Study of Voncento® in Subjects with Von Willebrand Disease

Phase 1

Conditions: Von Willebrand Disease
MedDRA version: 18.1Level: PTClassification code 10047715Term: Von Willebrand's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2013-003305-25-IE

Lead Sponsor: CSL Behring GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 30

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.