Back to resultsStudy of Cysteine Hydrochloride for Erythropoietic Protoporphyria
Primary Purpose
Erythropoietic Protoporphyria
Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
cysteine hydrochloride
Sponsored by
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
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
NCT ID
NCT00004831
First Posted
February 24, 2000
Last Updated
March 24, 2015
Sponsor
FDA Office of Orphan Products Development
Collaborators
St. Luke's-Roosevelt Hospital Center
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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