62. Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details


Clinical trials : 271 Drugs : 163 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 106

  
16 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1EUCTR2020-004438-39-IT
(EUCTR)
10/11/202127/01/2022BCX9930 treatment in PNH patients with an inadequate response to C5 inhibitorsA 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 in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: ULTOMIRIS - 300 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO (VETRO) 3 ML (100 MG / ML) - 1 FLACONCINO
Product Name: ravulizumab
Product Code: [ravulizumab]
INN or Proposed INN: Ravulizumab
Other descriptive name: Ultomiris
Trade Name: SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML)
Product Name: Eculizumab
Product Code: [Eculizumab]
INN or Proposed INN: ECULIZUMAB
Other descriptive name: Soliris
Product Name: BCX9930
Product Code: [BCX9930]
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
BIOCRYST PHARMACEUTICALS INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
81Phase 2United States;Taiwan;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of
2EUCTR2020-004438-39-ES
(EUCTR)
02/11/202123/07/2021BCX9930 treatment in patients with an inadequate response to C5 inhibitorsA 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 in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Trade Name: Solaris
Product Name: eculizumab
Product Code: eculizumab
INN or Proposed INN: Eculizumab
Other descriptive name: Soliris
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ravulizumab
INN or Proposed INN: RAVULIZUMAB
BioCryst Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
81Phase 2United States;Taiwan;Spain;Turkey;Colombia;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of
3EUCTR2020-004438-39-HU
(EUCTR)
01/09/202102/07/2021BCX9930 treatment in patients with an inadequate response to C5 inhibitorsA 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 in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Trade Name: Solaris
Product Name: eculizumab
Product Code: eculizumab
INN or Proposed INN: Eculizumab
Other descriptive name: Soliris
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ravulizumab
INN or Proposed INN: RAVULIZUMAB
BioCryst Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
81Phase 2United States;Taiwan;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of
4EUCTR2020-004438-39-FR
(EUCTR)
26/08/202116/06/2021BCX9930 treatment in patients with an inadequate response to C5 inhibitorsA 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 in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Trade Name: Solaris
Product Name: eculizumab
Product Code: eculizumab
INN or Proposed INN: Eculizumab
Other descriptive name: Soliris
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ravulizumab
INN or Proposed INN: RAVULIZUMAB
BioCryst Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
81Phase 2United States;Taiwan;Turkey;Colombia;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of
5EUCTR2019-004665-40-CZ
(EUCTR)
31/03/202103/11/2020Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatmentA 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 treatment with an intravenous anti-C5 antibody Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
91Phase 3United States;Czechia;Taiwan;Spain;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of
6EUCTR2019-004665-40-DE
(EUCTR)
29/01/202122/09/2020Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatmentA 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 treatment with an intravenous anti-C5 antibody Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: iptacopan
Product Code: LNP023
INN or Proposed INN: iptacopan
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: iptacopan
Product Code: LNP023
INN or Proposed INN: iptacopan
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris 300mg/30ml
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris 300mg/30ml
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
91Phase 3United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of
7EUCTR2019-004665-40-NL
(EUCTR)
20/11/202009/11/2020Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatmentA 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 treatment with an intravenous anti-C5 antibody Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: iptacopan
Product Code: LNP023
INN or Proposed INN: iptacopan
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: iptacopan
Product Code: LNP023
INN or Proposed INN: iptacopan
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris 300mg/30ml
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris 300mg/30ml
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
91Phase 3United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of
8EUCTR2019-004665-40-HU
(EUCTR)
17/11/202023/09/2020Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatmentA 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 treatment with an intravenous anti-C5 antibody Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Novartis Pharma AGNULLNot RecruitingFemale: yes
Male: yes
91Phase 3United States;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of
9EUCTR2019-003440-74-GB
(EUCTR)
14/05/202017/03/2020Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose EculizumabPhase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab - ALXN1210-PNH-401 Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: ULTOMIRIS
Product Name: ULTOMIRIS
Product Code: ALXN1210
INN or Proposed INN: RAVULIZUMAB
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 4United Kingdom
10EUCTR2019-001453-10-ES
(EUCTR)
16/09/201909/08/2019A 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 with Paroxysmal Nocturnal HemoglobinuriaA 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 WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Soliris 300
Product Name: ECULIZUMAB
INN or Proposed INN: ECULIZUMAB
Trade Name: ULTOMIRIS
Product Name: Ultomiris
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: Ultomiris
Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
200Phase 4United States;Taiwan;Greece;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Romania;Netherlands;Germany;Japan;Sweden;Korea, Republic of
11EUCTR2017-002370-39-SE
(EUCTR)
27/03/201930/11/2018This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With EculizumabA Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Trade Name: ULTOMIRIS
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Alexion Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
105Phase 3United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of
12EUCTR2017-002820-26-NL
(EUCTR)
21/03/201810/01/2018A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Ultomiris
Product Name: ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Ravulizumab
Other descriptive name: ALXN1210
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
13Phase 3France;United States;Russian Federation;Norway;Netherlands;United Kingdom
13EUCTR2017-002820-26-GB
(EUCTR)
24/11/201726/10/2017A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Ravulizumab
Other descriptive name: ALXN1210
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
13Phase 3United States;France;Russian Federation;Norway;Netherlands;United Kingdom
14EUCTR2016-002026-36-NL
(EUCTR)
27/07/201727/03/2017ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With EculizumabA Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Ravulizumab
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
197Phase 3United States;Spain;Germany;Netherlands;United Kingdom
15EUCTR2016-002025-11-BE
(EUCTR)
14/03/201717/10/2016Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Ultomiris
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
301Phase 3Portugal;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Czechia;Finland;Korea, Democratic People's Republic of;Turkey;United States Minor Outlying Islands;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden
16EUCTR2016-002025-11-EE
(EUCTR)
20/02/201723/01/2017Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Ultomiris
Product Name: Ultomiris
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Trade Name: Soliris
Product Name: Soliris
INN or Proposed INN: ECULIZUMAB
Alexion Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
301Phase 3Portugal;United States;Taiwan;Estonia;Thailand;Spain;Russian Federation;Colombia;Italy;France;Malaysia;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Finland;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Singapore;Germany;Japan;Sweden