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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   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 ).  
     
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    58 result(s) found for: PNH. Displaying page 3 of 3.
    « Previous 1  2  3 
    EudraCT Number: 2020-001350-21 Sponsor Protocol Number: APL2-PNH-209 Start Date*: 2020-12-08
    Sponsor Name:Apellis Pharmaceuticals, Inc.
    Full Title: An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric 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: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: CZ (Prematurely Ended) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004665-40 Sponsor Protocol Number: CLNP023C12302 Start Date*: 2020-11-03
    Sponsor Name:Novartis Pharma AG
    Full Title: A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite tre...
    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: FR (Completed) DE (Completed) HU (Completed) CZ (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-003526-16 Sponsor Protocol Number: ACH471-101 Start Date*: 2018-07-20
    Sponsor Name:Alexion Pharmaceuticals Inc.
    Full Title: A Phase 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy
    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: GB (GB - no longer in EU/EEA) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-002761-33 Sponsor Protocol Number: R3918-PNH-2022 Start Date*: 2022-05-02
    Sponsor Name:REGENERON PHARMACEUTICALS, INC.
    Full Title: A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGL...
    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 (Prematurely Ended) DE (Completed) ES (Prematurely Ended) FR (Prematurely Ended) PL (Completed) GR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-003172-41 Sponsor Protocol Number: CLNP023C12301 Start Date*: 2021-06-03
    Sponsor Name:Novartis Pharma AG
    Full Title: A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy
    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: FR (Completed) CZ (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2018-004220-11 Sponsor Protocol Number: APL2-308 Start Date*: 2019-07-23
    Sponsor Name:Apellis Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in 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: PL (Completed)
    Trial results: View results
    EudraCT Number: 2019-003440-74 Sponsor Protocol Number: ALXN1210-PNH-401 Start Date*: 2020-05-14
    Sponsor Name:Alexion Pharmaceuticals Inc.
    Full Title: Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose 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 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2020-005005-17 Sponsor Protocol Number: R3918-PNH-2092 Start Date*: 2021-05-07
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglo...
    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 (Completed)
    Trial results: View results
    EudraCT Number: 2019-001453-10 Sponsor Protocol Number: BA41510 Start Date*: 2019-09-16
    Sponsor Name:Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche Ltd
    Full Title: A MULTICENTER, LOW-INTERVENTIONAL STUDY DOCUMENTING THE EFFICACY, HEALTH-RELATED QUALITY OF LIFE, AND SAFETY OF STANDARD-OF-CARE TREATMENT WITH ECULIZUMAB OR TREATMENT WITH RAVULIZUMAB IN PATIENTS ...
    Medical condition: Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004857 10055629 Paroxysmal nocturnal hemoglobinuria LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-004129-18 Sponsor Protocol Number: AK581 Start Date*: 2017-03-17
    Sponsor Name:Akari Therapeutics Plc
    Full Title: CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study
    Medical condition: Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10005329 - Blood and lymphatic system disorders 10018932 Haemolytic uraemic syndrome PT
    21.1 10038359 - Renal and urinary disorders 10034042 Paroxysmal nocturnal haemoglobinuria PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) PL (Prematurely Ended) NL (Prematurely Ended) LT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-000174-19 Sponsor Protocol Number: 9345 Start Date*: 2015-01-27
    Sponsor Name:Universitätsklinikum Ulm
    Full Title: Efficacy and Safety of Eltrombopag in Patients with Acquired Moderate Aplastic Anemia (EMAA) who are treated with Ciclosporin A Prospective Randomized Multicenter Study comparing Thrombopoetin-Re...
    Medical condition: Moderate Aplastic Anemia (MAA)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-004364-35 Sponsor Protocol Number: CETB115AUS01T Start Date*: 2018-07-25
    Sponsor Name:National Heart, Lung, and Blood
    Full Title: Eltrombopag added to standard immunosuppression in treatment-naïve severe aplastic anemia.
