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	NCT01194804 (ClinicalTrials.gov)	April 2008	30/8/2010	E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients	Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol	Paroxysmal Nocturnal Hemoglobinuria	Drug: Eculizumab	Alexion Pharmaceuticals	CMIC Co, Ltd. Japan	Completed	12 Years	N/A	All	27	Phase 2	NULL
2	NCT01192399 (ClinicalTrials.gov)	November 2007	30/8/2010	Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients	Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients	Paroxysmal Nocturnal Hemoglobinuria	Biological: Eculizumab	Alexion Pharmaceuticals	NULL	Completed	12 Years	N/A	All	29	Phase 2	NULL
3	EUCTR2005-000043-28-IT (EUCTR)	01/09/2005	06/02/2007	A Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studies	A Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studies	paroxymal nocturnal hemoglobinuria MedDRA version: 6.1;Level: PT;Classification code 10034042	Product Name: Eculizumab Product Code: h5G1.1-mAb Product Name: Eculizumab Product Code: h5G1.1-mAb	ALEXION PHARMACEUTICALS INC	NULL	Not Recruiting			Female: yes Male: yes	170	Phase 3	Germany;United Kingdom;Spain;Ireland;Italy;Sweden

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

NCT01194804
(ClinicalTrials.gov)

April 2008

30/8/2010

E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol

Paroxysmal Nocturnal Hemoglobinuria

Drug: Eculizumab

Alexion Pharmaceuticals

CMIC Co, Ltd. Japan

Completed

12 Years

N/A

All

Phase 2

NULL

NCT01192399
(ClinicalTrials.gov)

November 2007

30/8/2010

Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Paroxysmal Nocturnal Hemoglobinuria

Biological: Eculizumab

Alexion Pharmaceuticals

NULL

Completed

12 Years

N/A

All

Phase 2

NULL

EUCTR2005-000043-28-IT
(EUCTR)

01/09/2005

06/02/2007

A Phase III Open label extension study of Eculizumab in patients with transfusion dependent hemolytic paroxysmal nocturnal hemoglobinuria PNH who have participated in Triumph C04-001 , Shepherd C04-002 or X03-001 studies

paroxymal nocturnal hemoglobinuria
MedDRA version: 6.1;Level: PT;Classification code 10034042

Product Name: Eculizumab
Product Code: h5G1.1-mAb
Product Name: Eculizumab
Product Code: h5G1.1-mAb

ALEXION PHARMACEUTICALS INC

NULL

Not Recruiting

Female: yes
Male: yes

170

Phase 3

Germany;United Kingdom;Spain;Ireland;Italy;Sweden