62. 発作性夜間ヘモグロビン尿症 Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details


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

  
16 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2020-004385-19-NL
(EUCTR)
11/01/202217/11/2021Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal HemoglobinuriaAn 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) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. 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
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
165Phase 3United States;Taiwan;Spain;Lithuania;Italy;France;Brazil;Malaysia;Singapore;Netherlands;China;Japan;Korea, Republic of
2JPRN-jRCT2021210059
09/12/202117/12/2021Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal HemoglobinuriaAn 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) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. Paroxysmal Nocturnal HemoglobinuriaTaken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oralSuzuki KazuyukiNULLRecruiting>= 18age oldNot applicableBoth19Phase 3Italy;Republic of Korea;Taiwan;Japan
3EUCTR2020-004385-19-FR
(EUCTR)
31/08/202115/07/2021Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal HemoglobinuriaAn 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) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. 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
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
165Phase 3United States;France;Taiwan;Spain;Malaysia;Brazil;Singapore;Lithuania;Italy;Japan;China;Korea, Republic of
4EUCTR2020-003172-41-CZ
(EUCTR)
12/08/202110/06/2021Study of Efficacy and Safety of Twice Daily Oral iptacopan in Adult PNH Patients who are naïve to complement inhibitor therapyA 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 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
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3France;United States;Czechia;Czech Republic;Malaysia;Singapore;Germany;United Kingdom;Italy;Japan;China;Korea, Republic of
5EUCTR2020-004385-19-ES
(EUCTR)
30/07/202123/06/2021Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal HemoglobinuriaAn 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) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. 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
Novartis Farmacéutica, S.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
165Phase 3United States;Taiwan;Malaysia;Brazil;Spain;Singapore;Japan;China;Korea, Republic of
6NCT04747613
(ClinicalTrials.gov)
July 27, 20215/2/2021Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal HemoglobinuriaAn 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) Who Have Completed PNH Phase 2 and Phase3 Studies With IptacopanParoxysmal Nocturnal HemoglobinuriaDrug: IptacopanNovartis PharmaceuticalsNULLRecruiting18 YearsN/AAll167Phase 3France;Italy;Japan;Korea, Republic of;Malaysia;Singapore;Taiwan
7EUCTR2020-003172-41-IT
(EUCTR)
20/07/202117/08/2021Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients who are naïve to complement inhibitor therapy.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. - - 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: HIBERIX - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE PER USO INTRAMUSCOLARE 1 FLACONCINO DI POLVERE + 1 SIRINGA PRERIEMPITA DI SOLVENTE DA 0.5 ML
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO HAEMOPHILUS INFLUENZAE B CONIUGATO CON TOSSOIDETETANICO
Trade Name: MENVEO – Polvere e soluzione per soluzione iniettabile
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135)
Trade Name: PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO PNEUMOCOCCICO
Trade Name: BEXSERO – Sospensione iniettabile – Siringa preriempita
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO CONTRO IL MEMINGOCOCCO DI GRUPPO B
Trade Name: ACT-HIB Polvere e solvente per soluzione iniettabile in siringa preriempita 0,5 ml
Product Name: -
Product Code: [-]
INN or Proposed INN: HAEMOPHILUS INFLUENZAE
NOVARTIS PHARMA AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3France;United States;Czechia;Malaysia;Singapore;Germany;United Kingdom;Japan;China;Italy;Korea, Republic of
8NCT04820530
(ClinicalTrials.gov)
July 19, 202125/3/2021Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyA 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 TherapyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: Iptacopan (LNP023)Novartis PharmaceuticalsNULLRecruiting18 YearsN/AAll40Phase 3China;Czechia;France;Germany;Italy;Japan;Korea, Republic of;Malaysia;Singapore;United Kingdom
9EUCTR2020-004385-19-LT
(EUCTR)
