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Pilot Study of TTI-0102 in Cystinosis
Sponsor: Thiogenesis Therapeutics, Inc.
Summary
White blood cell (WBC) cystine levels serve as the primary therapeutic target for monitoring cysteamine treatment in cystinosis, with a goal of \<1.9 nmol ½ cystine/mg protein as measured 5-6 hours post-dose when treated with immediate-release formulation, Cystagon® , or 11-12 hours post-dose when treated with delayed-release formulation, Procysbi®, using UCSD granulocyte assay. Although cysteamine dosing in cystinosis has traditionally been individualized based on WBC cystine levels, the FDA-approved Cystagon® prescribing information now includes an approximate recommended maintenance dose of 1.30 g/m²/day. When this dosing strategy is plotted against body weight, it aligns with the dosing regimen of TTI-0102 that was found effective in mitochondrial diseases, demonstrating a linear relationship between dose and weight. The main objective of this study is to demonstrate that administration of a single dose of 60 ± 5 mg/kg/day of TTI-0102 (\~26 mg/kg cysteamine base equivalent) allows maintenance of WBC cystine at \<1.9 nmol ½ cystine/mg protein over 24 hours as determined by UCSD granulocyte assay.
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
6
Start Date
2026-10
Completion Date
2027-03
Last Updated
2026-09-23
Healthy Volunteers
No
Conditions
Interventions
TTI-0102 (cysteamine-pantetheine disulfide)
TTI-0102 (cysteamine-pantetheine disulfide) - a prodrug of other FDA-approved forms of cysteamine