Severe hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Trade Name: KLOTT - 500 UI/10 ML POLV E SOLVENTE PER SOLUZIONE PER INFUSIONE 1 FLAC.NO POLVERE + 1 FLAC.NO SOLVENTE DA 10 ML + SET PER LA RICOSTITUZIONE/SOMMINISTRAZION
Product Name: Fattore VIII plas
Sponsors
FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE POLICLINICO
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Male subjects 2. Any ethnicity 3. Age =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Previous history of inhibitor 2. Other congenital or acquired bleeding defects 3. Plasma FVIII level = 1% as assayed at the central laboratory. Patients diagnosed locally as severe and not confirmed by the central evaluation will be considered screening failure and excluded from the study. 4. Concomitant congenital or acquired immunodeficiencies 5. Concomitant treatment with systemic immunosuppressive drugs
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Aim of the study is to assess whether or not switching to the use of a rFVIII (standard and extended half-life) product after the period of higher risk for inhibitor occurrence (first 50 EDs) is as safe as continuing with plasma-derived products lifelong, with no occurrence of a new peak of inhibitor development;Secondary Objective: to investigate the frequency of transient inhibitors; the modality of occurrence of inhibitors (number of EDs, titre at onset etc.); the clinical factors potentially associated to inhibitor development (age at first treatment, family history of haemophilia, family history of inhibitor, surgery, severity of treatment); the laboratory measurable factors potentially involved in inhibitor development (FVIII gene defect, FVIII antigen levels, subclass of inhibitor, epitope mapping); the incidence of any potential adverse event related to this proposed treatment schedule. As ancillary end-points, the bleeding history will be evaluated measuring the annualized bleeding rate (ABR). ;Primary end point(s): The primary end-point is the development of inhibitors during 50-100 EDs, centrally confirmed, and non-transient. ;Timepoint(s) of evaluation of this end point: Inhibitor monitoring will include an assessment at the central laboratory with a Nijmegen-modified Bethesda assay 1) at screening, 2) every 5 EDs, for the first 20 EDs, then 3) every 10 EDs for the remaining 30 EDs or at any time inhibitor development will be clinically suspected, 4) after the first 50 EDs, again 5) every 5 EDs, for the additional first 20 EDs, 6) every 10 EDs for the last 30 EDs and 7) at the end of the study or at any time inhibitor development will be clinically suspected. Patients will be followed until inhibitor development or until 100EDs, whichever comes first. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): to investigate the frequency of transient inhibitors; the modality of occurrence of inhibitors (number of EDs, titre at onset etc.); the clinical factors potentially associated to inhibitor development (age at first treatment, family history of haemophilia, family history of inhibitor, surgery, severity of treatment); the laboratory measurable factors potentially involved in inhibitor development (FVIII gene defect, FVIII antigen levels, subclass of inhibitor, epitope mapping); the incidence of any potential adverse event related to this proposed treatment schedule. As ancillary end-points, the bleeding history will be evaluated measuring the annualized bleeding rate (ABR). ;Timepoint(s) of evaluation of this end point: At the end of the study | — |
Countries
Egypt, India, Iran, Islamic Republic of, Italy, Saudi Arabia, South Africa, United States
Contacts
Public ContactUOC Medicina Generale - Emostasi e
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Outcome results
None listed