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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   44393   clinical trials with a EudraCT protocol, of which   7405   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 2 of 3.
    « Previous 1  2  3  Next»
    EudraCT Number: 2017-005140-16 Sponsor Protocol Number: APL2-202 Start Date*: 2018-07-18
    Sponsor Name:Apellis Pharmaceuticals
    Full Title: A Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in 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: BG (Completed) GR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-004438-39 Sponsor Protocol Number: BCX9930-202 Start Date*: 2021-08-26
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria i...
    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: FR (Prematurely Ended) HU (Prematurely Ended) ES (Prematurely Ended) SK (Completed) NL (Prematurely Ended) IT (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: 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: 2017-000665-79 Sponsor Protocol Number: ACH471-103 Start Date*: 2018-07-23
    Sponsor Name:Achillion Pharmaceuticals, Inc.
    Full Title: An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100
    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 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Completed)
    Trial results: View results
    EudraCT Number: 2017-004268-36 Sponsor Protocol Number: APL2-302 Start Date*: 2018-10-01
    Sponsor Name:Apellis Pharmaceuticals, Inc.
    Full Title: A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Medical condition: PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic epis...
    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 (Completed) BE (Completed) ES (Completed) NL (Completed) FR (Completed) GB (Completed) 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: 2018-002857-31 Sponsor Protocol Number: SB12-3003 Start Date*: 2019-09-09
    Sponsor Name:Samsung Bioepis Co., Ltd.
    Full Title: A Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity between SB12 (proposed eculizumab biosimilar) and Soliris® in Subjects ...
    Medical condition: Paroxysmal Nocturnal Haemoglobinuria
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: RO (Ongoing)
    Trial results: View results
    EudraCT Number: 2014-005338-74 Sponsor Protocol Number: CLFG316X2201 Start Date*: 2015-06-17
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with 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: CZ (Completed) LT (Completed)
    Trial results: View results
    EudraCT Number: 2020-000597-26 Sponsor Protocol Number: BO42161 Start Date*: Information not available in EudraCT
    Sponsor Name:F.Hoffmann-La Roche Ltd
    Full Title: A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF CROVALIMAB VERSUS ECULIZUMAB IN PATIENTS WITH PA...
    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: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Trial now transitioned) DE (Trial now transitioned) FR (Trial now transitioned) PL (Trial now transitioned) BE (Trial now transitioned) GR (Trial now transitioned) HU (Completed) CZ (Trial now transitioned) PT (Trial now transitioned) IE (Trial now transitioned) NL (Trial now transitioned) EE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    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: 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: 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: 2015-002674-20 Sponsor Protocol Number: ALXN1210-PNH-201 Start Date*: 2015-12-04
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to...
    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 Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) SE (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2019-004931-21 Sponsor Protocol Number: BO42162 Start Date*: 2020-10-26
    Sponsor Name:F. Hoffman-La Roche Ltd.
    Full Title: A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTUR...
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: SE (Trial now transitioned) DE (Trial now transitioned) HU (Completed) FR (Trial now transitioned) PL (Trial now transitioned) GR (Completed) PT (Trial now transitioned) BE (Completed) IE (Prematurely Ended) NL (Trial now transitioned) EE (Completed) LT (Trial now transitioned) IT (Prematurely Ended) RO (Trial now transitioned)
    Trial results: (No results available)
    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: 2017-003847-39 Sponsor Protocol Number: AK580 Start Date*: 2018-03-12
    Sponsor Name:Akari Therapeutics Plc.
    Full Title: CAPSTONE: Phase III Confirmatory Assessment Protocol: rVA576 Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH)
    Medical condition: Paroxysmal Nocturnal Haemoglobinuria (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 (Completed)
    Trial results: View results
    EudraCT Number: 2017-000888-33 Sponsor Protocol Number: CLNP023X2201 Start Date*: 2018-02-05
    Sponsor Name:Novartis Pharma AG
    Full Title: An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with p...
    Medical condition: Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10005329 - Blood and lymphatic system disorders 10018911 Haemolyses and related conditions HLGT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-003956-19 Sponsor Protocol Number: RA101495-01.301 Start Date*: 2019-02-13
    Sponsor Name:Ra Pharmaceuticals, Inc.
    Full Title: Phase 3, Multicenter, Open-Label, Single-Arm Study to Confirm the Safety and Efficacy of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria
    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: FI (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-004486-40 Sponsor Protocol Number: R3918-PNH-2021 Start Date*: 2022-04-04
    Sponsor Name:REGENERON PHARMACEUTICALS, INC.
    Full Title: A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO A...
    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) ES (Ongoing) PL (Trial now transitioned) GR (Trial now transitioned) HU (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
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