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

Not Applicable

Completed

• Conditions: Cystinosis

• Registration Number: NCT00010426
• Lead Sponsor: FDA Office of Orphan Products Development

Brief 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.

Detailed Description

PROTOCOL OUTLINE: This is a randomized, multicenter study Patients are randomized to receive the current formulation of cysteamine hydrochloride as drops in one eye and the new formulation of cysteamine hydrochloride as drops in the other eye.

Patients receive the two formulations of cysteamine hydrochloride in their assigned eyes every hour during waking hours daily for 6 months (safety study) or for 1 year (efficacy study).

Study Timeline

• Study Start: 1999-12
• Study Completion: 2001-02
• Study First Submitted QC: 2001-02-01
• Study First Submitted: 2001-02-02
• Study First Posted: 2001-02-02
• Status Verified: 2001-04
• Last Update Submitted: 2015-03-24
• Last Update Posted: 2015-03-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified