CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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New hope for rare disease: testing alternative to harsh current treatment
Disease control Recruiting nowThis early-stage study is testing a new oral medication called NPI-001 for people with cystinosis, a rare genetic disorder. It will compare NPI-001's safety and its ability to lower harmful cystine levels in the body to the current standard treatment, cysteamine. The study involv…
Matched conditions: CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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Decades-Long study tracks rare disease treatment
Disease control Recruiting nowThis study monitors people with cystinosis, a rare inherited disease that causes kidney failure and organ damage, who are taking the medication cysteamine. Researchers will follow 330 patients over many years to track how well the treatment works and watch for any new complicatio…
Matched conditions: CYSTINOSIS
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control
Last updated Mar 24, 2026 12:01 UTC
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Europe launches major study to track rare 'Crystal' disease
Knowledge-focused Recruiting nowThis study aims to create a detailed European registry of 400 people with cystinosis, a rare genetic disease where crystals damage organs. Researchers will collect health information over time to understand how the disease progresses and identify ways to improve patient care. The…
Matched conditions: CYSTINOSIS
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:12 UTC