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Sponsors and Collaborators: |
FDA Office of Orphan Products Development Brigham and Women's Hospital |
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Information provided by: | FDA Office of Orphan Products Development |
ClinicalTrials.gov Identifier: | NCT00004940 |
OBJECTIVES:
I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.
Condition | Intervention | Phase |
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Erythropoietic Protoporphyria |
Drug: cysteine hydrochloride |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment |
Estimated Enrollment: | 50 |
Study Start Date: | May 1996 |
Estimated Study Completion Date: | May 1996 |
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.
Ages Eligible for Study: | 18 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
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--
Study ID Numbers: | 199/13376, BWH-FDR000996-DR |
Study First Received: | February 24, 2000 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00004940 History of Changes |
Health Authority: | United States: Federal Government |
erythropoietic protoporphyria inborn errors of metabolism porphyria rare disease |
Liver Diseases Metabolic Diseases Protoporphyria Porphyrias Skin Diseases Protoporphyria, Erythropoietic Rare Diseases Porphyria, Congenital Erythropoietic Metabolism, Inborn Errors |
Digestive System Diseases Genetic Diseases, Inborn Erythropoietic Protoporphyria Porphyrias, Hepatic Porphyria, Erythropoietic Porphyria Metabolic Disorder Skin Diseases, Genetic |
Metabolism, Inborn Errors Liver Diseases Metabolic Diseases Digestive System Diseases Skin Diseases Genetic Diseases, Inborn |
Porphyrias Skin Diseases, Metabolic Protoporphyria, Erythropoietic Porphyrias, Hepatic Skin Diseases, Genetic |