One-Time gene therapy could free hemophilia patients from frequent infusions

NCT ID NCT06224907

Ongoing Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

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

Summary

This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to help their bodies produce their own clotting factor, reducing or stopping the need for regular factor VIII infusions. Participants will be monitored for safety and changes in bleeding episodes over about a year.

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Conditions

The condition(s) this trial relates to.

blood coagulation disease coagulation protein disease hematologic disorder hemophilia A hemorrhagic disease hereditary disease inherited blood coagulation disorder severe hemophilia A vascular hemostatic disease

As listed by the trial registrant

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

HEMOPHILIA A

Contacts and locations

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Locations

Asahikawa Medical University Hospital

Hokkaido, Asahikawa, 078-8510, Japan
Nagoya University Hospital

Aichi, Nagoya, 466-8560, Japan
Saitama Medical University Hospital

Saitama, Iruma-gun, 350-0495, Japan
Tokyo Medical University Hospital

Tokyo, Shinjuku-ku, 160-0023, Japan