NEPHROPATHIC CYSTINOSIS
Clinical trials for NEPHROPATHIC CYSTINOSIS explained in plain language.
Never miss a new study
Get alerted when new NEPHROPATHIC CYSTINOSIS trials appear
Sign up with your email to follow new studies for NEPHROPATHIC CYSTINOSIS, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Gene therapy may free kids from daily cystinosis meds
Disease control Recruiting nowThis study tests a gene therapy called DFT383 in 30 children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages kidneys and other organs. The treatment aims to fix the genetic problem so children may no longer need daily cysteamine medication. Researchers will …
Matched conditions: NEPHROPATHIC CYSTINOSIS
Phase: PHASE1, PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC
-
Scientists investigate why cystinosis causes early aging
Knowledge-focused Recruiting nowThis study looks at how energy production inside cells (mitochondria) works in people with cystinosis, a rare disease that causes cystine buildup and early aging. Researchers will measure cell energy activity in 25 patients and compare it to healthy people. The goal is to underst…
Matched conditions: NEPHROPATHIC CYSTINOSIS
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Aug 05, 2026 00:00 UTC