Clinical trials · Pittsburgh, PA
Fabry Disease clinical trials in Pittsburgh
We’re tracking 5 recruiting fabry disease studies with a site in or near Pittsburgh — including Phase 1/Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Pittsburgh
- NCT06270316RecruitingPhase 1/Phase 2
Gene therapy for classic Fabry disease
This trial tests a new gene therapy called AMT-191 for men with classic Fabry disease who still have symptoms despite standard enzyme replacement therapy. It aims to see if the treatment is safe and can improve symptoms.
Pittsburgh, PennsylvaniaAges 18–50 - NCT06904261RecruitingPhase 3
Study of migalastat for children with Fabry disease
This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.
Pittsburgh, PennsylvaniaAges 2–11 - NCT06906367Recruiting
Long-term study of Fabry disease treatments in the US
This study looks at how well available Fabry disease treatments (like migalastat or ERT) work over time in real life. It may help doctors understand who benefits most from these medicines.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT06858397RecruitingPhase 1/Phase 2
Study of a new drug for Fabry disease
This study tests a new investigational drug called HM15421/GC1134A for people with Fabry disease. It aims to see if the drug is safe and tolerable, and to learn how well it works. You may be eligible if you have Fabry disease with symptoms, have not had treatment for at least 6 months, and your kidneys are functioning well.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT00196742Recruiting
Fabry disease registry and pregnancy follow-up
This study collects health information from people with Fabry disease and specifically from pregnant people with Fabry disease. It helps researchers better understand the condition and how pregnancy affects health, using information you already experience in routine care.
Pittsburgh, PennsylvaniaAges Any age
Hear when a new Fabry Disease trial opens near Pittsburgh, PA
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Fabry Disease trials in other cities
Where these studies are running near Pittsburgh
Institutions with a site for the recruiting fabry disease studies listed above.
- UPMC Children's Hospital of Pittsburgh · 4 studies
- University of Pittsburgh Medical Center
What taking part in a fabry disease study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Looking wider? See all recruiting fabry disease studies in Pennsylvania.
Common questions
- Are there fabry disease clinical trials near Pittsburgh?
- Yes. We're currently tracking 5 recruiting fabry disease studies with a site near Pittsburgh, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a fabry disease study near Pittsburgh?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pittsburgh to take part?
- Not necessarily. These studies have a site near Pittsburgh, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a fabry disease trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New fabry disease studies open near Pittsburgh regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.