Text Size

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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2000 by FDA Office of Orphan Products Development.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Brigham and Women's Hospital
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004940
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: April 2000
History of Changes
  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:

MedlinePlus related topics: Porphyria
U.S. FDA Resources

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

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

  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--

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004940

Sponsors and Collaborators
FDA Office of Orphan Products Development
Brigham and Women's Hospital
Investigators
Study Chair: Micheline Mary Mathews-Roth Brigham and Women's Hospital
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004940     History of Changes
Other Study ID Numbers: 199/13376, BWH-FDR000996-DR
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:
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Porphyrias
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Skin Diseases, Metabolic
Metabolic Diseases

ClinicalTrials.gov processed this record on May 21, 2013