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Clinical trials for PNH

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   43690   clinical trials with a EudraCT protocol, of which   7245   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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    58 result(s) found for: PNH. Displaying page 1 of 3.
    1  2  3  Next»
    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 (Ongoing)
    Trial results: (No results available)
    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: 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: 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: 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: 2016-002025-11 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 (Ongoing) FI (Prematurely Ended) ES (Ongoing) NL (Completed) AT (Completed) EE (Completed) PL (Completed) IT (Completed)
    Trial results: View results
    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 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-003830-17 Sponsor Protocol Number: ACH228-110 Start Date*: 2020-09-16
    Sponsor Name:Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc.
    Full Title: A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient...
    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: GB (GB - no longer in EU/EEA) IT (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-002128-10 Sponsor Protocol Number: BP39144 Start Date*: 2016-09-14
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)
    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 Gender: Male
    Trial protocol: NL (Completed) DE (Restarted) HU (Ongoing) FR (Temporarily Halted) IT (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2016-002652-25 Sponsor Protocol Number: ACH471-100 Start Date*: 2018-02-02
    Sponsor Name:Achillion Pharmaceuticals, Inc.
    Full Title: A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Medical condition: Untreated Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-003778-34 Sponsor Protocol Number: VIP578 Start Date*: 2016-01-13
    Sponsor Name:Volution Immuno Pharmaceuticals (UK) Ltd
    Full Title: Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms
    Medical condition: Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (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: 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: 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: 2020-004403-14 Sponsor Protocol Number: BCX9930-203 Start Date*: 2021-11-11
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Noct...
    Medical condition: 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 (Prematurely Ended) LT (Completed) CZ (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-001106-23 Sponsor Protocol Number: APL2-307 Start Date*: 2019-08-09
    Sponsor Name:Apellis Pharmaceuticals, Inc.
    Full Title: An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
    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: BG (Ongoing) DE (Completed) GB (GB - no longer in EU/EEA) FR (Ongoing) ES (Ongoing) BE (Completed)
    Trial results: (No results available)
    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 (Ongoing) PL (Ongoing) GR (Ongoing) HU (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-002067-33 Sponsor Protocol Number: AK579 Start Date*: 2016-07-18
    Sponsor Name:Akari Therapeutics Plc
    Full Title: COBALT: Coversin Global Study: An Open-Label, Safety and Efficacy Trial in PNH Patients
    Medical condition: Paroxysmal nocturnal haemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (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 (Ongoing) IT (Ongoing)
    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
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