DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2018-001631-46-NL (EUCTR)	14/02/2019	13/02/2019	Effects of pharmacokinetic models in dosing of DDAVP and/or von Willebrand factor-containing concentrates in patients with von Willebrand disease	Implementation of pharmacokinetic-guided dosing of DDAVP and VWF-containing concentrates in von Willebrand disease - OPTI-CLOT: To WiN	Von Willebrand disease MedDRA version: 20.0;Level: LLT;Classification code 10055168;Term: Von Willebrand's factor deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Trade Name: Minrin INN or Proposed INN: DESMOPRESSIN Trade Name: Octostim INN or Proposed INN: DESMOPRESSIN Trade Name: Octostim INN or Proposed INN: DESMOPRESSIN Trade Name: Haemate P INN or Proposed INN: HUMAN VON WILLEBRAND FACTOR Other descriptive name: HUMAN VON WILLEBRAND FACTOR INN or Proposed INN: HUMAN COAGULATION FACTOR VIII Other descriptive name: HUMAN COAGULATION FACTOR VIII Trade Name: Wilate INN or Proposed INN: HUMAN COAGULATION FACTOR VIII Other descriptive name: HUMAN COAGULATION FACTOR VIII INN or Proposed INN: HUMAN VON WILLEBRAND FACTOR Other descriptive name: HUMAN VON WILLEBRAND FACTOR Trade Name: Wilfactin INN or Proposed INN: HUMAN VON WILLEBRAND FACTOR Other descriptive name: HUMAN VON WILLEBRAND FACTOR	Erasmus University Medical Center	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	120	Phase 4	Netherlands
2	EUCTR2009-017060-17-NL (EUCTR)	10/12/2009	05/11/2009	STIMULATE study: Statins influence on Minrin upregulation of von Willebrand factor and factor VIII - STIMULATE	STIMULATE study: Statins influence on Minrin upregulation of von Willebrand factor and factor VIII - STIMULATE	Type 1 von Willebrand disease and mild hemophilia A	Trade Name: simvastatine Product Name: simvastatin INN or Proposed INN: SIMVASTATIN Trade Name: Minrin Product Name: Desmopressine INN or Proposed INN: DESMOPRESSIN	Academic Medical Center	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes			Netherlands

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

EUCTR2018-001631-46-NL
(EUCTR)

14/02/2019

13/02/2019

Effects of pharmacokinetic models in dosing of DDAVP and/or von Willebrand factor-containing concentrates in patients with von Willebrand disease

Implementation of pharmacokinetic-guided dosing of DDAVP and VWF-containing concentrates in von Willebrand disease - OPTI-CLOT: To WiN

Von Willebrand disease
MedDRA version: 20.0;Level: LLT;Classification code 10055168;Term: Von Willebrand's factor deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Trade Name: Minrin
INN or Proposed INN: DESMOPRESSIN
Trade Name: Octostim
INN or Proposed INN: DESMOPRESSIN
Trade Name: Octostim
INN or Proposed INN: DESMOPRESSIN
Trade Name: Haemate P
INN or Proposed INN: HUMAN VON WILLEBRAND FACTOR
Other descriptive name: HUMAN VON WILLEBRAND FACTOR
INN or Proposed INN: HUMAN COAGULATION FACTOR VIII
Other descriptive name: HUMAN COAGULATION FACTOR VIII
Trade Name: Wilate
INN or Proposed INN: HUMAN COAGULATION FACTOR VIII
Other descriptive name: HUMAN COAGULATION FACTOR VIII
INN or Proposed INN: HUMAN VON WILLEBRAND FACTOR
Other descriptive name: HUMAN VON WILLEBRAND FACTOR
Trade Name: Wilfactin
INN or Proposed INN: HUMAN VON WILLEBRAND FACTOR
Other descriptive name: HUMAN VON WILLEBRAND FACTOR

Erasmus University Medical Center

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

120

Phase 4

Netherlands

EUCTR2009-017060-17-NL
(EUCTR)

10/12/2009

05/11/2009

STIMULATE study: Statins influence on Minrin upregulation of von Willebrand factor and factor VIII - STIMULATE

Type 1 von Willebrand disease and mild hemophilia A

Trade Name: simvastatine
Product Name: simvastatin
INN or Proposed INN: SIMVASTATIN
Trade Name: Minrin
Product Name: Desmopressine
INN or Proposed INN: DESMOPRESSIN

Academic Medical Center

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

Netherlands