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Clinical trials for hemophilia OR haemophilia

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   44346   clinical trials with a EudraCT protocol, of which   7374   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.
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    283 result(s) found for: hemophilia OR haemophilia. Displaying page 8 of 15.
    EudraCT Number: 2015-002136-40 Sponsor Protocol Number: 261203 Start Date*: Information not available in EudraCT
    Sponsor Name:Baxalta Innovations GmbH
    Full Title: Phase 3, prospective, multi-center, open label study to investigate safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) < 6 y...
    Medical condition: Severe hemophilia A (FVIII <1%)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) BG (Prematurely Ended) ES (Completed) CZ (Prematurely Ended) AT (Completed) HU (Completed) DE (Completed) NL (Prematurely Ended) BE (Completed) NO (Completed) DK (Completed) FI (Completed) IT (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-004156-65 Sponsor Protocol Number: 3090A1-3305 Start Date*: 2015-04-06
    Sponsor Name:Wyeth Pharmaceutical Co, Ltd.
    Full Title: An Evaluation of the Safety and Efficacy of On-Demand Treatment with BeneFIX (Nonacog Alfa, Recombinant Factor IX) in Chinese Subjects with Hemophilia B
    Medical condition: Hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2020-004430-38 Sponsor Protocol Number: B1821059 Start Date*: 2020-10-16
    Sponsor Name:Pfizer, Inc.
    Full Title: A SINGLE COUNTRY, MULTICENTER, OPEN-LABEL AND NON-RANDOMIZED CLINICAL TRIAL WITH NONACOG ALFA PROPHYLAXIS AND TREATMENT OF BLEEDING EPISODES IN PREVIOUSLY TREATED PATIENTS WITH MODERATELY-SEVERE TO...
    Medical condition: Moderately severe to severe Hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018939 Haemophilia B (Factor IX) LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-020113-85 Sponsor Protocol Number: CSLCT-BIO-l0-67 Start Date*: 2011-09-20
    Sponsor Name:CSL Behring GmbH
    Full Title: A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to evaluate Plasma-Derived Antihaemophilic Factor/von Willebrand Factor Concentrate (Biostate®) for Immune To...
    Medical condition: Male paediatric subjects with Haemophilia A who develped high titre antibodies to human coagulation Factor VIII
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10018941 Haemophilia NOS LLT
    Population Age: Infants and toddlers, Children, Under 18, Adults, Elderly Gender: Male
    Trial protocol: DE (Prematurely Ended) FR (Completed) AT (Completed) GR (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-000504-11 Sponsor Protocol Number: NN7415-4616 Start Date*: 2022-04-05
    Sponsor Name:NOVO NORDISK. S.P.A.
    Full Title: Open-label study investigating efficacy, safety and pharmacokinetics of concizumab prophylaxis in children below 12 years with haemophilia A or B with or without inhibitors
    Medical condition: Haemophilia A with or without inhibitors Haemophilia B with our without inhibitors
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10053752 Hemophilia B with anti factor IX LLT
    20.1 100000004850 10053754 Hemophilia B without inhibitors LLT
    20.0 100000004850 10053753 Hemophilia A without inhibitors LLT
    20.0 100000004850 10053751 Hemophilia A with anti factor VIII LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IT (Trial now transitioned) NO (Trial now transitioned) GR (Trial now transitioned) SE (Trial now transitioned) LT (Trial now transitioned) EE (Completed) BG (Trial now transitioned) PL (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-003371-18 Sponsor Protocol Number: MAA-202 Start Date*: 2021-03-30
    Sponsor Name:Catalyst Biosciences, Inc.
    Full Title: Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog alfa (activated) in Treatment of Episodic Bleeding in Subjects with Inherited Bleeding Disorders
    Medical condition: Factor VII deficiency, Glanzmann thrombasthenia (GT) and Hemophilia A with inhibitors on emicizumab prophylaxis (HAwI-E)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10016079 Factor VII deficiency PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-004480-48 Sponsor Protocol Number: 20446 Start Date*: 2021-12-14
    Sponsor Name:Bayer AG
    Full Title: A prospective, lead-in study to collect bleeding episodes, Factor VIII (FVIII) infusions, and patient-reported outcomes in patients with hemophilia A
    Medical condition: Hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10053753 Hemophilia A without inhibitors LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: ES (Prematurely Ended) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-000662-29 Sponsor Protocol Number: 270-203 Start Date*: 2018-02-01
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1...
