C5 Inhibitor ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
62発作性夜間ヘモグロビン尿症16

62. 発作性夜間ヘモグロビン尿症


臨床試験数 : 271 薬物数 : 163 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 106
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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Secondary_
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2019-003829-18-GR
(EUCTR)
08/03/202214/01/2022Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA)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 Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan
Product Code: ALXN2040
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan
Product Code: ALXN2040
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Czechia;Taiwan;Greece;Thailand;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Canada;Argentina;Poland;Brazil;Malaysia;Netherlands;Germany;Japan;Korea, Republic of
2NCT05116774
(ClinicalTrials.gov)
December 6, 20212/11/2021BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor TherapyA Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor TherapyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: BCX9930;Drug: Eculizumab;Drug: RavulizumabBioCryst PharmaceuticalsNULLRecruiting18 YearsN/AAll81Phase 2United States;France;Hungary;Spain;Taiwan;United Kingdom
3EUCTR2020-004438-39-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
4EUCTR2020-004438-39-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
5EUCTR2019-003830-17-IT
(EUCTR)
13/10/202123/06/2020Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 InhibitorA 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) Patients as Monotherapy and with an Approved C5 Inhibitor as Background Therapy 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: ALXN2050
Product Code: ACH-0145228
INN or Proposed INN: N/A
Other descriptive name: ACH-0145228
Product Name: ALXN2050
Product Code: ACH-0145228
INN or Proposed INN: N/A
Other descriptive name: ACH-0145228
Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
26Phase 2United States;Spain;United Kingdom;New Zealand;Italy;Korea, Republic of
6EUCTR2020-004438-39-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
7EUCTR2020-004438-39-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
8JPRN-jRCT2021210014
28/06/202128/06/2021Pivotal study of danicopan as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Evident EVHA 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 Extravascular Hemolysis (EVH) Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria;D006457
Additional Danicopan (ALXN2040) given to patients diagnosed with paroxysmal nocturnal hemoglobinuria and receiving complement C5 inhibitorsKamata TakeoNULLRecruiting>= 18age oldNot applicableBoth84Phase 3America;Japan
9EUCTR2019-003829-18-NL
(EUCTR)
21/05/202107/10/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)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 Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Finland;Thailand;Spain;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
10EUCTR2019-003829-18-PL
(EUCTR)
08/04/202104/11/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)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 Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Spain;Thailand;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Germany;Netherlands;Japan;Korea, Republic of
11EUCTR2019-003829-18-DE
(EUCTR)
03/02/202123/01/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA)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 Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan
Product Code: ALXN2040
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan
Product Code: ALXN2040
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Spain;Thailand;United Kingdom;Italy;France;Canada;Brazil;Malaysia;Poland;Germany;Netherlands;Japan;Korea, Republic of
12NCT04469465
(ClinicalTrials.gov)
December 16, 20209/7/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)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 Extravascular Hemolysis (EVH)Paroxysmal Nocturnal HemoglobinuriaDrug: Danicopan;Drug: Placebo;Drug: C5 InhibitorAlexion PharmaceuticalsNULLRecruiting18 YearsN/AAll84Phase 3United States;Brazil;Canada;France;Germany;Italy;Japan;Korea, Republic of;Malaysia;Netherlands;Poland;Spain;Thailand;United Kingdom
13EUCTR2019-003829-18-FR
(EUCTR)
04/11/202022/01/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)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 Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Finland;Spain;Thailand;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
14EUCTR2019-003829-18-IT
(EUCTR)
03/11/202024/05/2021Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)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 Extravascular Hemolysis (EVH) - . Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan (ALXN2040)
Product Code: [ACH-0144471]
Product Name: Danicopan (ALXN2040)
Product Code: [ACH-0144471]
ALEXION PHARMACEUTICALS INCORPORATEDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Finland;Spain;Thailand;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
15EUCTR2019-003829-18-GB
(EUCTR)
18/09/202005/02/2020Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)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 Extravascular Hemolysis (EVH) Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
16EUCTR2019-003830-17-GB
(EUCTR)
16/09/202016/06/2020Study of the Oral Factor D (fD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with background use of an Approved C5 InhibitorA 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) Patients as Monotherapy and with an Approved C5 Inhibitor as Background Therapy 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: ALXN2050
Product Code: ACH-0145228
INN or Proposed INN: N/A
Other descriptive name: ACH-0145228
Product Name: ALXN2050
Product Code: ACH-0145228
INN or Proposed INN: N/A
Other descriptive name: ACH-0145228
Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
26Phase 2United States;New Zealand;Italy;United Kingdom;Korea, Republic of