Long-term follow-up for people previously treated for cystinosis
Part of Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You previously took part in the CTNS-RD-04 study
- You must have received CTNS-RD-04 at some point before
- You are not currently in a CTNS-RD-04 treatment study
- If you finished CTNS-RD-04, withdrew, or left early, you may still qualify
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
This study gathers information from people with cystinosis to learn more about the condition. You may be able to join if you have a confirmed diagnosis.
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.
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.
This is a follow-up (no new treatment) study for people who previously received a gene therapy made from their own cells for ADA-SCID. It mainly tracks long-term safety and whether a marker showing the gene change can still be found.
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.
Hear when a new Cystinosis trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.