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	NCT01760096 (ClinicalTrials.gov)	January 2013	1/1/2013	Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)	Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)	Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic	Drug: Levamisole +cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids	Institute of Hematology & Blood Diseases Hospital	NULL	Active, not recruiting	18 Years	N/A	Both	50	Phase 2	China
2	NCT01642979 (ClinicalTrials.gov)	July 2012	14/7/2012	Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria	Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria	Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic	Drug: Levamisole +cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids	Institute of Hematology & Blood Diseases Hospital	NULL	Active, not recruiting	18 Years	N/A	Both	120	Phase 2	China
3	NCT00397813 (ClinicalTrials.gov)	January 2006	9/11/2006	Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders	Low-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Center Trial	Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Chronic Myelomonocytic Leukemia;de Novo Myelodysplastic Syndrome;Essential Thrombocythemia;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Polycythemia Vera;Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase;Primary Myelofibrosis;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts	Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation	Fred Hutchinson Cancer Research Center	National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)	Completed	50 Years	75 Years	All	77	Phase 2	United States;Denmark;Germany;Italy

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

NCT01760096
(ClinicalTrials.gov)

January 2013

1/1/2013

Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)

Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)

Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic

Drug: Levamisole +cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids

Institute of Hematology & Blood Diseases Hospital

NULL

Active, not recruiting

18 Years

N/A

Both

Phase 2

China

NCT01642979
(ClinicalTrials.gov)

July 2012

14/7/2012

Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria

Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic

Drug: Levamisole +cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids

Institute of Hematology & Blood Diseases Hospital

NULL

Active, not recruiting

18 Years

N/A

Both

120

Phase 2

China

NCT00397813
(ClinicalTrials.gov)

January 2006

9/11/2006

Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders

Low-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Center Trial

Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Chronic Myelomonocytic Leukemia;de Novo Myelodysplastic Syndrome;Essential Thrombocythemia;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Polycythemia Vera;Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase;Primary Myelofibrosis;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts

Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation

Fred Hutchinson Cancer Research Center

National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)

Completed

50 Years

75 Years

All

Phase 2

United States;Denmark;Germany;Italy