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

Madrid Health Service

Status: Unknown

Conditions: Severe Haemophilia A With Inhibitors

Study type: Observational

Funder types: Other

Identifiers

NCT02018393

PI-1405

Details and patient eligibility

About

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

Full 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®.

Enrollment: 20 estimated patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

20 participants in 2 patient groups

Prophylaxis group

Description:

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

Description:

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.