Study of Voncento® in Subjects with Von Willebrand Disease

• Register: EUCTR
• Last refreshed on: 28 May 2018
• Main ID: EUCTR2013-003305-25-GB
• Date of registration: 05/05/2015
• Prospective Registration: Yes
• Primary sponsor: CSL Behring GmbH
• Public title: Study of Voncento® in Subjects with Von Willebrand Disease
• Scientific title: An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects with Von Willebrand Disease
• Date of first enrolment: 07/07/2015
• Target sample size: 30
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-003305-25
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Austria, Germany, Greece, Ireland, Poland, United Kingdom

Contacts

• Name: Kerstin Jung,Clinical Study Manager
• Affiliation: CSL Behring GmbH

• Name: Kerstin Jung,Clinical Study Manager
• Affiliation: CSL Behring GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
Subjects meeting all of the following inclusion criteria may be enrolled into the study:
1.Capable of providing written informed consent and willing and able to adhere to all protocol requirements, or the subject’s parent(s) or legally acceptable representative(s) is / are capable of providing written informed consent.
2.Male or female.
3.Subjects of any age.
4.Diagnosis of severe Type 1, 2A, or 3 VWD where VWF:RCo is <20% at screening.
5.Availability of detailed patient documentation (eg, medical record, diary, logbook) covering prior bleeding and VWD treatment history over the past 12 months prior to screening.
6.Subjects where DDAVP treatment is ineffective or contraindicated. In countries where DDAVP is not available only subjects with Type 3 VWD are allowed to be enrolled.
7.A documented vaccination against hepatitis A and B in the subject’s medical record (or presence of antibodies against hepatitis A and B due to either a previous infection or vaccination) prior to the first dose of Voncento.
8.Investigator believes that the subject or the subject’s parent(s) or legally acceptable representative(s) is / are willing and able to adhere to all protocol requirements.
9.Subjects who require a VWF product to control a NSB event or for ongoing prophylactic therapy.
Are the trial subjects under 18? yes
Number of subjects for this age range: 14
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 14
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion criteria:
Subjects meeting any of the following exclusion criteria must not be enrolled into the study:
1. A known history of VWF or FVIII inhibitors, or are suspected to have VWF or FVIII inhibitors.
2. Suffering any acute or chronic medical condition, other than VWD, which may, in the opinion of the investigator, affect the conduct of the study.
3. Known or suspected hypersensitivity or previous evidence of severe side effects to Voncento, VWF / FVIII concentrates, or human albumin.
4. Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
5. Female subjects who are pregnant, breast-feeding, or who have a positive pregnancy test at screening or have the intention to become pregnant during the course of the study.
6. Female subjects of childbearing potential or male subjects who have a female partner of childbearing potential, who either do not use or are not willing to use a medically reliable method of contraception or who are not sexually abstinent during the study, or not surgically sterile.
7. Alcohol, drug, or medication abuse within 1 year before the study.
8. Currently receiving a therapy not permitted during the study
9. Previous participation in a Voncento / Biostate study (patients can also not be enrolled a second time into the current study).
10. Mental condition rendering the subject (or the subject’s legally acceptable representative[s]) unable to understand the nature, scope, and possible consequences of the study.
11. Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Von Willebrand Disease
MedDRA version: 18.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Trade Name: Voncento
Product Name: Voncento
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
INN or Proposed INN: Human Coagulation Factor VIII
Other descriptive name: HUMAN COAGULATION FACTOR VIII
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 500-
INN or Proposed INN: Human Von Willebrand Factor
Other descriptive name: HUMAN VON WILLEBRAND FACTOR
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 1200-

Trade Name: Voncento
Product Name: Voncento
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
INN or Proposed INN: Human Coagulation Factor VIII
Other descriptive name: HUMAN COAGULATION FACTOR VIII
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 1000-
INN or Proposed INN: Human Von Willebrand Factor
Other descriptive name: HUMAN VON WILLEBRAND FACTOR
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 2400-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: • Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento in its usage for an NSB event: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Assessed by the Investigator at approximately Months 3, 6, 9 and 12.
• Number of infusions: For the duration of the subject's participation in the study (approximately 12 months)
•Total dose of Voncento (in IU VWF:RCo) required to treat an NSB event: For the duration of the subject's participation in the study (approximately 12 months).
•Number of NSB events per month: For the duration of the subject's participation in the study (approximately 12 months).

Secondary Objective: The secondary objectives of the study are:
• To assess the efficacy of Voncento in the prevention and treatment of surgical bleeding events.
• To collect long-term data on the safety of Voncento used as on-demand therapy to treat an NSB event or as prophylaxis therapy.

Main Objective: The primary objective of the study is to collect long-term data on the haemostatic efficacy of Voncento in subjects with VWD who require a VWF product to control an NSB event or as prophylaxis therapy.

Primary end point(s): The primary endpoints of the study are:
• Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento in its usage for an NSB event.
•Number of NSB events per month and annual bleeding rate (ABR).
•Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat an NSB event.

Secondary Outcome(s)

Secondary end point(s): The secondary endpoints of the study are:
•Subject’s and investigator’s subjective assessment of haemostatic efficacy of Voncento as prophylaxis therapy.
•Investigator’s or surgeon’s assessment of blood loss during a surgical event and an overall haemostatic efficacy assessment by the investigator after the surgical procedure.
•Number of infusions and total dose of Voncento (in IU VWF:RCo) required to treat a surgical bleeding event.
•Nature and incidence of adverse events (AEs), including serious adverse events (SAEs), AEs related to Voncento, and adverse events of special interest (AESIs), and number of VWF and FVIII inhibitors which occur during the administration of Voncento as on-demand therapy to treat an NSB or surgical bleeding event, or as prophylaxis therapy.

Timepoint(s) of evaluation of this end point: The secondary endpoints of the study are:
• Investigator's or surgeon's assessment of blood loss during a surgical
event and an overall haemostatic efficacy assessment by the investigator
after the surgical procedure.
• Number of infusions and total dose of Voncento (in IU VWF:RCo)
required to treat a surgical bleeding event.
• Nature and incidence of AEs, including serious adverse events (SAEs),
AEs related to Voncento, and adverse events of special interest (AESIs),
and number of VWF and FVIII inhibitors which occur during the
administration of Voncento as on-demand therapy to treat an NSB or
surgical bleeding event.
•Haemostatic efficacy - prophylaxis: App. every month (subject assessment) and every 3 months (Investigator assessment)during subject's participation in study

Secondary ID(s)

CSLCT-BIO-12-83

Source(s) of Monetary Support

CSL Behring GmbH