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Not Yet Recruiting NCT07837726

Pilot Study of TTI-0102 in Cystinosis

Conditions: Nephropathic Cystinosis, Cystinosis

Sex: All
Ages: 18 Years – 65 Years
Healthy volunteers: No
Phase: PHASE2
Enrollment: 6
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 \

Eligibility Criteria

Inclusion Criteria: * 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 Exclusion Criteria: * 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.

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07837726). StuddyBuddy aggregates publicly available trial information.