An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients with Hemophilia B (MAGNOLIA)

Biocad

Status and phase: Not yet enrolling; Phase 3

Conditions: Hemophilia B

Treatments: Genetic: ANB-002

Study type: Interventional

Funder types: Industry

Identifiers

NCT06700096

ANB-002-2

Details and patient eligibility

About

The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.

Full description

After signing the ICF and screening examination the subjects are to be included in the Non-interventional lead-in period in which the subject will receive standard FIX prevention. The lead-in period will last for at least 6 month for every subject.

After completiong the lead-in period subjects will enter the main (interventional) period.

At the first visit of the main period subjects will recieve investigational product ANB-002.

The main period ends 18 months after the administration of ANB-002, after which subjects will switch to the follow-up period and will be evaluated up to 5 years after the ANB-002 infusion.

Enrollment: 24 estimated patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Men diagnosed with hemophilia B aged 18 or older
• FIX activity ≤2%
• Absense of FIX inhibitor
• ≥150 previous exposure days of treatment with FIX concentrates

Exclusion criteria

• Any diseases of blood and hematopoietic organs other than hemophilia B
• A history of any gene therapy, including ANB-002
• Diagnosed HIV-infection, not controlled with anti-viral therapy
• Active HBV or HCV infection
• Anti-AAV5 antibodies
• Any active systemic infections or recurrent infections requiring systemic therapy
• Any other disorders associated with severe immunodeficiency
• Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder
• Malignancies with less than 5 years of remission

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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24 participants in 2 patient groups

Lead-in

No Intervention group

Description:

All subjects will be enrolled in the Lead-in period in order to collect information on individual bleeding rate anf FIX concentrates consumption

ANB-002

Experimental group

Description:

After completion of lead-in period subjects will enter the main period which starts with ANB-002 infusion.

Treatment:

Genetic: ANB-002