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Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Primary Purpose

Erythropoietic Protoporphyria

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
cysteine 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 Erythropoietic Protoporphyria focused on measuring erythropoietic protoporphyria, inborn errors of metabolism, porphyria, rare disease

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Documented erythropoietic protoporphyria Determination of elevated protoporphyrin within the past year Experiencing photosensitivity --Prior/Concurrent Therapy-- No concurrent use of betacarotene --Patient Characteristics-- Other: Not pregnant or nursing Fertile female patients must use effective contraception while on study and for 3 weeks thereafter

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 24, 2000
    Last Updated
    March 24, 2015
    Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    St. Luke's-Roosevelt Hospital Center
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004831
    Brief Title
    Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 1998
    Overall Recruitment Status
    Completed
    Study Start Date
    October 1996 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    July 1998 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    St. Luke's-Roosevelt Hospital Center

    4. Oversight

    5. Study Description

    Brief Summary
    OBJECTIVES: I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.
    Detailed Description
    PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, crossover study. Patients are randomly assigned to 1 of 2 groups to receive cysteine hydrochloride orally twice daily, 2 capsules with breakfast and 2 with lunch. Group 1 receives cysteine hydrochloride in drug ingestion period 1 followed by placebo in period 2. Group 2 receives placebo in period 1 followed by cysteine hydrochloride in period 2. Both groups ingest placebo for 1 week between the periods. Each drug ingestion period lasts 8 weeks. Follow up phone calls are made at the end of months 1 and 3. All patients schedule follow up visits at the end of each drug ingestion period.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Erythropoietic Protoporphyria
    Keywords
    erythropoietic protoporphyria, inborn errors of metabolism, porphyria, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Interventional Study Model
    Crossover Assignment
    Masking
    Double
    Allocation
    Randomized
    Enrollment
    20 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    cysteine hydrochloride

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Documented erythropoietic protoporphyria Determination of elevated protoporphyrin within the past year Experiencing photosensitivity --Prior/Concurrent Therapy-- No concurrent use of betacarotene --Patient Characteristics-- Other: Not pregnant or nursing Fertile female patients must use effective contraception while on study and for 3 weeks thereafter
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Micheline M Mathews-Roth
    Organizational Affiliation
    St. Luke's-Roosevelt Hospital Center
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

    Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

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