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

Mass General Brigham

Status and phase: Completed; Phase 3

Conditions: Erythropoietic Protoporphyria

Treatments: Drug: cysteine hydrochloride

Study type: Interventional

Funder types: Other

Identifiers

NCT00004940

BWH-FDR000996-DR

199/13376

Details and patient eligibility

About

OBJECTIVES:

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

Full 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

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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