Clinical trials
Nephropathic Cystinosis clinical trials
Below are recruiting nephropathic cystinosis clinical trials, each written for real people, not researchers. We’re tracking 4 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06910813RecruitingPhase 1/Phase 2
Gene therapy trial for children with cystinosis
This trial tests a new gene therapy called DFT383 for children aged 2 to 5 with nephropathic cystinosis. It aims to see if the treatment is safe and can help improve kidney function.
Phoenix, ArizonaAges 2–5 - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT04246060Enrolling by invitation
Quality-of-life study for people with cystinosis
This is an observational study that asks people with nephropathic cystinosis about their quality of life while taking cystine-depleting treatment (cysteamine). It may help researchers better understand how this condition and treatment affect daily living.
AntwerpAges Any age - NCT03919981Recruiting
Testing Cysteamine for cystinosis bone health
This study looks at how cysteamine affects the bone health of people with nephropathic cystinosis, a rare genetic condition. It includes children and adults who are already taking cysteamine by mouth and will follow the study plan closely.
BesançonAges 2 years+
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Common questions
- Are there clinical trials for nephropathic cystinosis?
- Yes. Clin2 currently lists 4 recruiting nephropathic cystinosis studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a nephropathic cystinosis trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a nephropathic cystinosis trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.