Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

This study has been completed.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	St. Luke's-Roosevelt Hospital Center
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004831

Purpose

OBJECTIVES:

I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.

Condition	Intervention
Erythropoietic Protoporphyria	Drug: cysteine hydrochloride

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind, Placebo Control, Crossover Assignment

Resource links provided by NLM:

Genetics Home Reference related topics: porphyria

Drug Information available for: Cysteine Cysteine hydrochloride

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	20
Study Start Date:	October 1996
Estimated Study Completion Date:	July 1998

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.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004831

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

More Information

No publications provided

Study ID Numbers:	199/13413, BWH-FDR000996-EF, SLRH-CU-FDR000996-EF
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004831 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

erythropoietic protoporphyria
inborn errors of metabolism
porphyria
rare disease

Additional relevant MeSH terms:

Metabolism, Inborn Errors
Liver Diseases
Metabolic Diseases
Digestive System Diseases
Skin Diseases
Genetic Diseases, Inborn

Porphyrias
Skin Diseases, Metabolic
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Skin Diseases, Genetic

ClinicalTrials.gov processed this record on November 09, 2009