DDrare: Database of Drug Development for Rare Diseases

62. 発作性夜間ヘモグロビン尿症
［臨床試験数：202，薬物数：120（DrugBank：25），標的遺伝子数：15，標的パスウェイ数：90］

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ABP 959; ABP 959 - biosimilar to eculizumab; ABP 959 - proposed biosimilar to eculizumab; ACH-0144471; ACH-0145228; ALN-62643; ALN-CC5; ALXN1102; ALXN1103; ALXN1210; ALXN2040 (ACH-0144471); ALXN2050; APL-2; Anti-C5 antibody; Anti-thymocyte globulin; BCD-148; BCX9930; BCX9930 hydrochloride; Bryostatin 1; C5 Inhibitor; C5 inh MAb, SKY59, RO/CH7092230; C5 inhibitor background therapy; COVERSIN; CROVALIMAB; Campath; Campath, Chemo and/or TBI Allo SCT; Coversin; Crovalimab; Cyclophosphamide; Cyclosporin; Cyclosporin A; Cyclosporin A+Glucocorticoids; Cyclosporine; Cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant; DANICOPAN; Danicopan; Danicopan (ALXN2040); Device: Miltenyi Biotec CliniMACS; Device: Miltenyi CliniMACS; Dextran; ECULIZUMAB; Eculizumab; Elizaria®; Fc- and CDR-modified humanised monoclonal antibody against C5; Filgrastim; Fludarabine; Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Glucocorticoids; H5G1.1-mAb; Human monoclonal antibody directed against complement 5 protein; Iron Dextran; Iron sucrose; L04AA25; LFG316; LNP023; Levamisole; Levamisole+cyclosporin A+Glucocorticoids; Melphalan; Methotrexate; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Myeloablative Preparative Regimen; No; Other: Blood collection for measurement of eculizumab peak concentrations; Other: Laboratory Biomarker Analysis; Other: Standard of care (SOC); Other: laboratory biomarker analysis; Other: pharmacological study; Pegcetacoplan; Pegcetacoplan (APL-2); Phosphate; Procedure: Allogeneic stem cell transplantation; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Stem Cell Transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; RA101495; RA101495 sodium; RAVULIZUMAB; REGN3918; RO7112689; RO7112689/F01; RO7112689/F03-01; RO7112689/F03-03; RVA576; RVA576 (COVERSIN); RVA576 (Coversin); Radiation: Total-Body Irradiation; Radiation: total-body irradiation; Ravulizumab; Reduced Intensity Preparative Regimen; Reduced Toxicity Ablative Regimen; SB12 (proposed eculizumab biosimilar); SOLIRIS; Sargramostim; Sirolimus; Soliris; Soliris (eculizumab); Soliris 300; Soliris®; Sterile Normal Saline (0.9% NaCl); Sucrose; Synthetic 15-amino-acid macrocyclic peptide; Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24; Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker; Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker; Systematic chemotherapy and antibodies; Systemic chemotherapy and antibodies; Tacrolimus; ULTOMIRIS; Ultomiris; VARI; ZILUCOPLAN; Zilucoplan; Zilucoplan (RA101495);  LNP023 HYDROCHLORIDE SALT

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2016-004129-18-LT (EUCTR)	15/03/2019	29/01/2019	An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Code: rVA576 INN or Proposed INN: rVA576 Other descriptive name: COVERSIN	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom;India
2	EUCTR2016-004129-18-GB (EUCTR)	17/03/2017	21/12/2016	An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Code: rVA576 INN or Proposed INN: rVA576 Other descriptive name: rVA576	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom
3	NCT03829449 (ClinicalTrials.gov)	March 13, 2017	20/12/2018	rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal Nocturnal Hemoglobinuria;Atypical Hemolytic Uremic Syndrome	Drug: rVA576 (Coversin)	AKARI Therapeutics	NULL	Recruiting	18 Years	N/A	All	50	Phase 3	Poland
4	EUCTR2016-004129-18-PL (EUCTR)	07/03/2017	18/01/2017	An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;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: rVA576 (Coversin) Product Code: rVA576 INN or Proposed INN: rVA576 (COVERSIN) Other descriptive name: rVA576 (COVERSIN)	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2016-004129-18-LT
(EUCTR)

15/03/2019

29/01/2019

An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Product Code: rVA576
INN or Proposed INN: rVA576
Other descriptive name: COVERSIN

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom;India

EUCTR2016-004129-18-GB
(EUCTR)

17/03/2017

21/12/2016

An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Product Code: rVA576
INN or Proposed INN: rVA576
Other descriptive name: rVA576

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom

NCT03829449
(ClinicalTrials.gov)

March 13, 2017

20/12/2018

rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Paroxysmal Nocturnal Hemoglobinuria;Atypical Hemolytic Uremic Syndrome

Drug: rVA576 (Coversin)

AKARI Therapeutics

NULL

Recruiting

18 Years

N/A

All

Phase 3

Poland

EUCTR2016-004129-18-PL
(EUCTR)

07/03/2017

18/01/2017

An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Product Name: rVA576 (Coversin)
Product Code: rVA576
INN or Proposed INN: rVA576 (COVERSIN)
Other descriptive name: rVA576 (COVERSIN)

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom