DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
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)］	1

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	EUCTR2016-001477-33-DE (EUCTR)	12/06/2018	27/07/2017	A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.	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.PIP decision numbers P/0091/2012 and P/0214/2015 and P/0394/2019 - BAX 111 rVWF in Pediatrics	Hereditary severe von Willebrand Disease in children 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: VEYONDI 650IU powder and solvent for solution for injection Product Name: Recombinant von Willebrand Factor 650IU Product Code: BAX111 INN or Proposed INN: Vonicog alfa Other descriptive name: HUMAN VON WILLEBRAND FACTOR Trade Name: ADVATE 500 IU powder and solvent for solution for injection Product Name: Advate INN or Proposed INN: OCTOCOG ALFA Product Name: Recombinant von Willebrand Factor 1300IU Product Code: BAX111 INN or Proposed INN: Vonicog alfa Other descriptive name: HUMAN VON WILLEBRAND FACTOR Trade Name: ADVATE 1000IU powder and solvent for solution for injection Product Name: Advate INN or Proposed INN: OCTOCOG ALFA	Baxalta Innovations GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	34	Phase 3	United States;Finland;Spain;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Germany;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

EUCTR2016-001477-33-DE
(EUCTR)

12/06/2018

27/07/2017

A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.

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.PIP decision numbers P/0091/2012 and P/0214/2015 and P/0394/2019 - BAX 111 rVWF in Pediatrics

Hereditary severe von Willebrand Disease in children
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: VEYONDI 650IU powder and solvent for solution for injection
Product Name: Recombinant von Willebrand Factor 650IU
Product Code: BAX111
INN or Proposed INN: Vonicog alfa
Other descriptive name: HUMAN VON WILLEBRAND FACTOR
Trade Name: ADVATE 500 IU powder and solvent for solution for injection
Product Name: Advate
INN or Proposed INN: OCTOCOG ALFA
Product Name: Recombinant von Willebrand Factor 1300IU
Product Code: BAX111
INN or Proposed INN: Vonicog alfa
Other descriptive name: HUMAN VON WILLEBRAND FACTOR
Trade Name: ADVATE 1000IU powder and solvent for solution for injection
Product Name: Advate
INN or Proposed INN: OCTOCOG ALFA

Baxalta Innovations GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;Finland;Spain;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Germany;Netherlands