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

Primary Purpose

Cystinosis

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
cysteamine hydrochloride
Sponsored by
FDA Office of Orphan Products Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystinosis focused on measuring cystinosis, rare disease, renal and genitourinary disorders

Eligibility Criteria

1 Year - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 2, 2001
    Last Updated
    March 24, 2015
    Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Leadiant Biosciences, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00010426
    Brief Title
    Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2001
    Overall Recruitment Status
    Completed
    Study Start Date
    December 1999 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    February 2001 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Leadiant Biosciences, Inc.

    4. Oversight

    5. 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. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.
    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).

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystinosis
    Keywords
    cystinosis, rare disease, renal and genitourinary disorders

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Enrollment
    30 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    cysteamine hydrochloride

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    1 Year
    Maximum Age & Unit of Time
    50 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Edward F. Lemanowicz
    Organizational Affiliation
    Leadiant Biosciences, Inc.
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

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

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