Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

• Status: Completed
• Trial end date: 2001-02-01
• Target enrollment: 0
• Participant gender: All

Summary

OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation.

II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: FDA Office of Orphan Products Development
• Collaborators: Leadiant Biosciences, Inc.; Sigma Tau Pharmaceuticals, Inc.
• Treatments: Cysteamine

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