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Gene therapy breakthrough? new trial targets hemophilia b patients previously excluded

NCT ID NCT06003387

Disease control Sponsor: CSL Behring 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Aug 28, 2026 · Updated 6 times

Summary

This study tests a single-dose gene therapy called CSL222 (Etranacogene dezaparvovec) in 35 adults with severe or moderately severe hemophilia B who have detectable AAV5 antibodies—a group often excluded from gene therapy. The goal is to see if it reduces bleeding episodes and is safe. Participants receive one IV infusion and are monitored for side effects and bleeding rates.

What this could mean

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

Active substance
CSL222 (Etranacogene dezaparvovec) gene therapy
What this could lead to
If successful, this could offer a one-time gene therapy option for hemophilia B patients who currently have no gene therapy options due to pre-existing antibodies.
What could go wrong
This is an early Phase 3 study with only 35 participants, so results may not apply to everyone. The gene therapy may not work as expected in people with antibodies, and there are risks like liver issues or allergic reactions.

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 35 people

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

Started

Jan 2024

Expected to finish

Apr 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

Anyone

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: * Considered legally an adult, as defined by country regulations. * Has congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to \[\<=\] 2% of normal circulating FIX) for which the participant is on continuous routine FIX prophylaxis. * Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results). * Has greater than (\>) 150 previous exposure days to FIX replacement therapy. * Has been on stable FIX prophylaxis for at least 2 months before Screening. * Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator. * Acceptance to adhere to contraception guidelines. * Able to provide informed consent after receipt of verbal and written information about the study. * Investigator believes that the participant (or the participant's legally acceptable representative\[s\]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures. Exclusion Criteria: * History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results). * Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin \> 2 × the upper limit of normal (ULN) (except if caused by Gilbert's syndrome). * Screening or Visit L-Final laboratory values (based on central laboratory results) of any of the following laboratory abnormalities: 1. ALT \> 2 × the ULN 2. AST \> 2 × the ULN 3. Alkaline phosphatase \> 2 × the ULN 4. Serum creatinine \> 2 × the ULN 5. Hemoglobin less than (\<) 8 g/dL * Any condition other than hemophilia B resulting in an increased bleeding tendency. * Thrombocytopenia, defined as a platelet count \<50 × 10\^9/L, at Screening or Visit L Final (based on central laboratory results). * Any uncontrolled or untreated infection (human immunodeficiency virus \[HIV\], hepatitis B virus \[HBV\] and hepatitis C virus \[HCV\], or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder evaluated by the investigator to interfere with adherence to the clinical study protocol procedures or with the degree of tolerance to CSL222. * Known history of allergy to corticosteroids or known medical condition that would require chronic administration of oral corticosteroids. * Known uncontrolled allergic conditions or allergy / hypersensitivity to any component of the CSL222 excipients (ie, sucrose, potassium chloride, potassium dihydrogen phosphate, sodium chloride, and disodium hydrogen phosphate). * Previous AAV5 gene therapy treatment. * Receipt of an experimental agent or device within 60 days before Screening until the end of the study.

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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 places running it

    32 sites in 15 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

  • Centro de Investigacion Clinica GRAMEL S.C.

    RECRUITING

    Mexico City, Mexico City, 3720, Mexico

  • Changhua Christian Hospital (CCH)

    RECRUITING

    Chang-hua, 500, Taiwan

  • Ege University Medical Faculty

    RECRUITING

    Bornova, 35100, Turkey (Türkiye)

  • Gaziantep University Sahinbey Research and Practice Hospital

    RECRUITING

    Gaziantep, 27310, Turkey (Türkiye)

  • Haemophilia Comprehensive Care Centre

    RECRUITING

    Johannesburg, 2193, South Africa

  • Hemophilia Center of Western Pennsylvania (HCWP)

    RECRUITING

    Pittsburgh, Pennsylvania, 15260, United States

  • Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

    RECRUITING

    São Paulo, 05403-010, Brazil

  • Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)

    RECRUITING

    Kaohsiung City, Sanmin District, 80756, Taiwan

  • King Faisal Specialist Hospital and Research Center

    RECRUITING

    Riyadh, 11471, Saudi Arabia

  • Klinika Zaburzen Hemostazy i Chorob Wewnetrznych

    RECRUITING

    Warsaw, 02776, Poland

  • Kyung Hee University Hospital at Gangdong

    RECRUITING

    Seoul, 05278, South Korea

  • Kyungpook National University Hospital

    RECRUITING

    Daegu, 41944, South Korea

  • McMaster University - Hamilton

    RECRUITING

    Hamilton, Ontario, L8N 3Z5, Canada

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 100225, Taiwan

  • National University Hospital

    RECRUITING

    Singapore, 110974, Singapore

  • Prince of Wales Hospital Chinese University of Hong Kong

    RECRUITING

    Shatin, 999077, Hong Kong

  • Queen Mary Hospital

    RECRUITING

    Hong Kong, 999077, Hong Kong

  • Royal Adelaide Hospital

    RECRUITING

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane Hospital

    RECRUITING

    Herston, Queensland, 4029, Australia

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • Severance Hospital, Yonsei University Health System

    RECRUITING

    Seoul, 3722, South Korea

  • Sheba Medical Center

    RECRUITING

    Tel Litwinsky, 5265601, Israel

  • Singapore General Hospital

    RECRUITING

    Singapore, 169608, Singapore

  • Specialized Hospital for Active Treatment of Hematological Diseases

    RECRUITING

    Sofia, 1756, Bulgaria

  • Taichung Veterans General Hospital -

    RECRUITING

    Taichung, 40705, Taiwan

  • The Alfred Hospital

    RECRUITING

    Melbourne, Victoria, 3004, Australia

  • The University of Texas Health Science Center at Houston

    RECRUITING

    Houston, Texas, 77030, United States

  • Tri-Service General Hospital

    RECRUITING

    Taipei, Neihu District, 114, Taiwan

  • UNICAMP Universidade Estadual de Campinas

    RECRUITING

    São Paulo, 13083-878, Brazil

  • University of California, San Diego (UCSD)

    RECRUITING

    San Diego, California, 92121, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Özel Acibadem Adana Hastanesi

    RECRUITING

    Seyhan, 01130, Turkey (Türkiye)

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