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

Sponsor
FDA Office of Orphan Products Development (U.S. Fed)
Overall Status
Completed
CT.gov ID
NCT00010426
Collaborator
Leadiant Biosciences, Inc. (Industry)
30
Enrollment
14.1
Duration (Months)

Study Details

Study Description

Brief 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.

  1. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.
Condition or Disease Intervention/Treatment Phase
  • Drug: cysteamine hydrochloride
 N/A

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).

Study Design

Study Type:
Interventional
Primary Purpose:
Treatment
Study Start Date :
Dec 1, 1999
Study Completion Date :
Feb 1, 2001

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Year to 50 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Locations

    No locations specified.

    Sponsors and Collaborators

    • FDA Office of Orphan Products Development
    • Leadiant Biosciences, Inc.

    Investigators

    • Study Chair: Edward F. Lemanowicz, Leadiant Biosciences, Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00010426
    Other Study ID Numbers:
    • 199/15704
    • SIGMATAU-FDR001769
    First Posted:
    Feb 2, 2001
    Last Update Posted:
    Mar 25, 2015
    Last Verified:
    Apr 1, 2001
    Keywords provided by , ,
    cystinosis
    rare disease
    renal and genitourinary disorders
    Additional relevant MeSH terms:
    Cystinosis
    Cysteamine

    Study Results

    No Results Posted as of Mar 25, 2015