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Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Study on AMT-191 for Fabry Disease: Safety and Effectiveness.

Recruiting
18-50 years
Male
Phase 1/2

This study looks at how safe and effective a new treatment called AMT-191 is for people with Classic Fabry Disease (FD). Fabry Disease is a rare illness caused by a missing enzyme called α-galactosidase A. This study gives a single dose of AMT-191 to help create this enzyme in the body. AMT-191 uses a special virus, called a viral vector, to deliver the treatment directly to the liver through an IV infusion (a needle in a vein).

  • The study needs men aged 18-50 with Classic Fabry Disease.
  • Participants will keep taking their usual enzyme replacement therapy (ERT) during the study.
  • Participants must agree to use condoms for up to 18 months to prevent transferring the treatment to others.

If you join, you will have to follow specific rules, such as no vaccinations around the time of treatment and no other experimental drugs. Risks include possible side effects from the treatment or its delivery method. Only consider joining if you meet the health conditions and requirements, and discuss it with your doctor.

Study details
    Fabry Disease

NCT06270316

UniQure Biopharma B.V.

1 March 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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