Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A

A Phase II, Multicentre, Double-blinded, Randomised, Cross-over Study to Evaluate Efficacy, Safety and Pharmacokinetics of Biostate® in Subjects With Haemophilia A.

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00879541

Enrollment

Registered

2009-04-10

Start date

2009-02-28

Completion date

2010-10-31

Last updated

2011-02-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A

Keywords

Hemophilia A

Brief summary

The aim of this study are to * assess the efficacy of Biostate® \[Study Product (SP)\] in subjects with Haemophilia A * compare the pharmacokinetics of Biostate® \[SP\] with the previously marketed product Biostate® (here referred to as Biostate® \[Reference Product (RP)\]). This study is divided into 3 parts: Part 1: Cross-over pharmacokinetic (PK) component. PK subjects will be randomised to determine the order in which they receive the two study products. This part of the study is double-blinded. Part 2: Efficacy component. All subjects will receive Biostate® \[SP\] as required to manage their haemophilia condition for an estimated period of 6 months (or minimum of 50 exposure days) to assess efficacy and safety of the product. This part of the study is open-label. Part 3: Repeat pharmacokinetic assessment. Subjects who participated in Part 1 (PK component) will undergo a repeat PK assessment on Day 180 following administration of Biostate® \[SP\].

Interventions

BIOLOGICALBiostate® [SP]

Single bolus intravenous dose of 50 IU/kg

BIOLOGICALBiostate® [RP]

Single bolus intravenous dose of 50 IU/kg.

Study design

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

MALE

Age

12 Years to No maximum

Healthy volunteers

Inclusion criteria

* Diagnosed with Haemophilia A with ≤ 1% Factor VIII (FVIII) levels in the absence of factor replacement * Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunisation) within 10 years prior to Day 1 documented in the medical notes * At least 150 days of prior exposure to a FVIII replacement product * Written informed consent given

Exclusion criteria

(for participation in the pharmacokinetic (PK) component): * Active bleeding * Body weight \> 100 kg

Design outcomes

Primary

Measure	Time frame
Number of treatments/units required to resolve any bleeding event	From Day 1 until final study visit
Assessment of blood loss during any surgical procedure	From Day 1 until final study visit
Pharmacokinetics of FVIII activity	Up to 48 hours following infusions (Part 1 and Part 3 only)
Haemostatic efficacy	Monthly, until final study visit
FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)	From Day 1 until final study visit

Secondary

Measure	Time frame
Development of FVIII inhibitors	From Day 1 until final study visit
The nature, frequency and incidence of adverse events	From Day 1 until final study visit

Countries

Bulgaria, North Macedonia, Poland, Russia

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026