Pilot Study of TTI-0102 in Cystinosis

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.