A Phase 2 interventional study of TTI-0102 (cysteamine-pantetheine disulfide) in Nephropathic Cystinosis and Cystinosis, sponsored by Thiogenesis Therapeutics, Inc.. Not yet recruiting. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-23.
Sponsored by Thiogenesis Therapeutics, Inc. · Phase 2, Interventional, and Treatment
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.
47 studies on the registry are indexed under Cystinosis; 15 are open to participants now.
This study's planned enrollment of 6 is below the median of 30 across 27 interventional studies indexed under Cystinosis.
Browse Cystinosis studies →Thiogenesis Therapeutics, Inc. is the lead sponsor of 4 studies on the registry; 2 are open to participants now.
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Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Given once daily for 7 days
Drug: TTI-0102 (cysteamine-pantetheine disulfide)
TTI-0102 (cysteamine-pantetheine disulfide) - a prodrug of other FDA-approved forms of cysteamine
White blood cell (WBC) cystine
The primary endpoint is the change in white blood cell (WBC) cystine levels following treatment with TTI-0102. The study will evaluate whether a weight-based dose of 60 ± 5 mg/kg/day maintains WBC cystine levels below 1 nmol ½ cystine/mg protein over 24 hours.
Time frame: Comparison of baseline, Day 1-2, Day 3-4, and treatment end (Day 7)
Pharmacokinetic parameter: Cmax
cysteamine in plasma
Time frame: At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 7
Pharmacokinetic parameter: Tmax
cysteamine in plasma
Time frame: At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 7
Pharmacokinetic parameter: AUC
cysteamine in plasma
Time frame: At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 7
Pharmacodynamic biomarker: lactate in plasma
Time frame: Baseline (Day 0) to treatment end (Day 7)
Pharmacodynamic biomarker: pyruvate in whole blood
Time frame: Baseline (Day 0) to treatment end (Day 7)
Pharmacodynamic biomarker: tryptophan in plasma
Time frame: Baseline (Day 0) to treatment end (Day 7)
No study locations are listed for this record.
Plan to share: Undecided
No publications or documents are linked to this record.
Start is estimated for Oct 2026; primary completion for Dec 2026, 2 months later.
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This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Cysteamine-pantetheine disulfide
Thiogenesis Therapeutics, Inc.