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	NCT02973087 (ClinicalTrials.gov)	December 22, 2017	22/11/2016	rVWF IN PROPHYLAXIS	A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE	Von Willebrand Disease	Biological: von Willebrand factor (Recombinant);Biological: Antihemophilic Factor (Recombinant)	Baxalta now part of Shire	Baxalta Innovations GmbH, now part of Shire	Completed	18 Years	N/A	All	23	Phase 3	United States;Canada;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey
2	NCT02932618 (ClinicalTrials.gov)	December 18, 2017	12/10/2016	BAX 111 rVWF in Pediatrics	A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease	Von Willebrand Disease	Biological: von Willebrand factor (Recombinant);Biological: Antihemophilic Factor (Recombinant)	Baxalta now part of Shire	Baxalta Innovations GmbH, now part of Shire	Recruiting	N/A	17 Years	All	34	Phase 3	United States;Austria;Belgium;Czechia;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;Ukraine;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

NCT02973087
(ClinicalTrials.gov)

December 22, 2017

22/11/2016

rVWF IN PROPHYLAXIS

A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE

Von Willebrand Disease

Biological: von Willebrand factor (Recombinant);Biological: Antihemophilic Factor (Recombinant)

Baxalta now part of Shire

Baxalta Innovations GmbH, now part of Shire

Completed

18 Years

N/A

All

Phase 3

United States;Canada;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey

NCT02932618
(ClinicalTrials.gov)

December 18, 2017

12/10/2016

BAX 111 rVWF in Pediatrics

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

Von Willebrand Disease

Biological: von Willebrand factor (Recombinant);Biological: Antihemophilic Factor (Recombinant)

Baxalta now part of Shire

Baxalta Innovations GmbH, now part of Shire

Recruiting

N/A

17 Years

All

Phase 3

United States;Austria;Belgium;Czechia;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;Ukraine;United Kingdom