SEVERE TYPE A HAEMOPHILIA MedDRA version: 9.1 Level: LLT Classification code 10010432 Term: Congenital deficiency of other clotting factors
Conditions
Interventions
Trade Name: FANHDI 500UI
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Coagulation factor VIII
Concentration unit: IU international unit(s)
Concentration type:
Sponsors
FONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: a. severe hemophilia A (FVIII 5 BU); d. any inhibitor level at study enrolment; e. ability and willingness to participate in to the study; f. no concomitant systemic treatment with drugs with immunosuppressive side effects (eg. Corticosteroids, if used more than 5 days every iii months and/or at a dose of > 2mg/kg or 60 mg/day), azathioprine, cyclophosphamide, high?dose immunoglobulin as well as the use of a protein A column or plasmapheresis, interferons); g. no concomitant experimental treatment; h. at least one of the following risk factors for ITI failure: i. peak inhibitor titer > 200 BU ii. titer at ITI start > 10 BU iii. age > 7 years iv. time between inhibitor occurrence and ITI > 2 years i. no previous ITI attempt. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: see inclusion criteria
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The study is aimed at evaluating whether FVIII/VWF concentrates can induce more frequently or more rapidly immune tolerance to FVIII in haemophilia A patients with high-responding inhibitors at high risk to fail in comparison with VWF-free FVIII concentrates.;Secondary Objective: I.Maintenance of immune tolerance II.Time to achieve success III.Safety - compliance to treatment IV.Cost of care;Primary end point(s): The study is aimed to evaluate whether FVIII/VWF concentrates can induce more frequently or more rapidly immune tolerance to FVIII in haemophilia A patients with high-responding inhibitors at high risk to fail in comparison with VWF-free FVIII concentrates. | — |
Countries
Italy, Spain
Outcome results
None listed