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