Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Erythropoietic Protoporphyria Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004831
• Other study ID #: 199/13413
• Secondary ID: BWH-FDR000996-EF
• Status: Completed
• Phase: N/A
• First received: February 24, 2000
• Last updated: March 24, 2015
• Start date: October 1996
• Est. completion date: July 1998

Study information

• Verified date: August 1998
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

OBJECTIVES:

I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.

Description:

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, crossover study.

Patients are randomly assigned to 1 of 2 groups to receive cysteine hydrochloride orally twice daily, 2 capsules with breakfast and 2 with lunch. Group 1 receives cysteine hydrochloride in drug ingestion period 1 followed by placebo in period 2. Group 2 receives placebo in period 1 followed by cysteine hydrochloride in period 2. Both groups ingest placebo for 1 week between the periods. Each drug ingestion period lasts 8 weeks.

Follow up phone calls are made at the end of months 1 and 3. All patients schedule follow up visits at the end of each drug ingestion period.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: July 1998
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Documented erythropoietic protoporphyria Determination of elevated protoporphyrin within the past year Experiencing photosensitivity --Prior/Concurrent Therapy-- No concurrent use of betacarotene --Patient Characteristics-- Other: Not pregnant or nursing Fertile female patients must use effective contraception while on study and for 3 weeks thereafter

Study Design

Allocation: Randomized, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Erythropoietic Protoporphyria

Intervention

Drug:
• cysteine hydrochloride

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
FDA Office of Orphan Products Development	St. Luke's-Roosevelt Hospital Center