Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria
Primary Purpose
Erythropoietic Protoporphyria
Status
Completed
Phase
Phase 3
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-- Erythropoietic protoporphyria RBC protoporphyrin greater than 50 micrograms/100 dL --Prior/Concurrent Therapy-- At least 3 months since prior betacarotene or L-cysteine No concurrent betacarotene --Patient Characteristics-- Fertile female patients must use effective contraception for duration of trial and for 3 weeks thereafter Not pregnant or nursing
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004940
First Posted
February 24, 2000
Last Updated
March 24, 2015
Sponsor
Brigham and Women's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT00004940
Brief Title
Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria
Study Type
Interventional
2. Study Status
Record Verification Date
April 2000
Overall Recruitment Status
Completed
Study Start Date
May 1996 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
September 2001 (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
Brigham and Women's Hospital
4. Oversight
5. Study Description
Brief Summary
OBJECTIVES:
I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.
Detailed Description
PROTOCOL OUTLINE: This is a phase III study, lasting 3 years; 1996-1999. Patients are administered L-cysteine orally twice daily, 2 capsules with breakfast and 2 with lunch.
Patients fill in questionnaires and diary sheets about their reaction to sunlight exposure, and have blood tested 3 times a year.
Completion date provided represents the completion date of the grant per OOPD records
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
Phase 3
Enrollment
50 (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--
Erythropoietic protoporphyria RBC protoporphyrin greater than 50 micrograms/100 dL
--Prior/Concurrent Therapy--
At least 3 months since prior betacarotene or L-cysteine
No concurrent betacarotene
--Patient Characteristics--
Fertile female patients must use effective contraception for duration of trial and for 3 weeks thereafter
Not pregnant or nursing
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Micheline Mary Mathews-Roth
Organizational Affiliation
Brigham and Women's Hospital
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria
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