Brief Title
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
Official Title
Phase 1/2 Study to Assess the Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Previously Treated Adult Patients With Severe Hemophilia A
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-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 undergo a 3-month prophylactic treatment with OCTA101, following an
initial 4 to 6-week run-in period with Human-cl rhFVIII.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Adverse Events
Secondary Outcome
Efficacy: Area under the concentration-time curve (AUC) of FVIII:C
Condition
Severe Hemophilia A
Intervention
OCTA101
Study Arms / Comparison Groups
Cohort 1
Description: 50 IU/kg (n=4): single-period investigation with a single sc dose of 50 IU/kg OCTA101 profiled up to 72 hours after dosing in adult male patients with severe hemophilia A. Following review of safety and tolerability data by Data Monitoring Committee, proceed with Cohort 2, alongside daily prophylactic dosing (40-60 IU/kg) for 3 months.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
36
Start Date
July 3, 2019
Completion Date
September 2022
Primary Completion Date
September 2022
Eligibility 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
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, (0)55 451 21 41, [email protected]
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT04046848
Organization ID
SubQ8-01
Responsible Party
Sponsor
Study Sponsor
Octapharma
Study Sponsor
, ,
Verification Date
January 2021