Clinical trial NCT02018393: Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients With Haemophilia A and Inhibitors
Official title: Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Severe Haemophilia A With Inhibitors
Keywords: FEIBA®
Lead sponsor: Hospital Universitario La Paz
Sponsor class: OTHER
First posted: 2013-12-23
Enrollment: 20
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: CROSS_SECTIONAL
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.

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

Eligibility criteria: 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.

Primary outcomes:
measure: Measurement of plasma levels of anti-FVIII antibodies (neutralising and non-neutralising)
timeFrame: At inclusion
Secondary outcomes:
measure: Measurement of the serum levels of inflammatory cytokines
timeFrame: At inclusion
Other outcomes:
Locations:
city: Madrid
country: Spain
facility: University Hospital La Paz
Study officials:
affiliation: Hospital Universitario La Paz
name: Victor Jimenez-Yuste, Medical doctor, PhD
role: PRINCIPAL_INVESTIGATOR
References: