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

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.

Completed

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Key information

Conditions

Cystinosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Urogenital Diseases

Age range

1 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

cysteamine hydrochloride

Drug

Sponsors and collaborators

Lead sponsor

FDA Office of Orphan Products Development

Fed

Collaborators

Leadiant Biosciences, Inc.

Registry information

Important dates

Study start: 1999
Study completion: 2001
First posted: Feb 2, 2001
Registry last updated: Mar 25, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed