Clinical trials
Cystinosis clinical trials
Below are recruiting cystinosis clinical trials, each written for real people, not researchers. We’re tracking 12 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - NCT00359684Recruiting
Cysteamine treatment for cystinosis
This trial looks at the use of cysteamine to treat cystinosis, a rare inherited condition. It may help lower harmful cystine buildup in the body and improve outcomes, especially early in life.
Bethesda, MarylandAges 1 week–115 years - NCT07319091Recruiting
Cystinosis and Mitochondrial Energy Study
This study looks at how cystinosis affects the energy centers (mitochondria) in your cells. It may help doctors understand how cysteamine treatment works in patients with kidney cystinosis.
BronAges 2 years+ - 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 - NCT05843851Recruiting
Newborn screening for rare cystinosis and hyperoxaluria
This study offers genetic screening to newborns to look for two rare metabolic conditions. It helps doctors find these conditions earlier, so treatment can start sooner if needed.
Hanover, Lower SaxonyAges 1 day–3 days - NCT05959668Recruiting
Questionnaire study for people with cystinosis in 4 languages
This study is testing a health-and-quality-of-life questionnaire for children and young adults with cystinosis. If you join, you may complete surveys and take part in interviews to help make the questions clearer and more useful.
HamburgAges 8–26 - NCT05901077Recruiting
European cystinosis patient follow-up study
This study follows people with confirmed cystinosis to better understand the condition. It mainly checks that you have the right diagnosis and can join voluntarily.
Paris, Île-de-France RegionAges Any age - NCT05146830Enrolling by invitation
Long-term follow-up for people previously treated for cystinosis
This study checks how people with cystinosis are doing long-term after finishing an earlier study medicine called CTNS-RD-04. It may help researchers understand the lasting effects and safety of that treatment over time.
La Jolla, CaliforniaAges 14–50 - 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+ - NCT05508009RecruitingPhase 1/Phase 2
Kidney transplant with donor-matched stem cell treatment
This early-phase trial tests a stem cell transplant from a donor who is an extremely close match, given to people who are about to receive a kidney transplant from the same donor. The goal is to improve how well the new kidney works by resetting the immune system to better accept the transplant.
Palo Alto, CaliforniaAges 1–30 - NCT06065852Recruiting
National registry for rare kidney diseases
This study creates a registry to collect health information from people with rare kidney diseases. It aims to improve understanding and future treatments by tracking patient experiences.
Bristol, South WestAges Any age
Hear when a new Cystinosis trial opens
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Common questions
- Are there clinical trials for cystinosis?
- Yes. Clin2 currently lists 12 recruiting 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 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 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.