    Medical condition: Severe aplastic anemia (SAA)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2014-000363-40 Sponsor Protocol Number: 8409032 Start Date*: 2016-01-07
    Sponsor Name:European Society for Blood and Marrow Transplantation
    Full Title: A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients.
    Medical condition: Idiopathic (Severe) Aplastic Anemia, a bone marrow failure syndrome which results in the normal hematopoietic tissue completely missing from the bone marrow, accounting for the subsequent pancytope...
    Disease: Version SOC Term Classification Code Term Level
    19.0 10005329 - Blood and lymphatic system disorders 10002967 Aplastic anaemia PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-002814-29 Sponsor Protocol Number: CETB115E2403 Start Date*: 2017-03-21
    Sponsor Name:Novartis Farmacéutica, S.A
    Full Title: SOAR Trial, A two-part study: Interventional phase II single-arm trial to assess efficacy and safety of Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired...
    Medical condition: First-line severe aplastic anaemia
    Disease: Version SOC Term Classification Code Term Level
    19.0 10005329 - Blood and lymphatic system disorders 10002967 Aplastic anaemia PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Restarted) NL (Completed) HU (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-001151-22 Sponsor Protocol Number: Alesaa Start Date*: 2007-06-06
    Sponsor Name:UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
    Full Title: ALEMTUZUMAB AND LOW-DOSE CYCLOSPORINE-A AS ALTERNATIVE IMMUNOSUPPRESSIVE TREATMENT FOR SEVERE APLASTIC ANEMIA (SAA) AND SINGLE-LINEAGE APLASTIC PATIENTS.
    Medical condition: Aplastic anemia and single-lineage bone marrow failure
    Disease: Version SOC Term Classification Code Term Level
    13.1 10005329 - Blood and lymphatic system disorders 10002965 Aplasia pure red cell PT
    13.1 10005329 - Blood and lymphatic system disorders 10047350 Very few granulocyte precursors LLT
    13.1 10005329 - Blood and lymphatic system disorders 10002967 Aplastic anaemia PT
    13.1 10005329 - Blood and lymphatic system disorders 10001507 Agranulocytosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-003166-91 Sponsor Protocol Number: CETB115E2201 Start Date*: 2019-05-22
    Sponsor Name:Novartis Pharma AG
    Full Title: A phase II, open-label, non-controlled, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, rela...
    Medical condition: Refractory, relapsed or treatment naïve severe aplastic anemia (SAA) or recurrent aplastic anemia (AA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10005329 - Blood and lymphatic system disorders 10002967 Aplastic anaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) PT (Completed) NL (Ongoing)
    Trial results: View results
    EudraCT Number: 2011-005562-38 Sponsor Protocol Number: M2011-238 Start Date*: 2012-11-13
    Sponsor Name:Miltenyi Biotec GmbH
    Full Title: A multi-center phase I/II safety and feasibility study using CliniMACS TCRα/β and CD19 depleted stem cell grafts from haploidentical donors for haematopoietic progenitor cell transplantation in chi...
    Medical condition: Hematological and non-hematological malignancies, and non-malignant diseases, requiring allogeneic blood stem cell transplantation without available HLA-identical donor.
    Disease: Version SOC Term Classification Code Term Level
    18.1 10042613 - Surgical and medical procedures 10063581 Stem cell transplant PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2010-019501-41 Sponsor Protocol Number: MPD-RC112 Start Date*: 2012-02-15
    Sponsor Name:CONSORZIO MARIO NEGRI SUD
    Full Title: Randomized Trial of Pegylated Interferon Alfa-2a versus Hydroxyurea Therapy in the Treatment of High Risk Polycythemia Vera and High Risk Essential Thrombocythemia
    Medical condition: High Risk Polycythemia Vera or High Risk Essential Thrombocythemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10036057 Polycythaemia vera PT
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10015493 Essential thrombocythaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing) SE (Ongoing) GB (Completed) DE (Prematurely Ended)
    Trial results: (No results available)
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    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.

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