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One-Time gene shot could free teens from bleeding disorder

NCT ID NCT07080905

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 17, 2026 · Updated 6 times

Summary

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement therapy.

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 20 people

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

Started

Jul 2025

Expected to finish

Oct 2033

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

138 to 206 months

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: * Key Inclusion Criteria for the Lead-in Period: Assigned male sex at birth * Aged ≥138 months (11 years and 6 months) to less than (\<) 206 months (17 years and 2 months) at the time of informed consent / assent. * 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 has been on continuous FIX prophylaxis. * On stable continuous FIX prophylaxis for at least 2 months before Screening. * Minimum of 75 previous exposure days of treatment with FIX protein before Screening. * Additional Key Inclusion Criteria for the Treatment Period: Completed the Lead-in Period: minimum of 6 months (26 weeks) of lead-in data collected and eligibility has been confirmed. * Aged ≥ 12 to \< 18 years at the time of CSL222 treatment. Exclusion Criteria: * Key Exclusion Criteria for the Lead-in Period: History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results). * Screening laboratory values (based on central laboratory results): * Total bilirubin \> 2 × the upper limit of normal (ULN). * Alanine aminotransferase (ALT) \> 2 × the ULN. * Aspartate aminotransferase (AST) \> 2 × the ULN. * Alkaline phosphatase (ALP) \> 2 × the ULN. * Serum creatinine \> 2 × the ULN. * Hemoglobin \< 8 g/dL. * Any condition other than hemophilia B resulting in an increased bleeding tendency. * Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at screening (based on central laboratory results). * Any uncontrolled or untreated infection (human immunodeficiency virus, hepatitis C, etc) or any other significant concurrent, uncontrolled medical condition, as evaluated by the investigator, 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. * Additional Key Exclusion Criteria for the Treatment Period: Positive FIX inhibitor test at Visit L-Final (based on central laboratory results) * AAV5 NAb titer \> 1:900 as assessed at Visit LX (last visit before Visit L-Final). * Visit L-Final laboratory values (based on central laboratory results) of: * Total bilirubin \> 2 × the ULN * ALT \> 2 × the ULN. * AST \> 2 × the ULN. * ALP \> 2 × the ULN. * Serum creatinine \> 2 × the ULN. * Hemoglobin \< 8 g/dL. * Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at Visit L-Final (based on central laboratory results).

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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

    14 sites in 8 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

  • Arthur M. Blank Hospital - Children's Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • CHU Hopital de la Timone

    RECRUITING

    Marseille, 13009, France

  • Center for Inherited Blood Disorders

    RECRUITING

    Orange, California, 92868, United States

  • Chaim Sheba Medical Center

    RECRUITING

    Ramat Gan, 5262000, Israel

  • Hospital Sant Joan de Déu

    RECRUITING

    Barcelona, 08950, Spain

  • John Radcliffe Hospital - Oxford University Hospitals NHS

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

  • La Paz University Hospital

    RECRUITING

    Madrid, 28046, Spain

  • Medical University Vienna

    RECRUITING

    Vienna, 1090, Austria

  • Queensland Children's Hospital

    RECRUITING

    Brisbane, 4101, Australia

  • St Thomas Hospital

    RECRUITING

    London, SE1 7EH, United Kingdom

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

  • UZ Leuven - Centrum voor Moleculaire en Vasculaire Biologie

    RECRUITING

    Leuven, 3000, Belgium

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32611, United States

  • University of Michigan Medical Center

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

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