Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria

This study is ongoing, but not recruiting participants.

Sponsors and Collaborators:	FDA Office of Orphan Products Development Brigham and Women's Hospital
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004940

Purpose

OBJECTIVES:

I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.

Condition	Intervention	Phase
Erythropoietic Protoporphyria	Drug: cysteine hydrochloride	Phase III

Genetics Home Reference related topics: porphyria

Drug Information available for: Cysteine Cysteine hydrochloride

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment

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

Estimated Enrollment:	50
Study Start Date:	May 1996
Estimated Study Completion Date:	May 1996

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.

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--

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004940

Sponsors and Collaborators

FDA Office of Orphan Products Development

Brigham and Women's Hospital

Investigators

Study Chair:

Micheline Mary Mathews-Roth

Brigham and Women's Hospital

More Information

Study ID Numbers:	199/13376, BWH-FDR000996-DR
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004940
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

erythropoietic protoporphyria
inborn errors of metabolism
porphyria
rare disease

Study placed in the following topic categories:

Liver Diseases
Metabolic Diseases
Protoporphyria
Porphyrias
Skin Diseases
Protoporphyria, Erythropoietic
Rare Diseases
Porphyria, congenital erythropoietic

Metabolism, Inborn Errors
Digestive System Diseases
Genetic Diseases, Inborn
Porphyrias, Hepatic
Erythropoietic protoporphyria
Metabolic disorder
Porphyria, Erythropoietic
Skin Diseases, Genetic

Additional relevant MeSH terms:

Skin Diseases, Metabolic

ClinicalTrials.gov processed this record on January 16, 2009