Study of TTI-0102 for cystinosis
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new medicine called TTI-0102 for cystinosis. It aims to see if it can help manage cystine levels in the body, which may reduce kidney and other organ damage.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 65 years old.
- You have a confirmed diagnosis of cystinosis (with or without a kidney transplant).
- You are currently taking Cystagon or Procysbi at a stable dose for at least 3 months.
- Your weight is between 50 kg and 80 kg.
- You are able to attend study visits and have blood drawn over a 24-hour period.
- You are not allergic to cysteamine, pantetheine, or any of the study drug ingredients.
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 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 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.
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.
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.
This study is a follow-up registry for people with cystinosis to track their health over time. It helps doctors learn more about the condition and how it affects daily life, without giving any new treatments.
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.
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