15/07/202120/04/2021Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal HemoglobinuriaAn 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) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. 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
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
165Phase 3United States;Taiwan;Spain;Lithuania;Italy;France;Brazil;Malaysia;Singapore;Netherlands;China;Japan;Korea, Republic of
10EUCTR2020-004385-19-IT
(EUCTR)
15/06/202108/06/2021Long-term safety and tolerability of iptacopan in patients with Paroxysmal Nocturnal HemoglobinuriaAn 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) who have completed PNH Phase 2 and Phase 3 studies with iptacopan. - - 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]
Trade Name: PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO PNEUMOCOCCICO
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: ACTHIB - POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE 1 FLACONCINO POLVERE+1 SIRINGA PRERIEMPITA SOLVENTE 0.5 ML
Product Name: -
Product Code: [-]
INN or Proposed INN: HAEMOPHILUS INFLUENZAE
Trade Name: MENVEO - POLV.E SOLUZ.PER SOLUZ. INIETT.-USO INTRAMUSCOLARE-COMP. CONIUG.LIOFILIZZ.MENA:FLAC.(VETRO) COMP. GONIUG.LIQUI.MENCWY:FLAC.(VETRO)-1 FLAC+1FLAC
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135)
Trade Name: BEXSERO - SOSPENSIONE INIETTABILE - USO INTRAMUSCOLARE - SIRINGA P
NOVARTIS PHARMA AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
165Phase 3United States;Taiwan;Spain;Malaysia;Brazil;Singapore;Korea, Democratic People's Republic of;Lithuania;Japan;China;Italy
11EUCTR2020-003172-41-FR
(EUCTR)
03/06/202115/04/2021Study of Efficacy and Safety of Twice Daily Oral iptacopan in Adult PNH Patients who are naïve to complement inhibitor therapyA 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 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
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 3United States;France;Czechia;Malaysia;Singapore;Germany;United Kingdom;Italy;Japan;China;Korea, Republic of
12EUCTR2019-004665-40-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
13EUCTR2019-004665-40-IT
(EUCTR)
23/12/202015/06/2021Study 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 - Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia d 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
Product Name: Eculizumab
Product Code: [-]
INN or Proposed INN: ECULIZUMAB
Trade Name: ACT-HIB Polvere e solvente per soluzione iniettabile in siringa preriempita 0,5 ml
Product Name: -
Product Code: [-]
INN or Proposed INN: HAEMOPHILUS INFLUENZAE
Trade Name: MENVEO – Polvere e soluzione per soluzione iniettabile
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO POLISACCARIDICO ANTI-MENINGOCOCCICO (A.C.Y E W-135)
Trade Name: PNEUMOVAX – Soluzione iniettabile in siringa preriempita da 0,5 ml
Product Name: -
Product Code: [-]
INN or Proposed INN: VACCINO PNEUMOCOCCICO
NOVARTIS PHARMA AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
91Phase 3United States;Czechia;Taiwan;Spain;Korea, Democratic People's Republic of;Italy;United Kingdom;France;Hungary;Canada;Brazil;Germany;Netherlands;Japan
14EUCTR2019-004665-40-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
15EUCTR2014-005338-74-LT
(EUCTR)
07/07/201509/06/2015To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNHAn 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) - CLFG316X2201 Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: LFG316
INN or Proposed INN: No
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Product Code: LFG316
INN or Proposed INN: No
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Product Name: iptacopan
Product Code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: iptacopan
Product Code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Novartis Pharma Services AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 2Czechia;Czech Republic;Lithuania;Japan
16EUCTR2014-005338-74-CZ
(EUCTR)
17/06/201523/04/2015To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNHAn 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) - CLFG316X2201 Paroxysmal nocturnal hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Code: LFG316
INN or Proposed INN: No
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Product Code: LFG316
INN or Proposed INN: No
Other descriptive name: human monoclonal antibody directed against complement 5 protein
Product Name: iptacopan
Product Code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Product Name: iptacopan
Product Code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Novartis Pharma Services AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 2Czechia;Czech Republic;Lithuania;Japan