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2004-002648-88 Sponsor Protocol Number: 3090A-101795 Start Date*: 2005-11-17
    Sponsor Name:Wyeth Pharmaceuticals
    Full Title: In vitro evaluation of allergic reactions in hemophilia B subjects who have exhibited a systemic allergic response after exposure to BeneFIX (Nonacog Alfa; recombinant Factor IX)
    Medical condition: Hemophilia B
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-002554-23 Sponsor Protocol Number: GENA-05 Start Date*: 2013-02-19
    Sponsor Name:OCTAPHARMA AG
    Full Title: Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII in Previously Untreated Patients with Severe Haemophilia A
    Medical condition: Severe Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10018938 Haemophilia A (Factor VIII) LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: GB (Completed) PL (Completed) DE (Completed) ES (Completed) FR (Completed) PT (Completed) SI (Completed) HR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2012-004434-42 Sponsor Protocol Number: BAY94-9027/15912 Start Date*: 2013-04-15
    Sponsor Name:Bayer AG
    Full Title: A multi-center, phase III, non-controlled, open-label trial to evaluate the pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated chi...
    Medical condition: severe hemophilia A (<1% FVIII:C)
    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 10060612 Hemophilia A LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed) BE (Completed) IT (Completed) NL (Completed) LT (Completed) BG (Completed) Outside EU/EEA PL (Completed) AT (Completed) NO (Ongoing) ES (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2022-002333-34 Sponsor Protocol Number: CSL654_3004 Start Date*: 2024-05-01
    Sponsor Name:CSL Behring GmbH
    Full Title: A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Previously Treated Chinese Su...
    Medical condition: Hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2009-010172-21 Sponsor Protocol Number: ENJOIH01 Start Date*: 2009-08-27
    Sponsor Name:Elena Santagostino
    Full Title: An Investigator-sponsored study on rFVIIa prophylaxis in children with haemophilia A and inhibitors Estudio promovido por el investigador sobre profilaxis con rFVIIa en niños con hemofilia A e inh...
    Medical condition: Hemofilia A
    Disease: Version SOC Term Classification Code Term Level
    9.1 10056492 Haemophilia A with anti factor VIII LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Prematurely Ended) DE (Prematurely Ended) IT (Prematurely Ended) FR (Ongoing) PL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-003086-33 Sponsor Protocol Number: C0371002 Start Date*: 2020-01-28
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe he...
    Medical condition: severe to moderately severe hemophilia B <=2%
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned) DE (Trial now transitioned) GR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-004017-33 Sponsor Protocol Number: 3090X1-4405 Start Date*: 2008-04-17
    Sponsor Name:Wyeth Pharmaceuticals France
    Full Title: Reformulated BeneFIX efficacy and safety after conversion from a pdFIX.
    Medical condition: Hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    9.1 10016077 Factor IX deficiency LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2017-000852-24 Sponsor Protocol Number: 15/0552 Start Date*: 2017-08-23
    Sponsor Name:University College London (UCL)
    Full Title: A Phase I/II, Open label, Multicentre, Ascending Single Dose, Safety Study of a Novel Adeno-associated Viral Vector (FLT180a) in Patients With Haemophilia B
    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: GB (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-005489-37 Sponsor Protocol Number: CSL654_3003 Start Date*: 2014-07-11
    Sponsor Name:CSL Behring GmbH
    Full Title: A Phase 3b Open-label, Multicenter, Safety and Efficacy Extension Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects with Hemophilia B
    Medical condition: Hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Completed) IT (Completed) CZ (Completed) BG (Completed) ES (Completed) AT (Completed)
    Trial results: View results
    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: 2007-005100-41 Sponsor Protocol Number: IB1001-01 Start Date*: 2009-03-16
    Sponsor Name:Aptevo Europe Limited
    Full Title: Phase I/II/III Pharmacokinetic and Outcome Study of Recombinant Factor IX Product, IB1001, in Subjects with Haemophilia B
    Medical condition: Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed) FR (Ongoing) PL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-005781-39 Sponsor Protocol Number: 091501 Start Date*: 2019-03-25
    Sponsor Name:Baxalta Innovations GmbH
    Full Title: A Phase 3b/4, Prospective, Multicenter, Open-label, Randomized, Crossover Study of Tolerability and Safety of FEIBA Reconstituted in Regular or 50% Reduced Volume and of Faster Infusion Rates in Pa...
    Medical condition: Hemophilia A or B with inhibitors
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: HR (Completed) RO (Completed)
    Trial results: View results
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