Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Hemoglobinuria

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   44335   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 ).  
     
    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
     
    58 result(s) found for: Hemoglobinuria. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2005-000043-28 Sponsor Protocol Number: E05-001 Start Date*: 2005-04-19
    Sponsor Name:ALEXION PHARMACEUTICALS INCORPORATED
    Full Title: A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SH...
    Medical condition: paroxysmal nocturnal hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    7.0 10034042 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IE (Completed) SE (Completed) DE (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-003522-16 Sponsor Protocol Number: RA101495-01.201 Start Date*: 2017-06-20
    Sponsor Name:Ra Pharmaceuticals, Inc.
    Full Title: A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLO...
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000012950 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Completed) GB (Completed) HU (Completed) DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-010402-11 Sponsor Protocol Number: M07-005 Start Date*: 2012-04-30
    Sponsor Name:ALEXION PHARMACEUTICALS, INC.
    Full Title: AN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
    Medical condition: PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
    Disease: Version SOC Term Classification Code Term Level
    14.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-003523-34 Sponsor Protocol Number: RA101495-01.202 Start Date*: 2017-04-06
    Sponsor Name:Ra Pharmaceuticals, Inc.
    Full Title: A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Completed) HU (Completed) DK (Prematurely Ended) GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2004-000646-20 Sponsor Protocol Number: C04-001 Start Date*: 2004-09-21
    Sponsor Name:ALEXION PHARMACEUTICALS INCORPORATED
    Full Title: A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal...
    Medical condition: paroxysmal nocturnal hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    7.0 10034042 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) IE (Completed) GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2004-002795-42 Sponsor Protocol Number: C04-002 Start Date*: 2005-02-16
    Sponsor Name:ALEXION PHARMACEUTICALS INCORPORATED
    Full Title: Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study
    Medical condition: paroxysmal nocturnal hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    7.0 10034042 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) IE (Completed) DE (Completed) GB (Completed) IT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2019-003829-18 Sponsor Protocol Number: ALXN2040-PNH-301 Start Date*: 2020-11-04
    Sponsor Name:Alexion Pharmaceuticals Inc.
    Full Title: A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascula...
    Medical condition: Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) DE (Completed) GB (GB - no longer in EU/EEA) NL (Completed) PL (Completed) IT (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2017-002820-26 Sponsor Protocol Number: ALXN1210-PNH-304 Start Date*: 2017-11-24
    Sponsor Name:Alexion Pharmaceuticals Incorporated
    Full Title: A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Completed) FR (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2021-004253-22 Sponsor Protocol Number: ALXN2040-PNH-303 Start Date*: 2022-07-04
    Sponsor Name:Alexion Pharmaceuticals Inc.
    Full Title: A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglo...
    Medical condition: Patients with Paroxysmal Nocturnal Hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Trial now transitioned) IT (Trial now transitioned) GR (Trial now transitioned) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-006776-17 Sponsor Protocol Number: BCX9930-205 Start Date*: 2023-01-10
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst sponsored Study
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: HU (Completed) FR (Completed) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004385-19 Sponsor Protocol Number: CLNP023C12001B Start Date*: 2021-07-15
    Sponsor Name:Novartis Pharma AG
    Full Title: An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) w...
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: LT (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) CZ (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-002026-36 Sponsor Protocol Number: ALXN1210-PNH-302 Start Date*: 2017-05-30
    Sponsor Name:Alexion Pharmaceuticals Incorporated
    Full Title: A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) ES (Ongoing) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-002025-11 Sponsor Protocol Number: ALXN1210-PNH-301 Start Date*: 2016-12-02
    Sponsor Name:Alexion Pharmaceuticals Incorporated
    Full Title: A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) SE (Completed) BE (Completed) CZ (Completed) DK (Prematurely Ended) PT (Prematurely Ended) FI (Prematurely Ended) ES (Ongoing) NL (Completed) AT (Completed) EE (Completed) PL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-000501-93 Sponsor Protocol Number: BCX9930-201 Start Date*: 2020-09-29
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) AT (Completed) DK (Prematurely Ended) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-002943-40 Sponsor Protocol Number: ALN-CC5-003 Start Date*: 2016-11-21
    Sponsor Name:Alnylam Pharmaceuticals Inc
    Full Title: A Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab
    Medical condition: Paroxysmal nocturnal hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-002370-39 Sponsor Protocol Number: ALXN1210-PNH-303 Start Date*: 2019-07-02
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxys...
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) FR (Completed) BE (Completed) GB (GB - no longer in EU/EEA) NL (Ongoing) SE (Completed) FI (Completed) CZ (Completed) ES (Ongoing) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2017-001418-27 Sponsor Protocol Number: 20150168 Start Date*: 2019-01-29
    Sponsor Name:Amgen Inc.
    Full Title: A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) NL (Completed) GB (GB - no longer in EU/EEA) CZ (Completed) NO (Completed) SI (Completed) IE (Completed) FI (Completed) SE (Completed) PT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-000130-20 Sponsor Protocol Number: R3918-PNH-1868 Start Date*: 2020-04-01
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: An open-label extension study to evaluate the long-term safety, tolerability, and efficacy of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria
    Medical condition: Paroxysmal nocturnal hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Ongoing) CZ (Completed) GB (GB - no longer in EU/EEA) HU (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-002734-20 Sponsor Protocol Number: R3918-PNH-1852 Start Date*: 2020-08-07
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: An Open Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naive or Have Not Recently Re...
    Medical condition: Paroxysmal nocturnal hemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Completed) GB (GB - no longer in EU/EEA) HU (Completed) NL (Ongoing) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-004931-10 Sponsor Protocol Number: R3918-PNH-2050 Start Date*: 2022-12-27
    Sponsor Name:REGENERON PHARMACEUTICALS, INC.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria
    Medical condition: Paroxysmal nocturnal hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) HU (Trial now transitioned) ES (Ongoing) RO (Trial now transitioned)
    Trial results: (No results available)
    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  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-Tue May 13 17:01:33 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA