Status:

TERMINATED

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

Lead Sponsor:

Octapharma

Conditions:

Severe Hemophilia A

Eligibility:

MALE

18+ years

Phase:

PHASE1

PHASE2

Brief 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-c...

Eligibility Criteria

Inclusion

  • Severe hemophilia A (\<1% FVIII:C) as documented in medical records
  • Males ≥18 years of age
  • Subjects who have had ≥150 exposure days (EDs) with a FVIII product
  • Written informed consent for study participation obtained before undergoing any study specific procedures

Exclusion

  • Previous participation in this trial
  • Use of an Investigational Medicinal Product within 30 days prior to the first OCTA101 injection
  • History of FVIII inhibitors titre ≥0.6 BU/mL defined by medical records
  • Inhibitors to FVIII (≥0.6 BU/mL) at screening measured by Nijmegen modified Bethesda method at central laboratory
  • Human immunodeficiency virus (HIV) positive subjects with a CD4+ count \<200/mL
  • Clinically significant anemia at screening (hemoglobin \<8 g/dL)
  • 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
  • Any coagulation disorder other than hemophilia A
  • AST or ALT levels \>3 times the upper limit of normal
  • Creatinine \>120 μmol/L
  • Platelet count \<100,000 μL
  • BMI ≥30 kg/m²
  • For Cohort 6, patients with a positive LumiTope test at screening will be excluded

Key Trial Info

Start Date :

July 3 2019

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

February 18 2022

Estimated Enrollment :

36 Patients enrolled

Trial Details

Trial ID

NCT04046848

Start Date

July 3 2019

End Date

February 18 2022

Last Update

February 13 2025

Active Locations (1)

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1

Specialized Hospital for Active Treatment of Hematological Diseases EAD Clinic of Clinical Hematology

Sofia, Bulgaria