Pilot Study of TTI-0102 in Cystinosis

NCT07837726 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: TTI-CYST-001
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Nephropathic Cystinosis; Cystinosis

Outcome Measures

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.

  Comparison of baseline, Day 1-2, Day 3-4, and treatment end (Day 7)

Secondary Outcomes (6)

• Pharmacokinetic parameter: Cmax

  At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 7

• Pharmacokinetic parameter: Tmax

  At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 7

• Pharmacokinetic parameter: AUC

  At specified days/timepoints during the treatment period: Day1-2, Day 3-4, Day 7

• Pharmacodynamic biomarker: lactate in plasma

  Baseline (Day 0) to treatment end (Day 7)

• Pharmacodynamic biomarker: pyruvate in whole blood

  Baseline (Day 0) to treatment end (Day 7)

Study Arms (1)

TTI-0102 (cysteamine-pantetheine disulfide)

EXPERIMENTAL

Given once daily for 7 days

Drug: TTI-0102 (cysteamine-pantetheine disulfide)

Interventions

TTI-0102 (cysteamine-pantetheine disulfide) DRUG

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

TTI-0102 (cysteamine-pantetheine disulfide)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Able to provide informed consent in English.
• The first 3 patients must have WBC cystine levels over the last year no more than 50% greater than the upper limit of target level

You may not qualify if:

• 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).
• Any patient who is pregnant, plans to become pregnant or is unwilling to use contraceptive measures during study participation.

Sponsors & Collaborators

• Thiogenesis Therapeutics, Inc.: lead

MeSH Terms

Conditions

Cystinosis

Interventions

cysteamine-pantetheine disulfide

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Laurence A Greenbaum, MD, PhD, FAAP

  Emory University

  PRINCIPAL INVESTIGATOR

• Patrice P Rioux, MD, PhD

  Thiogenesis Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

Margo Kamel, PhD - Director of Research Projects

CONTACT

404-712-9923; mkamel@emory.edu

Kaiyang Zhu - Clinical Research Coordinator

CONTACT

404-727-2375; kzhu42@emory.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2026
• First Posted: September 23, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): March 1, 2027
• Last Updated: September 23, 2026

Record last verified: 2026-09