One-Time gene shot could free teens from bleeding disorder

NCT ID NCT07080905

Recruiting now Disease control Sponsor: CSL Behring Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

University of Florida - 84000399
RECRUITING

Gainesville, Florida, 32611, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
University of Michigan Medical Center - 84000491
RECRUITING

Ann Arbor, Michigan, 48109, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••