Hemophilia B: REGV131-LNP1265 Gene Therapy

We are studying a new gene therapy to see if it is safe and helps adults with Hemophilia B produce a vital clotting factor. The trial will also assess how it affects bleeding events.

>2 years≤5 visitsSafety phase (I)Efficacy phase (II)No PlaceboInvestigational MedicinesInjectionHematologyInternal Medicine

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Intrafusin

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Acetylcysteine
Acetylcysteine helps thin and loosen mucus to clear airways in respiratory conditions and treats acetaminophen (paracetamol) overdose.
Alanine
Alanine is an amino acid the body uses to build proteins and support energy production and blood sugar regulation.
Arginine
Arginine is an amino acid that helps the body produce nitric oxide to improve blood flow and support wound healing.

What experimental substances are being tested?

Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.

Regv131
Lnp1265
Adeno-associated Virus Vector Serotype 8

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Addex
Aminomel
Aminomix 1 Novum

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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Hospices Civils De Lyon
Clinical Hemostasis Unit
Bron, France
Centre Hospitalier Universitaire De Lille
Hemostasis and Transfusion Department
Loos, France
Assistance Publique Hopitaux De Paris
Adult Hematology Department
Montrouge, France
Sponsor: Regeneron Pharmaceuticals Inc.
Last updated: Nov 26, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.