Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A

• Status: Active, not recruiting
• Trial end date: 2022-09-01
• Target enrollment: 0
• Participant gender: Male

Summary

This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer) in previously treated adult patients with severe hemophilia A. The study also aims to assess the pharmacokinetics (PK) characteristics, dose proportionality, and subcutaneous bioavailability of OCTA101 compared with intravenous administration of Nuwiq (Human-cl rh FVIII), in order to define the prophylactic treatment (dose and injection interval) that would result in protective trough levels of FVIII:C for future Phase 3 studies. Cohorts 1, 2, 3 and 5 will undergo a single injection of OCTA101, with cohorts 1, 2 and 3 proceeding to 3-month daily dosing prophylactic treatment for 3 months by Data Monitoring Committee recommendation. Cohorts 1 and 2 will undergo a further PK at the end of the daily injection period. A further cohort, cohort 6, will have an initial 4 to 6-week run-in treatment period with Nuwiq intravenous prophylaxis followed by 12.5 IU/kg OCTA101 subcutaneous daily prophylaxis for >3 up to 6-7 months.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Octapharma
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

1. Severe hemophilia A (<1% FVIII:C) as documented in medical records

2. Males ≥18 years of age

3. Subjects who have had ≥150 exposure days (EDs) with a FVIII product

4. Written informed consent for study participation obtained before undergoing any study specific procedures

Exclusion Criteria:

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 <200/mL

6. Clinically significant anemia at screening (hemoglobin <8 g/dL)

7. Presence of any significant comorbidity (at the discretion of the investigator) that might confound the interpretation of the study data and/or that might put the patient at undue risk by participating in the trial

8. Any coagulation disorder other than hemophilia A

9. AST or ALT levels >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