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

OBJECTIVES:

I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.

Completed

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Key information

Conditions

Erythropoietic Protoporphyria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Disease Attributes Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Porphyrias Porphyrias, Hepatic Protoporphyria, Erythropoietic Rare Diseases Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

cysteine hydrochloride

Drug

Sponsors and collaborators

Lead sponsor

Brigham and Women's Hospital

Other

Registry information

Important dates

Study start: 1996
Study completion: 2001
First posted: Feb 25, 2000
Registry last updated: Mar 25, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed