Sponsor or institution

Clinical trials and research at Amicus Therapeutics

• 5Clinical trials
• 15Countries

Newest trials

All recruiting trials (5)

• Long-term outcomes for adults with Fabry disease on migalastat

  This project will see if migalastat (compared with enzyme replacement therapy or no treatment) helps adults with Fabry disease maintain kidney function, stay safe, and improve quality of life over several years.

  Recruiting at 7 sites Amicus Therapeutics · Birmingham, Alabama and 7 other locations · NCT06906367

• Migalastat for children aged 2 to <12 with Fabry disease and amenable GLA variants

  This test gives the oral drug migalastat to children aged 2 to <12 who have Fabry disease with GLA variants that respond to the drug to see if it is safe and how it acts in their bodies.

  Recruiting at 11 sites Phase 3 Amicus Therapeutics · Atlanta, Georgia and 11 other locations · NCT06904261

• Global registry for patients with Pompe disease

  This study is collecting information from people with Pompe disease to see how well current treatments work and how they affect daily life, including both those who are being treated and those who aren't.

  Recruiting at 29 sites Amicus Therapeutics · Little Rock, Arkansas and 40 other locations · NCT06121011

• Evaluating a new treatment for infantile onset glycogen storage disease

  This study is testing a new treatment for infants and young children with infantile onset Pompe disease to see if it is safe and effective.

  Recruiting at 13 sites Phase 3 Amicus Therapeutics · Gainesville, Florida and 13 other locations · NCT04808505

• Observational study of women with Fabry disease during pregnancy and breastfeeding

  This study looks at how pregnancy and breastfeeding affect women with Fabry disease and their babies, comparing those who have taken a treatment called migalastat to those who haven't.

  Recruiting Amicus Therapeutics · Philadelphia, Pennsylvania · NCT04252066

Quick answers

How many clinical trials run by Amicus Therapeutics are recruiting right now?

5, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 15 countries. The most sites are in United States (5 trials), Germany (3 trials), United Kingdom (3 trials), Belgium (2 trials), Italy (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 2. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

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