Severe hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850
Conditions
Interventions
Product Name: OCTA101 (human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer)
Pharmaceutical Form: Powder and solution for solution for injection
INN or Proposed INN: B-domain de
Sponsors
Octapharma AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Patients who meet all of the following criteria are eligible for the study: 1. Severe hemophilia A (=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: Patients who meet any of the following criteria are not eligible for the study: 1. Previous participation in this trial 2. Use of an Investigational Medicinal Product within 30 days prior to the first OCTA101 injection 3. History of FVIII inhibitors titre =0.6 BU/mL defined by medical records 4. Inhibitors to FVIII (=0.6 BU/mL) at screening measured by Nijmegen modified Bethesda method at central laboratory 5. Human immunodeficiency virus (HIV) positive subjects with a CD4+ count 3 times the upper limit of normal 10. Creatinine >120 µmol/L 11. Platelet count <100,000 µL 12. BMI =30 kg/m² 13. For Cohort 6, patients with a positive LumiTope test at screening will be excluded.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: The secondary objectives of this study are to assess • the pharmacokinetics of FVIII:C after single sc injection of OCTA101 • the dose proportionality of FVIII:C after sc injections of different doses of OCTA101 • the bioavailability of sc OCTA101 compared to iv injection of Nuwiq • the pharmacokinetics of OCTA12 (recombinant human von Willebrand Factor fragment dimer) after single injection of OCTA101 • plasma trough and peak levels of FVIII:C after daily injections of OCTA101 • plasma trough and peak levels of OCTA12 after daily injections of OCTA101 • the efficacy of sc injections of OCTA101 in preventing bleeding episodes ;Timepoint(s) of evaluation of this end point: Adverse events, Dose Limiting Toxicity, and thromboembolic events and local injection site reactions will be evaluated throughout the study. Patient diaries are reviewed by the investigator at every hospital visit. Local injection site reactions will be evaluated after each injection. At the screening visit all patients will be tested for presence of FVIII inhibitors, and patients testing positive will be excluded from the trial. During the study, factor VIII inhibitor formation will be evaluated at defined time points, 2 to 9 times, depending on the cohort the patient is assigned to. ;Main Objective: The primary objective is to assess the safety of various doses of OCTA101 after subcutaneous (sc) injection. ;Primary end point(s): - Adverse events - Dose Limiting Toxicity - Thromboembolic events - Local injection site reactions - Inhibitor formation to FVIII | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Antibody formation to OCTA12 - LumiTope assay results - OCTA12 plasma levels during daily dosing - Routine lab tests compared to baseline - Vital signs compared to baseline - Physical examination results compared to baseline ;Timepoint(s) of evaluation of this end point: Physical examinations will be performed for patients in cohort 1 to 3 at the beginning of the study and at the end of the 3-month prophylactic treatment period. For cohort 6, physical examination will be performed at the beginning of the study and at the end of the 6-month prophylactic treatment period. Routine lab parameters and vital signs will be evaluated at defined time points, 8 to 24 times, depending on the cohort the patient is assigned to. OCTA12 levels during 3 months will be evaluated 10 times. Antibody formation to OCTA12 will be evaluated at defined time points, 2 to 9 times, depending on the cohort the patient is assigned to. | — |
Countries
Bulgaria
Contacts
Public ContactJohann Bichler
Octapharma AG
Outcome results
None listed