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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004940
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.
Information provided by:
FDA Office of Orphan Products Development
  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 3

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


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

Estimated Enrollment: 50
Study Start Date: May 1996
Estimated Study Completion Date: September 2001
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.

Completion date provided represents the completion date of the grant per OOPD records

  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--

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
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): NCT00004940


Sponsors and Collaborators
Brigham and Women's Hospital
Investigators
Study Chair: Micheline Mary Mathews-Roth Brigham and Women's Hospital
  More Information

ClinicalTrials.gov Identifier: NCT00004940     History of Changes
Other Study ID Numbers: 199/13376
BWH-FDR000996-DR
First Submitted: February 24, 2000
First Posted: February 25, 2000
Last Update Posted: March 25, 2015
Last Verified: April 2000

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