FABRY DISEASE
Clinical trials for FABRY DISEASE explained in plain language.
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Tracking treatment for rare genetic disease
Disease control ENROLLING_BY_INVITATIONThis study is observing how well enzyme replacement therapy works for people with Fabry disease in Russia. Researchers will follow 100 patients who are already receiving this standard treatment as part of their regular care. They will collect data on symptoms, kidney and heart fu…
Matched conditions: FABRY DISEASE
Sponsor: NPO Petrovax • Aim: Disease control
Last updated Apr 01, 2026 21:56 UTC
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New pill aims to shrink enlarged hearts in rare genetic disease
Disease control OngoingThis study is testing whether a daily oral medicine called venglustat can better control heart damage in adults with Fabry disease compared to current standard treatments. About 104 participants with an enlarged heart will take either venglustat or their usual therapy for 18 mont…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated Apr 01, 2026 21:56 UTC
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New hope for fabry patients with failing kidneys: drug safety study underway
Disease control OngoingThis study aims to check the safety and measure how the drug migalastat behaves in the body of people with Fabry disease who also have severe kidney damage or need dialysis. It will enroll about 14 adults who have a specific genetic type of Fabry disease that responds to this dru…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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Fabry disease treatment faces Long-Term safety check
Disease control OngoingThis study follows adults with Fabry disease who previously took lucerastat in an earlier trial. Researchers want to understand the long-term safety and side effects of continuing this oral medication. The study will monitor participants for up to 8 years or until the drug become…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Idorsia Pharmaceuticals Ltd. • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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Extended treatment trial offers hope for managing rare genetic disorder
Disease control OngoingThis study continues testing an enzyme replacement therapy called pegunigalsidase alfa in adults with Fabry disease who finished a previous trial. Researchers want to understand the long-term safety and effectiveness of receiving this medication through an IV every four weeks. Th…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC
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Can exercise ease the burden of a rare disease?
Symptom relief ENROLLING_BY_INVITATIONThis study is testing whether a 12-week physiotherapy and exercise program can help people with Anderson-Fabry disease feel better and function better. Fifteen participants will follow a structured program of aerobic and strength exercises three times a week. The goal is to see i…
Matched conditions: FABRY DISEASE
Phase: NA • Sponsor: General University Hospital, Prague • Aim: Symptom relief
Last updated Mar 31, 2026 12:11 UTC
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New drug trial targets debilitating pain of rare genetic disease
Symptom relief OngoingThis study is testing whether an investigational drug called venglustat can reduce nerve pain and abdominal pain in people with Fabry disease. It will involve about 122 participants aged 16 and older who are not currently on treatment. For 12 months, some will receive the drug an…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Sanofi • Aim: Symptom relief
Last updated Mar 30, 2026 14:32 UTC
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Massive study screens 100,000 babies for hidden rare diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to gather information by offering expanded newborn screening to 100,000 babies in New York. It will test for 14 rare genetic disorders to see how accurate the tests are, how often these diseases occur, and if finding them early helps children's health. The main go…
Matched conditions: FABRY DISEASE
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC