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Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Sponsor
FDA Office of Orphan Products Development (U.S. Fed)
Overall Status
Completed
CT.gov ID
NCT00004831
Collaborator
St. Luke's-Roosevelt Hospital Center (Other)
20
Enrollment
21
Duration (Months)

Study Details

Study Description

Brief Summary

OBJECTIVES:
  1. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.
Condition or Disease Intervention/Treatment Phase
  • Drug: cysteine hydrochloride
 N/A

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.

Study Design

Study Type:
Interventional
Allocation:
Randomized
Intervention Model:
Crossover Assignment
Masking:
Double
Primary Purpose:
Treatment
Study Start Date :
Oct 1, 1996
Study Completion Date :
Jul 1, 1998

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 65 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • FDA Office of Orphan Products Development
    • St. Luke's-Roosevelt Hospital Center

    Investigators

    • Study Chair: Micheline M Mathews-Roth, St. Luke's-Roosevelt Hospital Center

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00004831
    Other Study ID Numbers:
    • 199/13413
    • BWH-FDR000996-EF
    • SLRH-CU-FDR000996-EF
    First Posted:
    Feb 25, 2000
    Last Update Posted:
    Mar 25, 2015
    Last Verified:
    Aug 1, 1998
    Keywords provided by , ,
    erythropoietic protoporphyria
    inborn errors of metabolism
    porphyria
    rare disease
    Additional relevant MeSH terms:
    Protoporphyria, Erythropoietic

    Study Results

    No Results Posted as of Mar 25, 2015