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

NCT01811875 | Terminated Phase 4 Results

• 1 other identifier: 8VWF07
• Study Type: interventional
• Target: 7
• Locations: 3 countries
• Sites: 4

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

Conditions

Haemophilia A

Keywords

Haemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of Participants That Did Not Develop Inhibitors to FVIII (<0.6BU)

  FVIII inhibitor status at any of the study visits was measured by a Nijmegen Bethesda assay and inhibitor screens. A result of ≥ 0.6 BU confirmed that the subject had developed inhibitors to FVIII. If this occurred, the test was repeated on a separate sample; if both tests were confirmed to be ≥ 0.6 BU, this was to be reported by the Investigator as a serious adverse event (SAE).

  At least 100 Exposure Days for each subject. Subjects will attend 5 visits over a period of up to 12 months

Secondary Outcomes (12)

• Recovery With Prior FVIII Concentrate (Screening Visit) Versus Recovery With First Dose With Optivate® (Visit 1) for the Protocol Population.

  Screening and Visit 1 (up to 4 weeks)

• Optivate® Recovery Across Visits 1 to 4 for the Protocol Population.

  Visits 1 to 4 (Up to 100 Optivate exposure days)

• Optivate® Therapy to Treat Breakthrough Bleeds Per Subject Per Year in the Protocol Population.

  Over a period of 12 months

• Overall Consumption of Optivate®: Number of Exposure Days for Each Subject Per Year/Subject in the Per Protocol Population.

  Over a period of 12 months

• Overall Consumption of Optivate®: Total Dose in IU/kg of Optivate® Per Subject for Prophylactic Use.

  Over a period of 12 months

Study Arms (1)

Optivate 500IU

EXPERIMENTAL

Optivate 500IU

Biological: Optivate 500IU

Interventions

Optivate 500IU BIOLOGICAL

Optivate 500IU

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bio Products Laboratory: lead

Study Sites (4)

Fundacion BIOS

Barranquilla, 80-216, Colombia

Location

Hospital general de Medellin

Medellín, 32-102, Colombia

Location

HZRM Haemophilia Centre Rhine Main

Darmstadt, Mörfelden-Walldorf, 64546, Germany

Location

Wojewodzki Szpital Specjalistyczny im. M. Kopernika

Lodz, 93-513, Poland

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

optivate

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: European Medical Affairs Lead
• Organization: Bio Products Laboratory Ltd

medinfo@bpl.co.uk

+44 20 8957 2200

Study Officials

• Eric Wolford

  Bio Products Laboratory

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2013
• First Posted: March 15, 2013
• Study Start: November 21, 2014
• Primary Completion: August 31, 2017
• Study Completion: August 31, 2017
• Last Updated: July 22, 2021
• Results First Posted: July 22, 2021

Record last verified: 2021-07

Locations

DE (1); CO (2); PL (1)