Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
Cystinosis
About this trial
This is an interventional treatment trial for Cystinosis focused on measuring cystinosis, rare disease, renal and genitourinary disorders
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of cystinosis with more than 2 nmole half-cystine/mg protein in leukocytes OR presence of corneal crystals consistent with cystinosis and distributed in corneal stroma observed by slit lamp biomicroscopy Clinical history consistent with cystinosis Safety study: History of adherence with current eye drop and follow-up schedule on protocol #86-El-0062 Any crystal density score, including zero, on photographs, that has been stable or improved over the past year Efficacy study: Crystal density score at least 1.00 on photographs Concurrently on cysteamine for at least the past 6 months --Prior/Concurrent Therapy-- No prior cysteamine drops (efficacy study) --Patient Characteristics-- Age: 1 to 50 (safety study) 2 to 12 (efficacy study) Other: Willingness and ability to tolerate corneal photographs