DDrare: Database of Drug Development for Rare Diseases

288. 自己免疫性後天性凝固因子欠乏症［自己免疫性出血病XIII (~2017.3)］
［臨床試験数：189，薬物数：219（DrugBank：29），標的遺伝子数：18，標的パスウェイ数：26］

Searched query = "Autoimmune acquired coagulation factor deficiency", "Coagulation factor deficiency", "Factor XIII deficiency", "Factor VIII deficiency", "Acquired hemophilia A", "von Willebrand Disease", "Factor V deficiency"
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 = ADVATE; ADVATE (rAHF - PFM); ADVATE (rAHF-PFM); ADVATE 1000 IU powder and solvent for solution for injection; ADVATE 1000IU powder and solvent for solution for injection; ADVATE 500 IU powder and solvent for solution for injection; ALPHANATE*INF 1F 1000UI+F 10ML; ALPHANATE*INF 1F 1500UI+F 10ML; ALPHANATE*INF 1F 250UI+F 5ML; ALPHANATE*INF 1F 500UI+F 5ML; APCC, aPCC + TXA; ARC 1779; ARC1779; ARC1779 Injection; Acetate; Advate; Alphanate; Alphanate 1000 I.U.; Alphanate 1500 I.U.; Alphanate SD/HT; Antihemophilic Factor (Recombinant); BAX 111; BAX111; BIVV001; BIVV001 (rFVIIIFc-VWF-XTEN); Biostate; Biostate®; Blood coagulation Factor VIII and vWF, human; Blood sample; Bortezomib; Catridecacog; Chloride; Cluvot; CoFactor; Coagulación factor VIII; Coagulation factor VIII; Coagulation factor XIII; Cyclophosphamide; DDAVP; DDAVP (Desmopressin); DESMOPRESSIN; Desmopressin; Desmopressin Acetate; Desmopressin acetate; Desmopressine; Device: Sublingual videomicroscopy; Diagnostic Test: blood sample , doppler of uterine vessel and placenta; Diagnostic Test: fibrinogen plasma concentration, coagulation factor XIII activity; Emicizumab; Emicizumab (ACE910, RO5534262); Emicizumab Injection; Eqwilate; F13CD; F8VR; FACTANE 200 IU/ml; FANHDI 100 UI; FANHDI 25 UI; FANHDI 250UI*1F 250UI+F 10ML; FANHDI 50 UI; FANHDI*INF FL 250UI+SIR SOLV+S; FANHDI*INF FL 500UI+SIR SOLV+S; FANHDI*INF FL1000UI+SIR SOLV+S; FANHDI*INF FL250UI+SIR SOLV+S; FXIII Concentrate (Human); FXIII Concentrate (Human) (FXIII); Factor VIII; Factor VIII concentrate; Factor XIII; Factor XIII Concentrate (Human); Factor XIII Concentrate (Human), Pasteurized; Factor von Willebrand; Fanhdi; Fanhdi 100 UI; Fanhdi 100 UI FVIII-120 UI FVW; Fanhdi 25 UI; Fanhdi 25 UI FVIII-30 UI FVW; Fanhdi 50 UI; Fanhdi 50 UI FVIII-60 UI FVW; Fibrinogen; Fibrogammin P; Fibrogammin® P; Fibrogammin®P; HEMOFIX; HUMAN COAGULATION FACTOR VIII; HUMAN COAGULATION FACTOR VIII, VON WILLEBRAND FACTOR COMPLEX; HUMAN VON WILLEBRAND FACTOR; Haemate P; Haemate P® (Human Coagulation Factor VIII); Haemate-P, Wilate; Haemate® P 250; Human Coagulation Factor VIII; Human Coagulation Factor VIII, Von Willebrand Factor Complex; Human VWF/FVIII concentrate; Human Von Willebrand Factor; Human coagulation Factor VIII / von Willebrand Factor; Human coagulation factor VIII; Human coagulation factor VIII associated with Von Willebrand factor (VWF); Human coagulation factor VIII associated with von Willebrand factor (VWF); Human von Willebrand Factor; Human von Willebrand factor; Human von Willebrand factor (VWF) and coagulation factor VIII (FVIII); Human von Willebrand factor (vWF) and coagulation factor VIII (FVIII); Human von willebrand factor; Humate-P; IL-11; Interleukin 11; Interleukin-11; Marketed plasma-derived VWF/FVIII concentrate; Minirin; Minrin; NN 1841; NN1841; Natriumchlorid; Natriumchlorid 0,9 %; Neumega (Oprelvekin, Interleukin 11, IL-11); OBI-1; OBIZUR; OCTANATE 100 UI/ml, poudre et solvant pour solution injectable; OCTANATE 50 UI/ml, poudre et solvant pour solution injectable; OCTOCOG ALFA; Obizur; Obyoctocog alfa; Octanate; Octocog alfa; Octostim; Oprelvekin; Oprelvekin, Interleukin 11, IL-11; Optivate; Optivate® (Human Coagulation Factor VIII); Other: Use of a postpartum diary and additional blood draws; Other: Use of a postpartum diary and additional blood draws.; Physiologische Kochsalzlösung Fresenius - Infusionslösung; Phytomenadione; Plasma-derived FVIII/VWF concentrate; Prednisone; Prothrombin; Prothrombin Complex Concentrate; Prothrombin complex concentrate; RAHF; RECOMBINANT HUMAN COAGULATION FACTOR VIII FC - VON WILLEBRAND FACTOR - XTEN FUSION PROTEIN; RFVIII; RFVIIa; RFVIIa, rFVIIa + TXA; RVWF; Recombinant Factor XIII; Recombinant Factor XIII (rFXIII); Recombinant Von Willebrand factor; Recombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein; Recombinant coagulation FVIII Fc – vonWillebrand factor – XTEN fusion protein; Recombinant factor VIIII (rFVIII); Recombinant factor XIII (rFXIII); Recombinant human von Willebrand Factor; Recombinant interleukin-11; Recombinant porcine coagulation factor VIII (B domain deleted); Recombinant porcine coagulation factor VIII (B-domain deleted); Recombinant von Willebrand Factor; Recombinant von Willebrand Factor (rVWF); Recombinant von Willebrand Factor 1300IU; Recombinant von Willebrand Factor 650IU; Recombinant von Willebrand factor; Recombinant von Willebrand factor (rVWF); Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII); Recomibnant human Von Willebrand Factor; Rituxan; Rituximab; Rurioctocog alfa pegol; SIMVASTATIN; SODIUM CHLORIDE SOLUTION 0.9%; Saline Solution; Simvastatin; Simvastatine; Sodium chloride; Standard of Care FVIII Replacement therapy; Standard of Care for Haemophilia A; Steroid; TAK-672; Tranexamic Acid; Tranexamic Acid Injection [Cyklokapron]; Tranexamic acid; Uterine; VEYONDI 650IU powder and solvent for solution for injection; VEYVONDI 1300IU powder and solvent for solution for injection; VEYVONDI 650IU powder and solvent for solution for injection; VON WILLEBRAND FACTOR; VONICOG ALFA; VWD factor VIII concentrate; VWF SD-35-DH; VWF replacement therapy with Wilate; VWF-containing products; VWF/FVIII products; Vessel; Von Willebrand Factor; Von Willebrand Factor Antigen; Von Willebrand Factor Ristocetin Cofactor; Von Willebrand Faktor; Von Willebrand factor; Von Willebrand factor (Recombinant); Von Willebrand factor and coagulation factor VIII in combination; Von willebrand factor; Voncento; Voncento, Biostate®; Voncento®; Vonicog Alfa; Vonicog alfa; WILATE; WILATE 1000 I.U.; WILATE 450; WILATE 500 I.U.; WILATE 900; WILATE® 450; WILFACTIN; Wilate; Wilate 1000; Wilate 1000 I.U.; Wilate 500; Wilate 500 I.U.; Wilfactin; Willfact; Wiloctin

