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Trial record 8 of 11 for:    "Protoporphyria"

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

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


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


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--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
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Please refer to this study by its identifier: NCT00004940

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

Additional relevant MeSH terms:
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Metabolic Diseases processed this record on October 21, 2016