Pilot Study of TTI-0102 in Cystinosis
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorThiogenesis Therapeutics, Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adults aged 18-65 years with a confirmed diagnosis of cystinosis based on clinical features and/or genetic testing, with or without history of kidney transplantation.
Currently treated with a stable dose of Cystagon® or Procysbi® for at least 3 months prior to enrollment.
Body weight between 50 kg and 80 kg at screening.
Able to attend all required study visits and comply with study procedures, including blood draws over a 24-hour period.
Disqualifiers
Known hypersensitivity or allergy to cysteamine, pantetheine, TTI-0102, or any excipients in the study drug.
Clinically significant uncontrolled medical conditions (e.g., unstable cardiac, hepatic, or renal disease) that, in the investigator's judgment, would increase risk or interfere with study participation.
Participation in another interventional clinical trial within 30 days prior to screening.
Any condition that, in the investigator's opinion, would make the participant an unsuitable candidate (e.g., inability to comply with procedures, significant cognitive impairment, active substance use disorder).
Trial design
Design model
Single group
Treatments tested in this trial
TTI-0102 (cysteamine-pantetheine disulfide)
DrugTTI-0102 (cysteamine-pantetheine disulfide) - a prodrug of other FDA-approved forms of cysteamine
Treatment groups
6 Participants
are divided into 1 treatment groupGroup A: TTI-0102 (cysteamine-pantetheine disulfide)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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)Secondary outcomes
1
Pharmacokinetic parameter: Cmax
cysteamine in plasma
Time frame
At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 72
Pharmacokinetic parameter: Tmax
cysteamine in plasma
Time frame
At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 73
Pharmacokinetic parameter: AUC
cysteamine in plasma
Time frame
At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 74
Pharmacodynamic biomarker: lactate in plasma
Time frame
Baseline (Day 0) to treatment end (Day 7)Locations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: