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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00004940

Enrollment

Registered

2000-02-25

Start date

1996-05-31

Completion date

2001-09-30

Last updated

2015-03-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Erythropoietic Protoporphyria

Keywords

erythropoietic protoporphyria, inborn errors of metabolism, porphyria, rare disease

Brief summary

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

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

Interventions

DRUGcysteine hydrochloride

Study design

Primary purpose

TREATMENT

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Inclusion 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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026