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NOT YET RECRUITING
NCT07837726
PHASE2

Pilot Study of TTI-0102 in Cystinosis

Sponsor: Thiogenesis Therapeutics, Inc.

View on ClinicalTrials.gov

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

Interventions

DRUG

TTI-0102 (cysteamine-pantetheine disulfide)

TTI-0102 (cysteamine-pantetheine disulfide) - a prodrug of other FDA-approved forms of cysteamine