Post-Marketing Study to monitor the safety of long term treatment with Optivate in patients with Haemophilia A.

Phase 1

• Conditions: Severe Haemophilia A; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2012-004606-10-PL
• Lead Sponsor: Bio Products Laboratory Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-06-29
• Last Update Posted: 21 August 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

- Written informed consent or, in the case of children and adolescents (less than 18 years of age) have given written assent (where possible) and whose parent/guardian has given written informed consent.

- Severe haemophilia A (< 1% FVIII:C). Subjects suffering from severe haemophilia A
(< 2%) may be enrolled, but only after approval by BPL. Subjects with a FVII
< 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. Basal
FVIII level taken from subject’s lowest level recorded, or the level measured at screening, whichever is lower.

- Previously Treated Patients (PTPs) with > 150 exposure days on prior FVIII therapy (of
which at least the last 50 EDs or 2 years treatment can be confirmed by way of subject
records).

- Immunocompetent subjects with CD4 (cluster differentiation 4) count > 200 /µL.

- Human Immunodeficiency Virus (HIV) negative subjects or a viral load < 200 particles
/µL.
Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 9
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

- History of inhibitor development to FVIII or a positive result on the Nijmegen-Bethesda at screening (quantitative result of > 0.6 Bethesda Units [BU]) prior to the administration of Optivate®.

- Known or suspected hypersensitivity to the Investigational Medicinal Product (IMP) or its excipients.

- Clinically significant i.e. symptomatic liver disease and/or (historical, within the last 12 months, serum Alanine Aminotransferase [ALT] levels greater than three times the
upper limit of the normal range), symptomatic renal disease and/or (historical, within the last 12 months, serum creatinine > 200?mol/L), 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified