Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

Plain-language summary

SAFRAN is a multicenter, two-stage, open-label Phase 1/2 study investigating the safety, immunogenicity and efficacy of ANB-002 (arvenacogene sanparvovec) in people with hemophilia B, using a dose-escalation design across three dose levels with seamless adaptive elements. Eligible participants are men aged 18 or older with FIX activity of 2% or below, no FIX inhibitor, and at least 150 previous exposure days to FIX concentrates. Prior gene therapy, other blood or hematopoietic disorders, and positive anti-AAV5 antibodies are exclusions.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

Biocad

Intervention

ANB-002, dose 1 (arvenacogene sanparvovec), ANB-002, dose 2 (arvenacogene sanparvovec), ANB-002, dose 3 (arvenacogene sanparvovec)

Start date

5/2/2023

Estimated completion

11/1/2029

Countries

Belarus, Russia

Related genes

Locations

• Homyel, Belarus
• Minsk, Belarus
• Chelyabinsk, Russia
• Gatchina, Russia
• Kemerovo, Russia
• Kirov, Russia
• Moscow, Russia
• Moscow, Russia
• Moscow, Russia
• Nizhny Novgorod, Russia
• Novosibirsk, Russia
• Saint Petersburg, Russia
• Saint Petersburg, Russia
• Saint Petersburg, Russia
• Samara, Russia
• Syktyvkar, Russia
• Ufa, Russia

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. Male with hemophilia B.
2. Age ≥18 years.
3. FIX activity at screening ≤2% without FIX inhibitor.
4. ≥150 previous exposure days of treatment with FIX concentrates.

Exclusion Criteria:

1. Previous gene therapy.
2. Other blood or hematopoietic disorders.
3. Positive Anti-AAV5 antibodies (for Cohorts 1-3).
4. Diagnosed HIV-infection, not controlled with anti-viral therapy.
5. Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
6. Any active systemic infections or recurrent infections requiring systemic therapy at screening.
7. Any other disorders associated with severe immunodeficiency.
8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
9. Malignancies with remission \<5 years.