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	NCT04567511 (ClinicalTrials.gov)	March 2021	29/7/2020	Hemlibra in Mild Hemophilia A	Prospective, Single-Arm, Open-Label Use of Hemlibra (Emicizumab) in the Treatment of Mild Hemophilia A	Factor VIII Deficiency, Congenital	Drug: Emicizumab	Indiana Hemophilia &Thrombosis Center, Inc.	Genentech, Inc.	Not yet recruiting	5 Years	45 Years	Male	40	Phase 4	NULL
2	JPRN-JapicCTI-205151	15/6/2020	07/02/2020	PHASE III STUDY OF EMICIZUMAB IN PATIENTS WITH ACQUIRED HEMOPHILIA A (AGEHA)	A MULTICENTER, OPEN-LABEL, NON-RANDOMIZED, PHASE III STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB IN PATIENTS WITH ACQUIRED HEMOPHILIA A	Acquired hemophilia A	Intervention name : emicizumab (ACE910, RO5534262) INN of the intervention : emicizumab Dosage And administration of the intervention : Emicizumab will be administered subcutaneously once weekly. Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : -	Chugai Pharmaceutical Co., Ltd.	NULL	recruiting	18		BOTH	11	Phase 3	Japan
3	NCT04188639 (ClinicalTrials.gov)	May 2020	4/12/2019	Emicizumab in Acquired Hemophilia A	Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial	Hemophilia A, Acquired	Drug: Emicizumab Injection	GWT-TUD GmbH	Hoffmann-La Roche;Hannover Medical School	Not yet recruiting	18 Years	N/A	All	51	Phase 2	NULL
4	NCT04158648 (ClinicalTrials.gov)	February 10, 2020	7/11/2019	A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors	A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors	Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor;Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor;Hemophilia A	Drug: Emicizumab	Hoffmann-La Roche	NULL	Recruiting	N/A	N/A	All	70	Phase 3	United States;Belgium;Canada;France;Germany;Italy;Netherlands;Poland;South Africa;Spain;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

NCT04567511
(ClinicalTrials.gov)

March 2021

29/7/2020

Hemlibra in Mild Hemophilia A

Prospective, Single-Arm, Open-Label Use of Hemlibra (Emicizumab) in the Treatment of Mild Hemophilia A

Factor VIII Deficiency, Congenital

Drug: Emicizumab

Indiana Hemophilia &Thrombosis Center, Inc.

Genentech, Inc.

Not yet recruiting

5 Years

45 Years

Male

Phase 4

NULL

JPRN-JapicCTI-205151

15/6/2020

07/02/2020

PHASE III STUDY OF EMICIZUMAB IN PATIENTS WITH ACQUIRED HEMOPHILIA A (AGEHA)

A MULTICENTER, OPEN-LABEL, NON-RANDOMIZED, PHASE III STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB IN PATIENTS WITH ACQUIRED HEMOPHILIA A

Acquired hemophilia A

Intervention name : emicizumab (ACE910, RO5534262)
INN of the intervention : emicizumab
Dosage And administration of the intervention : Emicizumab will be administered subcutaneously once weekly.
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -

Chugai Pharmaceutical Co., Ltd.

NULL

recruiting

BOTH

Phase 3

Japan

NCT04188639
(ClinicalTrials.gov)

May 2020

4/12/2019

Emicizumab in Acquired Hemophilia A

Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial

Hemophilia A, Acquired

Drug: Emicizumab Injection

GWT-TUD GmbH

Hoffmann-La Roche;Hannover Medical School

Not yet recruiting

18 Years

N/A

All

Phase 2

NULL

NCT04158648
(ClinicalTrials.gov)

February 10, 2020

7/11/2019

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor;Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor;Hemophilia A

Drug: Emicizumab

Hoffmann-La Roche

NULL

Recruiting

N/A

All

Phase 3

United States;Belgium;Canada;France;Germany;Italy;Netherlands;Poland;South Africa;Spain;United Kingdom