Brief Title
Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
Official Title
Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
Brief Summary
Primary objective: To assess post-marketing immunogenicity of Optivate® by monitoring plasma
inhibitor levels for at least 100 Exposure Days (EDs) for each subject.
Secondary objectives: To assess efficacy and tolerability by monitoring FVIII recovery and
adverse events
Detailed Description
The primary efficacy endpoint is to assess immunogenicity of Optivate® by monitoring plasma
inhibitor level for at least 100 EDs for each subject.
FVIII inhibitor evaluation FVIII inhibitor screen data will be listed. FVIII quantitative
inhibitor results will be listed. Shift tables will present the number of subjects with
positive (≥ 0.6 BU) and negative (< 0.6 BU) results and those for whom the results change
during the study. The number of exposure days until development of inhibitors will be
summarised.
For the secondary endpoints: Descriptive statistics will be presented on the number of
recoveries at each timepoint and for each subject. These will be presented for each visit and
for each subject and then for each batch of FVIII/ Optivate® used. All the AE data (from CRF
and study diary) will be pooled together and reported in terms of the type, duration,
treatment and/or severity.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Number of Participants That Did Not Develop Inhibitors to FVIII (<0.6BU)
Secondary Outcome
Recovery With Prior FVIII Concentrate (Screening Visit) Versus Recovery With First Dose With Optivate® (Visit 1) for the Protocol Population.
Condition
Haemophilia A
Intervention
Optivate 500IU
Study Arms / Comparison Groups
Optivate 500IU
Description: Optivate 500IU
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
7
Start Date
November 21, 2014
Completion Date
August 31, 2017
Primary Completion Date
August 31, 2017
Eligibility Criteria
Inclusion Criteria:
- Written informed consent or, if less than 18 years of age written assent (where
possible) and their parent/guardian's written informed consent.
- Severe haemophilia A (< 1%# FVIII:C).
- Previously Treated Patients (PTPs) with > 150 exposure days on prior Factor VIII
therapy (of which at least the last 50 EDs or 2 years treatment can be confirmed by
way of subject records).
- Immunocompetent with CD4 count > 200 / µl.
- HIV negative or a viral load < 200 particles / µl.
- subjects suffering from severe haemophilia A (<2%) may be enrolled, but only
after approval by BPL. Subjects with a Factor VIII of <2% may not constitute more
than 50% of the total patient population. A separate statistical evaluation will
be conducted for the <1% and <2% populations.
Exclusion Criteria:
- • History of inhibitor development to FVIII or a positive result on the Nijmegen
Bethesda at screening (quantitative result of > 0.6 BU) prior to the administration of
Optivate®.
- Known or suspected hypersensitivity to the investigational medicinal product or
its excipients.
- Clinically significant liver disease, renal disease, or coagulopathy other than
haemophilia A.
- History of unreliability or non cooperation (including not being able to complete
the study diary).
- Participating in, or have taken part in another trial within the last 30 days.
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Eric Wolford, ,
Location Countries
Colombia
Location Countries
Colombia
Administrative Informations
NCT ID
NCT01811875
Organization ID
8VWF07
Responsible Party
Sponsor
Study Sponsor
Bio Products Laboratory
Study Sponsor
Eric Wolford, Study Director, Bio Products Laboratory
Verification Date
July 2021