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

This study has been completed.

First Received on February 2, 2001. Last Updated on June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	Sigma Tau Pharmaceuticals, Inc.
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00010426

Purpose

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.

Condition	Intervention
Cystinosis	Drug: cysteamine hydrochloride

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: cystinosis Schindler disease

Drug Information available for: Cysteamine Cysteamine bitartrate

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	30
Study Start Date:	December 1999
Estimated Study Completion Date:	February 2001

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).

Eligibility

Ages Eligible for Study:	1 Year to 50 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00010426

Sponsors and Collaborators

FDA Office of Orphan Products Development

Sigma Tau Pharmaceuticals, Inc.

Investigators

Study Chair:

Edward F. Lemanowicz

Sigma Tau Pharmaceuticals, Inc.

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00010426 History of Changes
Other Study ID Numbers:	199/15704, SIGMATAU-FDR001769
Study First Received:	February 2, 2001
Last Updated:	June 23, 2005
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

cystinosis
rare disease
renal and genitourinary disorders

Additional relevant MeSH terms:

Cystinosis
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

Cysteamine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 09, 2012