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

This study has been completed.
Sigma Tau Pharmaceuticals, Inc.
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
First received: February 2, 2001
Last updated: March 24, 2015
Last verified: April 2001

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
Drug: cysteamine hydrochloride

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

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).


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


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

Sponsors and Collaborators
FDA Office of Orphan Products Development
Sigma Tau Pharmaceuticals, Inc.
Study Chair: Edward F. Lemanowicz Sigma Tau Pharmaceuticals, Inc.
  More Information

ClinicalTrials.gov Identifier: NCT00010426     History of Changes
Other Study ID Numbers: 199/15704  SIGMATAU-FDR001769 
Study First Received: February 2, 2001
Last Updated: March 24, 2015
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
rare disease
renal and genitourinary disorders

Additional relevant MeSH terms:
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on May 25, 2016