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	EUCTR2020-000501-93-AT (EUCTR)	27/11/2020	08/09/2020	A long term study of BCX9930 in patients with PNH	A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)	Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride	BioCryst Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 2	European Union;Austria;South Africa;United Kingdom
2	EUCTR2020-000501-93-GB (EUCTR)	29/09/2020	09/07/2020	A long term study of BCX9930 in patients with PNH	A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)	Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride	BioCryst Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 2	European Union;South Africa;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

EUCTR2020-000501-93-AT
(EUCTR)

27/11/2020

08/09/2020

A long term study of BCX9930 in patients with PNH

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride

BioCryst Pharmaceuticals Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

200

Phase 2

European Union;Austria;South Africa;United Kingdom

EUCTR2020-000501-93-GB
(EUCTR)

29/09/2020

09/07/2020