Comparing the effects of efanesoctocog alfa and other treatments in severe Hemophilia A
An Open-label, Multicentre Study to Compare the Pharmacokinetics of Efanesoctocog Alfa Versus rFVIII Products, Damactocog Alfa Pegol or Turoctocog Alfa Pegol, After a Single Intravenous Dose of 50 IU/kg in a Fixed Sequence in Previously Treated Adults With Severe Haemophilia A.
This study is testing how long a new treatment for severe Hemophilia A lasts in the body compared to other current treatments in men aged 18-65.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | Male |
| Sponsor | Swedish Orphan Biovitrum Industry-sponsored |
| Locations | 8 sites (Sofia and 7 other locations) |
| Trial ID | NCT06579144 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the pharmacokinetic profiles of efanesoctocog alfa compared to other extended half-life recombinant FVIII products in males aged 18-65 with severe Hemophilia A. Participants will receive a single intravenous injection of their current treatment followed by efanesoctocog alfa after a wash-out period. The study involves multiple visits for blood sampling to assess how the body processes these treatments. The goal is to understand the differences in how long these medications remain effective in the body.
Who should consider this trial
Good fit: Ideal candidates are males aged 18-65 with severe Hemophilia A who have previously been treated with specific FVIII products.
Not a fit: Patients with a history of positive inhibitor tests may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved treatment options for patients with severe Hemophilia A, enhancing their quality of life.
How similar studies have performed: Other studies have shown success with similar pharmacokinetic comparisons, indicating a promising approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participant must be male, 18 to 65 years of age, inclusive, at the time of signing the informed consent form (ICF). * Severe haemophilia A, defined as \<1 IU/dL (\<1%) endogenous FVIII activity, as documented in historical medical records from a clinical laboratory demonstrating \<1% FVIII coagulant activity or a documented genotype known to produce severe haemophilia A. * Previous treatment for haemophilia A with any marketed recombinant and/or plasma derived FVIII for at least 150 exposure days. * Currently receiving treatment with damoctocog alfa pegol or turoctocog alfa pegol at Screening. Exclusion Criteria: * Any history of a positive inhibitor test, defined as \>0.6 Bethesda units (BU)/mL in at least two consecutive Bethesda inhibitor assays, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL. Family history of inhibitors will not exclude the participant. * Positive FVIII inhibitor result (assessed by central laboratory), defined as ≥0.6 BU/mL at Screening.
Where this trial is running
Sofia and 7 other locations
- Sobi Investigational Site — Sofia, Bulgaria (Recruiting)
- Sobi Investigational site — Frankfurt, Germany (Recruiting)
- Sobi Investigational Site — Oldenburg, Germany (Recruiting)
- Sobi Investigational Site — Milan, Italy (Recruiting)
- Sobi Investigational Site — Naples, Italy (Not_yet_recruiting)
- Sobi Investigational Site — A Coruña, Spain (Recruiting)
- Sobi Investigational Site — Valencia, Spain (Recruiting)
- Sobi Investigational Site — Zaragoza, Spain (Recruiting)
Study contacts
- Study coordinator: Blank Clinical Study Physician, MD PhD
- Email: medical.info@sobi.com
- Phone: +46 8 697 20 00
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.