Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A

NCT ID: NCT01811875

Last Updated: 2021-07-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-11-21
• Study Completion Date: 2017-08-31

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.

Conditions

Haemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Optivate 500IU

Optivate 500IU

Group Type: EXPERIMENTAL

Optivate 500IU

Intervention Type: BIOLOGICAL

Interventions

Optivate 500IU

Intervention Type: BIOLOGICAL

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bio Products Laboratory

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Eric Wolford

Role: STUDY_DIRECTOR

Bio Products Laboratory

Locations

Fundacion BIOS

Barranquilla, , Colombia

Hospital general de Medellin

Medellín, , Colombia

HZRM Haemophilia Centre Rhine Main

Darmstadt, Mörfelden-Walldorf, Germany

Wojewodzki Szpital Specjalistyczny im. M. Kopernika

Lodz, , Poland

Countries

Colombia; Germany; Poland

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

8VWF07

Identifier Type: -

Identifier Source: org_study_id