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

Cystinosis Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00010426
• Other study ID #: 199/15704
• Secondary ID: SIGMATAU-FDR0017
• Status: Completed
• Phase: N/A
• First received: February 2, 2001
• Last updated: March 24, 2015
• Start date: December 1999
• Est. completion date: February 2001

Study information

• Verified date: April 2001
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: February 2001
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 50 Years

Eligibility

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

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystinosis

Intervention

Drug:
• cysteamine hydrochloride

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
FDA Office of Orphan Products Development	Sigma Tau Pharmaceuticals, Inc.