NCT00004940

Brief Summary

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

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started May 1996

Longer than P75 for phase_3

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 1996

Completed
3.8 years until next milestone

First Submitted

Initial submission to the registry

February 24, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 25, 2000

Completed
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2001

Completed
Last Updated

March 25, 2015

Status Verified

April 1, 2000

First QC Date

February 24, 2000

Last Update Submit

March 24, 2015

Conditions

Erythropoietic Protoporphyria

Keywords

erythropoietic protoporphyriainborn errors of metabolismporphyriarare disease

Interventions

cysteine hydrochlorideDRUG

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

MeSH Terms

Conditions

Protoporphyria, ErythropoieticMetabolism, Inborn ErrorsPorphyriasRare Diseases

Interventions

Cysteine

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Amino Acids, SulfurSulfur CompoundsOrganic ChemicalsSulfhydryl CompoundsAmino Acids, NeutralAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • Micheline Mary Mathews-Roth

    Brigham and Women's Hospital

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 3
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

May 1, 1996

Study Completion

September 1, 2001

Last Updated

March 25, 2015

Record last verified: 2000-04