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Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004831
First Posted: February 25, 2000
Last Update Posted: March 25, 2015
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.
Collaborator:
St. Luke's-Roosevelt Hospital Center
Information provided by:
FDA Office of Orphan Products Development
  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: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
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
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 ClinicalTrials.gov identifier (NCT number): 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

ClinicalTrials.gov Identifier: NCT00004831     History of Changes
Other Study ID Numbers: 199/13413
BWH-FDR000996-EF
SLRH-CU-FDR000996-EF
First Submitted: February 24, 2000
First Posted: February 25, 2000
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
porphyria
rare disease

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