One-Time gene therapy shows promise for hemophilia a

NCT ID NCT04323098

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

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

Summary

This study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The treatment aims to help the body produce its own clotting factor VIII, reducing the need for regular infusions. Participants also received steroids to manage immune reactions. Results showed increased factor VIII levels and less need for replacement therapy.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HEMOPHILIA A are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Locations

Alfred Hospital

Melbourne, Australia
Campinas University Clinical Hospital

Campinas, Brazil
Changhua Christian Medical Foundation Changhua Christian Hospital

Changhua, Taiwan
Fiona Stanley Hospital

Perth, Australia
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

São Paulo, Brazil
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Royal Brisbane and Women's Hospital

Brisbane, Australia
Royal Prince Alfred Hospital

Sydney, Australia
Taichung Veterans General Hospital

Taichung, Taiwan
The Royal Adelaide Hospital

Adelaide, Australia
University of California Davis Health

Sacramento, California, 95817, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.