Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ANB-002, dose 1, ANB-002, dose 2, ANB-002, dose 3.
- Who it may be relevant to
- Registry conditions: Hemophilia B. Basic parameters: from 18 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Belarus, Russia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B
Overview
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
Detailed description
The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.
In Cohort 2, the subjects are treated with a single dose of ANB-002 (dose 2). In Cohort 3, the subject are treated with a single dose of ANB-002 (dose 3).
The decision to continue enrolling in cohorts will be made at the Independent Data Monitoring Committee (IDMC) meeting. After the IDMC makes a decision regarding the dosing subjects, further enrolment to the cohort will be carried out.
Based on the data from the follow-up period of subjects included in Сohorts 1-3, a potential therapeutic dose for further study will be determined and additional patients will be included to recieve this dose.
In exploratory Cohort 4 patients with anti-AAV5 antibodies and/or hepatitis B in anamnesis will be included. These subjects will recieve the dose 3 of ANB-002.
The total duration of participation of one subjects in the study will be 5 years.
Interventions
- Genetic ANB-002, dose 1
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1. - Genetic ANB-002, dose 2
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2. - Genetic ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Primary outcome measures
- Change in FIX activity from baseline [Time frame: 12 months; final assessment - 5 years]
- Proportion of subjects with adverse reactions [Time frame: 12 months; final assessment - 5 years]
Secondary outcome measures (9)
- Changes in FIX activity [Time frame: 5 years]
- Annualized use of FIX concentrates [Time frame: 5 years]
- Annualized bleeding rate [Time frame: 5 years]
- Annualized rate of bleedings requiring therapy with FIX concentrates [Time frame: 5 years]
- Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline [Time frame: 5 years]
- Changes in EuroQol-5D-3L (European Quality of Life Questionnaire) scores from baseline [Time frame: 5 years]
- Changes in SF-36 (Short Form-36) scores from baseline [Time frame: 5 years]
- Response duration based on FIX activity [Time frame: 5 years]
- Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS) [Time frame: 5 years]
Eligibility criteria
Inclusion criteria
- Male with hemophilia B.
- Age ≥18 years.
- FIX activity at screening ≤2% without FIX inhibitor.
- ≥150 previous exposure days of treatment with FIX concentrates.
Exclusion criteria
- Previous gene therapy.
- Other blood or hematopoietic disorders.
- Positive Anti-AAV5 antibodies (for Cohorts 1-3).
- Diagnosed HIV-infection, not controlled with anti-viral therapy.
- Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
- Any active systemic infections or recurrent infections requiring systemic therapy at screening.
- Any other disorders associated with severe immunodeficiency.
- Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
- Malignancies with remission <5 years.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Russia · 15 centers
- State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital" — Chelyabinsk
- State budgetary healthcare institution Leningrad Regional Clinical Hospital — Gatchina
- Kuzbass Clinical Hospital named after S.V. Belyaev — Kemerovo
- Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology an — Kirov
- Federal State Budgetary Institution "National Medical Research Centre for Hematology" of t — Moscow
- Federal State Budgetary Institution "National Medical Research Centre for Hematology" of t — Moscow
- Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin" — Moscow
- LLC "Medis" — Nizhny Novgorod
- … and 7 more centers
Belarus · 2 centers
- Republican Scientific and Practical Center for Radiation Medicine and Human Ecology — Homyel
- Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology — Minsk
Identifiers
NCT: NCT06120582 · ANB-002-1