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Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004831
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : March 25, 2015
St. Luke's-Roosevelt Hospital Center
Information provided by:
FDA Office of Orphan Products Development

Brief Summary:


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

Condition or disease Intervention/treatment Phase
Erythropoietic Protoporphyria Drug: cysteine hydrochloride Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
Primary Purpose: Treatment
Study Start Date : October 1996
Study Completion Date : July 1998

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004831

Sponsors and Collaborators
FDA Office of Orphan Products Development
St. Luke's-Roosevelt Hospital Center
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Study Chair: Micheline M Mathews-Roth St. Luke's-Roosevelt Hospital Center

Layout table for additonal information Identifier: NCT00004831     History of Changes
Other Study ID Numbers: 199/13413
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: August 1998

Keywords provided by FDA Office of Orphan Products Development:
erythropoietic protoporphyria
inborn errors of metabolism
rare disease

Additional relevant MeSH terms:
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Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Metabolic Diseases