One-Time gene therapy shot aims to ease severe hemophilia a

NCT ID NCT03370172

Completed Disease control Sponsor: Baxalta now part of Shire Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This early-phase trial tested a gene therapy called BAX 888 in 4 men with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene for factor VIII, a blood-clotting protein they lack. Participants received a single intravenous infusion, and the study focused on safety and finding the right dose. The goal is to see if this one-time treatment can reduce or replace the need for regular factor VIII injections.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: BAX 888 (a gene therapy using a harmless virus to deliver a working factor VIII gene)
What this could lead to: If successful, this could lead to a one-time treatment that reduces or eliminates the need for regular factor VIII infusions for people with severe hemophilia A.
What could go wrong: This is a very early, small study (only 4 participants) focused on safety. The therapy may not work well, could cause side effects like immune reactions, and long-term effects are unknown.

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Conditions

The condition(s) this trial relates to.

hemophilia A severe hemophilia A

As listed by the trial registrant

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

HEMOPHILIA A

Contacts and locations

Locations

AKH - Medizinische Universität Wien

Vienna, 1090, Austria
CHU Rennes - Hopital Pontchaillou

Rennes, Ille Et Vilaine, 35000, France
CHU Tours - Hôpital Trousseau

Tours, Indre Et Loire, 37044, France
CHU de Nantes Site Hotel Dieu

Nantes, Loire Atlantique, 44093, France
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Groupement Hospitalier Est- Hôpital Louis Pradel

Bron, Rhone, 69677, France
Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, 77030-4009, United States
Hopital Jeanne de Flandre - CHU Lille

Lille, Nord, 59037, France
Hospital Clinico Universitario de Salamanca

Salamanca, 37007, Spain
Hospital Regional Universitario de Malaga

Málaga, 29010, Spain
Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain
Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain
Hospital Universitario La Paz

Madrid, 28046, Spain
Hôpital Bicêtre

Le Kremlin-Bicêtre, Val De Marne, 94275, France
Hôpital Morvan

Brest, Finistere, 29609, France
Hôpital de la Timone

Marseille, Bouches-du-Rhône, 13385, France
Klinikum der Johann Wolfgang Goethe-Universitaet

Frankfurt am Main, Hesse, 60590, Germany
Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States
Mount Sinai Medical Center

New York, New York, 10029, United States
Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center

Los Angeles, California, 90007, United States
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Semmelweis Egyetem

Budapest, 1083, Hungary
UC Davis Medical Center

Sacramento, California, 95817, United States
Universitaetsklinikum Carl Gustav Carus TU Dresden

Dresden, Saxony, 01307, Germany
University of Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, 80045, United States
Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

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