Severe Haemophilia A MedDRA version: 14.1 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850
Conditions
Interventions
Trade Name: ADVATE
Pharmaceutical Form: Concentrate and solvent for solution for infusion
INN or Proposed INN: OCTOCOG ALFA
CAS Number: 139076-62-3
Concentration unit: IU/mg international unit(s)/mill
Sponsors
St. James' Hospital
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Inclusion criteria: • Severe Haemophilia A (baseline Factor VIII level of =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Presence of a target joint on prophylaxis (defined as 3 bleeds into one joint, during a 6 month period, during the last year). • The occurrence of more than 3 haemarthroses in the last year which required more than 2 infusions to resolve. • Pregnant or breastfeeding women • Patients with a learning disability or dementia • Prisoners • Adults who are unconscious/unable to give informed consent • Participants with a pacemaker or implanted medical devices which are unsuitable to have a MRI will be excluded from the MRI scans during the trial but may proceed with other components.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare efficacy and safety of standard weight based Factor VIII prophylaxis with individually tailored pharmacokinetically based FVIII prophylaxis ;Secondary Objective: To compare the number of clinically significant bleeds and factor VIII usage in patients who are taking: a. Prophylaxis prescribed according to routine clinical practice with b. Prophylaxis prescribed on alternate days to maintain predicted target trough of =1.5IU/dL based on sparse blood sampling and Bayesian pharmacokinetic estimation 2) Compare the rate of all haemarthroses and soft tissue bleeds between the two regimens 3) To compare the number of clinically evident joint bleeds with any MRI joint changes and Pettersson scores 4) Establish the intra-patient variability of pharmacokinetic parameters over time by a population pharmacokinetic method 5) Establish the accuracy of factor VIII levels predicted from studies using sparse (2-3 blood sample) data, versus measured factor VIII levels in patients on prophylaxis and to investigate patient-related variables that affect factor VIII pharmacokinetics;Primary end point(s): Number of bleeding events experienced while on standard and Pk tailored prophylaxis;Timepoint(s) of evaluation of this end point: 6,18 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. The factor VIII usage 2. The total number of all haemarthroses and soft tissue bleeds 3. Quality of life (EQ5D) 4. Patients’ joint status (HJHS), MRI joint score (IPSG score ) and Pettersson score 5. The accuracy of factor VIII levels predicted from studies using sparse (2-3 blood sample) data, versus measured factor VIII levels in patients on prophylaxis 6. The intra-patient variability of pharmacokinetic parameters over time, as estimated by a population pharmacokinetic method ;Timepoint(s) of evaluation of this end point: 6,18 month | — |
Countries
Ireland
Contacts
Public ContactClinical Trials Information
National Centre for Hereditary Coagulation Disorders
Outcome results
None listed