One-Shot gene therapy aims to free patients from lifelong injections

NCT ID NCT04323098

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

Summary

This study tested a single-dose gene therapy, called valoctocogene roxaparvovec, in 22 adult men with severe hemophilia A. The goal was to see if the therapy could safely help the liver produce its own blood-clotting factor (Factor VIII), reducing or eliminating the need for regular replacement injections. Researchers measured changes in clotting factor levels, bleeding rates, and quality of life over one year.

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Contacts and locations

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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

Conditions

Explore the condition pages connected to this study.

HEMOPHILIA A