CYSTINOSIS
Clinical trials for CYSTINOSIS explained in plain language.
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Hope for cystinosis: drug may slow damage, but cure still elusive
Disease control Recruiting nowThis study follows 330 people with cystinosis, a rare inherited disease that causes kidney failure and growth problems. The drug cysteamine helps lower harmful cystine buildup in cells. Researchers aim to track long-term effects, find new complications, and support genetic testin…
Matched conditions: CYSTINOSIS
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control
Last updated May 04, 2026 16:30 UTC
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New cystinosis drug shows promise in early trial
Disease control Recruiting nowThis study tests a new oral medicine called NPI-001 for people with cystinosis, a rare disease that causes cystine crystals to build up in the body. It compares NPI-001 to the current standard treatment, cysteamine, to see if it is safe and how well it controls cystine levels. Th…
Matched conditions: CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Apr 26, 2026 19:36 UTC
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Rare disease registry aims to improve cystinosis care across europe
Knowledge-focused Recruiting nowThis study is building a large European database of people with cystinosis, a rare genetic disease that causes cystine buildup in cells. Researchers will collect medical and quality-of-life data from about 400 participants to better understand the disease and improve patient care…
Matched conditions: CYSTINOSIS
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated May 06, 2026 16:03 UTC