Case-control Pilot Study of the Immune Modulating Effect... | NCT02018393

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

NCT02018393

Sponsor

Hospital Universitario La Paz

Status

Unknown status

Last Update Posted

Dec 23, 2013Estimated

Enrollment

20Estimated

Phase

Not provided

Conditions

Severe Haemophilia A With Inhibitors

Interventions

Not provided

Countries

Spain

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2013

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Not yet recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2014

Primary Completion Date

Jun 2014Estimated

Completion Date

Dec 2014Estimated

First Submitted Date

Dec 17, 2013

First Submission Date that Met QC Criteria

Dec 20, 2013

First Posted Date

Dec 23, 2013Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 20, 2013

Last Update Posted Date

Dec 23, 2013Estimated

Description Module

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®.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

20Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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.

Interventions

Not provided

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

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

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

The subject has signed and dated the Informed Consent form for participation in this study.
Age ≥ 18 years old.
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.
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:

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

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

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

Sampling Method

Non-Probability Sample