COVID-19: Documentation of humoral and cellular immune response of hemodialysis patients after vaccination against SARS-CoV-2
    Medical condition: hemodialysis patients hemodialysis patients with comorbidities
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-002012-27 Sponsor Protocol Number: 0708 Start Date*: 2019-12-09
    Sponsor Name:University of Leicester
    Full Title: A Single Centre, Open-label, Single-group Exploratory Study of the Safety and Activity of Sparsentan for the Treatment of Incident Patients with Immunoglobulin A Nephropathy
    Medical condition: Immunoglobulin A (IgA) nephropathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10038359 - Renal and urinary disorders 10021263 IgA nephropathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-006878-23 Sponsor Protocol Number: APHP200023 Start Date*: 2022-08-12
    Sponsor Name:Assistance Publique Hopitaux Paris - APHP
    Full Title: Efficacy of Ruxolitinib as first line treatment in primary haemophagocytic lymphohistiocytosis (HLH) in children: a Phase 2, multicentre, non-comparative study R-HLH
    Medical condition: • Patient with Haemophagocytic lymphohistiocytosis (HLH) or lymphohistiocytic syndrome
    Disease:
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    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: 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: 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: 2014-004525-41 Sponsor Protocol Number: 14VR4 Start Date*: 2015-04-10
    Sponsor Name:Versartis, Inc.
    Full Title: Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children w...
    Medical condition: Growth hormone deficiency
    Disease: Version SOC Term Classification Code Term Level
    19.0 10014698 - Endocrine disorders 10056438 Growth hormone deficiency PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: SE (Completed) BE (Completed) NL (Ongoing) PL (Completed)
    Trial results: View results
    EudraCT Number: 2014-003880-38 Sponsor Protocol Number: 270-201 Start Date*: 2015-06-01
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A
    Medical condition: Haemophilia A
    Disease:
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    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: 2017-004305-40 Sponsor Protocol Number: CSL222-3001 Start Date*: 2018-10-12
    Sponsor Name:CSL Behring LLC
    Full Title: Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene ...
    Medical condition: Hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10018939 Haemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: DK (Trial now transitioned) GB (GB - no longer in EU/EEA) IE (Trial now transitioned) NL (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) SE (Trial now transitioned) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-006114-14 Sponsor Protocol Number: 62326B1658 Start Date*: 2012-04-17
    Sponsor Name:Research and Development department
    Full Title: “Effects of testosterone on glycaemic control and other Cardiovascular Risk factors in Hypogonadal Men with uncontrolled Type 2 Diabetes: A randomized double – blinded placebo controlled add on tr...
    Medical condition: Hypogonadism Type 2 Diabetes Cardiovascular risk
    Disease:
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-002249-13 Sponsor Protocol Number: 3125001 Start Date*: 2019-02-04
    Sponsor Name:Orion Corporation Orion Pharma
    Full Title: SAFETY AND PHARMACOKINETICS OF ODM-209 IN PATIENTS WITH METASTATIC CASTRATION-RESISTANT PROSTATE CANCER OR ESTROGEN RECEPTOR-POSITIVE, HUMAN EPIDERMAL GROWTH FACTOR RECEPTOR 2-NEGATIVE ADVANCED BRE...
    Medical condition: METASTATIC CASTRATION-RESISTANT PROSTATE CANCER OR ESTROGEN RECEPTOR-POSITIVE, HUMAN EPIDERMAL GROWTH FACTOR RECEPTOR 2-NEGATIVE ADVANCED BREAST CANCER
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004864 10076506 Castration-resistant prostate cancer LLT
    21.1 100000004864 10072737 Advanced breast cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Completed) DK (Completed) ES (Prematurely Ended) IT (Completed)
    Trial results: View results
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    1  2  3  4  5  6  7  8  9  Next»
    Query did not match any studies.
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Sun Jun 15 08:11:57 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA