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One-Shot gene therapy could free hemophilia b patients from frequent injections

NCT ID NCT06700096

Disease control Sponsor: Biocad Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial is testing a single dose of ANB-002, a gene therapy that delivers a working copy of the FIX gene, in 24 adults with hemophilia B. The goal is to see if it can reduce bleeding episodes compared to regular preventive factor IX infusions. Participants will be followed for up to 18 months after the infusion.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ANB-002 (arvenacogene sanparvovec) - a gene therapy that delivers a working FIX gene via a single infusion
What this could lead to
If successful, this could offer a long-term reduction in bleeding episodes and decrease the need for regular factor IX injections for people with hemophilia B.
What could go wrong
This is a small, early-phase trial with only 24 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or the treatment not lasting long-term.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2024

Expected to finish

Mar 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia B

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    14 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Almazov National Medical Research Centre

    RECRUITING

    Saint Petersburg, 197341, Russia

  • Chelyabinsk Regional Clinical Hospital

    RECRUITING

    Chelyabinsk, Russia

  • Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"

    RECRUITING

    Moscow, 125284, Russia

  • Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation

    RECRUITING

    Ufa, 450008, Russia

  • Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation

    RECRUITING

    Samara, 443099, Russia

    Contact Email: •••••@•••••

  • Federal State Budgetary Institution "National Medical Research Centre for Hematology" of the Ministry of Health of Russian Federation (Department of Clinical Diagnostics for Hematology and Hemostasis Disorders)

    RECRUITING

    Moscow, 125167, Russia

  • Federal State Budgetary Institution "National Medical Research Centre for Hematology" of the Ministry of Health of Russian Federation (Department of Traumatology and Reconstructive and Restorative Orthopedics for Patients with Hemophilia)

    RECRUITING

    Moscow, 125167, Russia

  • Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"

    RECRUITING

    Kirov, 610027, Russia

  • King Chulalongkorn Memorial Hospital

    NOT_YET_RECRUITING

    Bangkok, 10330, Thailand

  • Ramathibodi Hospital

    NOT_YET_RECRUITING

    Bangkok, 10400, Thailand

  • Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency

    RECRUITING

    Saint Petersburg, 193024, Russia

  • Siriraj Hospital

    NOT_YET_RECRUITING

    Bangkok, 10700, Thailand

  • State Novosibirsk Regional Clinical Hospital

    RECRUITING

    Novosibirsk, 630087, Russia

  • State budgetary healthcare institution Leningrad Regional Clinical Hospital

    RECRUITING

    Saint Petersburg, 188663, Russia

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