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Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

This study has been completed.
St. Luke's-Roosevelt Hospital Center
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: February 24, 2000
Last updated: March 24, 2015
Last verified: August 1998


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: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:

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.


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Genders Eligible for Study:   Both
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

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Please refer to this study by its identifier: NCT00004831

Sponsors and Collaborators
FDA Office of Orphan Products Development
St. Luke's-Roosevelt Hospital Center
Study Chair: Micheline M Mathews-Roth St. Luke's-Roosevelt Hospital Center
  More Information Identifier: NCT00004831     History of Changes
Other Study ID Numbers: 199/13413  BWH-FDR000996-EF  SLRH-CU-FDR000996-EF 
Study First Received: February 24, 2000
Last Updated: March 24, 2015
Health Authority: United States: Federal Government

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

Additional relevant MeSH terms:
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Metabolic Diseases processed this record on October 21, 2016