Infants or children with severe haemophilia A previously untreated with factor concentrates. MedDRA version: 16.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Advate
Product Name: Advate
Pharmaceutical Form: Lyophilisate for solution for infusion
Sponsors
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: The principal inclusion criteria are: 1) An informed consent, approved by the appropriate Institutional Review Board(IRB)/Independent Ethics Committee (IEC) has been administered, signed and dated by the parents or legal rapresentative of the patients 2) Subject diagnosed with severe haemophilia A defined by a baseline FVIII:C =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The principal exclusion criteria are: 1) Subjects with prior exposure to clotting factor concentrates or blood products. 2) Subjects with a clinically significant chronic disease other than hemophilia A. 3) Subjects that are currently partecipating to other investigational clinical trials
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to evaluate the changes in the immune system upon exposure to FVIII in patients with severe hemophilia A.;Secondary Objective: The secondary objective of the trial is to identify predictors of FVIII inhibitor development or tolerance to FVIII infusions.;Primary end point(s): In relation to the primary endpoints, an inhibitor is defined by a Nijmegen test > 0.6 BU in two consecutive tests conducted in the central lab. During the first 50 days of exposure to a single FVIII product, the following items will be evaluated: - FVIII inhibitor; - FVIII antibody titer, subclasses and FVIII-binding affinity; - FVIII-specific T-cell responses; - Total number of FOXP-3positive Treg; - RNA expression for the whole transcriptome; - F8 gene mutation and other known genomic predictors for inhibitor development.;Timepoint(s) of evaluation of this end point: The inhibitor FVIII will be evaluated during the following visits: day 1, day 5, day 10, day 20, day 30, day 40 and day 50. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints will be evaluated during the first 50 days of exposure to a single FVIII product. The items recorded in this period are: FVIII usage; bleeding episodes; occurrence of infection and/or immunization;Timepoint(s) of evaluation of this end point: At study completion (after 50 exposure days or a maximum of three years) | — |
Countries
Italy, United States
Contacts
Public ContactElena Santagostino, M.D.
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Haemophilia Unit
Outcome results
None listed