Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients With Haemophilia A and Inhibitors

Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients

This study aims to evaluate the immunomodulatory effect of FEIBA® in patients with severe haemophilia A and inhibitors.

Study Overview

• Status: Unknown
• Conditions: Severe Haemophilia A With Inhibitors

Detailed Description

This is an observational, multicentric, case-control and one-single-visit study of patients with severe haemophilia A and inhibitors against FVIII in therapy with FEIBA®.

Two subject groups will be included: Group 1 (cases) will be composed of patients with severe haemophilia A and inhibitors in prophylaxis with FEIBA®; Group 2 (controls) will be composed of patients with severe haemophilia A and inhibitors on-demand treatment with FEIBA®.

• Study Type: Observational
• Enrollment (Anticipated): 20

Contacts and Locations

• Study Contact: Name: Ihosvany Fernandez-Bello, Pharmacist; Phone Number: +34669089737; Email: ihosvanyf@yahoo.es

Study Locations

• Spain
  • Madrid, Spain, 28046
    • University Hospital La Paz

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Patients with severe haemophilia A and inhibitors treated with FEIBA® (on demand or prophylaxis) for the control of bleedings.

Description

Inclusion Criteria:

1. The subject has signed and dated the Informed Consent form for participation in this study.
2. Age ≥ 18 years old.
3. Patient with severe haemophilia A (FVIII <1%) and high-responding inhibitor (titre >5 UB) at some point in their life, currently on therapy with FEIBA® to control bleeding.
4. Group 1: Patients in prophylaxis with FEIBA®: The patient has been on prophylaxis regimen with FEIBA® for at least 6 months prior to the study visit. Group 2: Patients on-demand regimen with FEIBA®: The patient has been under on-demand treatment with FEIBA® for at 6 months prior to the study visit.

Exclusion Criteria:

1. The presence of any inflammatory condition at the time of the study visit or the previous 30 days that, according to the medical criterion, would affect the study objectives.
2. The patient is under immune tolerance treatment or has been at any time during the 30 days prior to the study visit.
3. The administration of any anti-inflammatory or immunosuppressive drug 15 days before the study visit.
4. Levels of CD4 <200/l regardless of the HIV status.
5. Altered hepatic or renal function defined by the presence of abnormal levels of alanine aminotransferase (ALAT) or serum creatinine, respectively.
6. Administration of any haemostatic treatment to control bleeding within the 5 days prior to the study visit.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Control
• Time Perspectives: Cross-Sectional

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Prophylaxis group; Prophylaxis with FEIBA is defined in this study as the regular infusion of FEIBA for the prevention of bleeding at a dose of ≥50 UF/kg on at least three non-consecutive days a week. Patients must have been on this modality for at least 6 months prior to the study visit
On demand group; On-demand Treatment is defined as the administration of FEIBA only to control bleeding. Patients must have been on this modality for at least 6 months prior to the study visit.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Measurement of plasma levels of anti-FVIII antibodies (neutralising and non-neutralising)	At inclusion

Secondary Outcome Measures

Outcome Measure	Time Frame
Measurement of the serum levels of inflammatory cytokines	At inclusion

Collaborators and Investigators

• Sponsor: Hospital Universitario La Paz
• Investigators: Principal Investigator: Victor Jimenez-Yuste, Medical doctor, PhD, Hospital Universitario La Paz

Study record dates

Study Major Dates

• Study Start: January 1, 2014
• Primary Completion (Anticipated): June 1, 2014
• Study Completion (Anticipated): December 1, 2014

Study Registration Dates

• First Submitted: December 17, 2013
• First Submitted That Met QC Criteria: December 20, 2013
• First Posted (Estimate): December 23, 2013

Study Record Updates

• Last Update Posted (Estimate): December 23, 2013
• Last Update Submitted That Met QC Criteria: December 20, 2013
• Last Verified: December 1, 2013

More Information

Terms related to this study

• Keywords: FEIBA®
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: PI-1405