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

This study has been completed.
Sponsor:
Collaborator:
Sigma Tau Pharmaceuticals, Inc.
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00010426
First received: February 2, 2001
Last updated: June 23, 2005
Last verified: April 2001

February 2, 2001
June 23, 2005
December 1999
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Complete list of historical versions of study NCT00010426 on ClinicalTrials.gov Archive Site
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Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
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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.

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).

Interventional
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Primary Purpose: Treatment
Cystinosis
Drug: cysteamine hydrochloride
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
30
February 2001
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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
Both
1 Year to 50 Years
No
Contact information is only displayed when the study is recruiting subjects
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NCT00010426
199/15704, SIGMATAU-FDR001769
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FDA Office of Orphan Products Development
Sigma Tau Pharmaceuticals, Inc.
Study Chair: Edward F. Lemanowicz Sigma Tau Pharmaceuticals, Inc.
FDA Office of Orphan Products Development
April 2001

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP