Trial record 8 of 11 for:    "Protoporphyria"

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

This study has been completed.
Sponsor:
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004940
First received: February 24, 2000
Last updated: March 24, 2015
Last verified: April 2000
  Purpose

OBJECTIVES:

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


Condition Intervention Phase
Erythropoietic Protoporphyria
Drug: cysteine hydrochloride
Phase 3

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 50
Study Start Date: May 1996
Estimated Study Completion Date: September 2001
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

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004940

Sponsors and Collaborators
Brigham and Women's Hospital
Investigators
Study Chair: Micheline Mary Mathews-Roth Brigham and Women's Hospital
  More Information

ClinicalTrials.gov Identifier: NCT00004940     History of Changes
Other Study ID Numbers: 199/13376  BWH-FDR000996-DR 
Study First Received: February 24, 2000
Last Updated: March 24, 2015
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
erythropoietic protoporphyria
inborn errors of metabolism
porphyria
rare disease

Additional relevant MeSH terms:
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias
Metabolic Diseases

ClinicalTrials.gov processed this record on August 22, 2016