Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

This study has been completed.
Sponsor:
Collaborator:
St. Luke's-Roosevelt Hospital Center
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004831
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: August 1998
  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-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.

  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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Sponsors and Collaborators
St. Luke's-Roosevelt Hospital Center
Investigators
Study Chair: Micheline M Mathews-Roth St. Luke's-Roosevelt Hospital Center
  More Information

No publications provided

ClinicalTrials.gov 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: June 23, 2005
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:
Porphyrias, Hepatic
Porphyrias
Protoporphyria, Erythropoietic
Liver Diseases
Digestive System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Skin Diseases, Metabolic
Metabolic Diseases

ClinicalTrials.gov processed this record on October 01, 2014