Haemophilia A
Conditions
Brief summary
The primary endpoint in Group 1 and Group 2 is to evaluate ITI outcome as determined by achievement of the following ITI criteria: 1. Inhibitor titre < 0.6 BU/mL for at least 2 consecutive measurements 2. FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg BW 3. FVIII half-life ≥ 6 h, The primary endpoint for Group 3 is to evaluate the ABR compared with the ABR in Group 1 and Group 2.
Detailed description
For participants in Group 1 and Group 2: 1. Time to achieve ITI outcome 2. Use of emicizumab, aPCC, rFVIIa during ITI 3. Rate of FVIII inhibitor relapses during a follow-up period in participants who have achieved complete ITI success, For participants in all 3 groups: 1. Frequency and severity of all bleeding episodes (BEs), all treated BEs, all spontaneous BEs, all joint BEs, and target joint BEs over time (≥ 3 bleeds in the same joint within 24 weeks) 2. Number of infusions required to control BEs 3. Frequency and severity of bleeding during and after surgical procedures, For participants in all 3 groups: 4. Proportion of participants experiencing ADRs 5. Thrombotic events (location, treatment, outcome) 6. Treatment costs
Interventions
DRUGOCTANATE 1000 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
DRUGFEIBA 1000 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung
DRUGWilate 1000
DRUG1000 I.E. VWF/1000 I.E. FVIII
DRUGPulver und Lösungsmittel zur Herstellung einer intravenösen Injektionslösung.
DRUGCEVENFACTA 1 mg (45 KIU) powder and solvent for solution for injection
DRUGCEVENFACTA 5 mg (225 KIU) powder and solvent for solution for injection
DRUGOCTANATE 500 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
DRUGFEIBA 500 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung.
DRUGOCTANATE 250 Pulver und Lösungsmittel zur Herstellung einer Injektionslösung
DRUGCEVENFACTA 2 mg (90 KIU) powder and solvent for solution for injection
DRUGFEIBA 2500 E Pulver und Lösungsmittel zur Herstellung einer Infusionslösung.
DRUGWilate 500
DRUG500 I.E. VWF/500 I.E. FVIII
Sponsors
HZRM Haemophilie-Zentrum Rhein Main GmbH, Emory University
Eligibility
Sex/Gender
Male
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint in Group 1 and Group 2 is to evaluate ITI outcome as determined by achievement of the following ITI criteria: 1. Inhibitor titre < 0.6 BU/mL for at least 2 consecutive measurements 2. FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg BW 3. FVIII half-life ≥ 6 h, The primary endpoint for Group 3 is to evaluate the ABR compared with the ABR in Group 1 and Group 2. | — |
Secondary
| Measure | Time frame |
|---|---|
| For participants in Group 1 and Group 2: 1. Time to achieve ITI outcome 2. Use of emicizumab, aPCC, rFVIIa during ITI 3. Rate of FVIII inhibitor relapses during a follow-up period in participants who have achieved complete ITI success, For participants in all 3 groups: 1. Frequency and severity of all bleeding episodes (BEs), all treated BEs, all spontaneous BEs, all joint BEs, and target joint BEs over time (≥ 3 bleeds in the same joint within 24 weeks) 2. Number of infusions required to control BEs 3. Frequency and severity of bleeding during and after surgical procedures, For participants in all 3 groups: 4. Proportion of participants experiencing ADRs 5. Thrombotic events (location, treatment, outcome) 6. Treatment costs | — |
Countries
Bulgaria, Croatia, Finland, Germany, Norway, Spain, Sweden
Outcome results
None listed