NCT00004789

Brief Summary

OBJECTIVES: I. Evaluate the efficacy and safety of heme arginate in the treatment of 20 patients with acute attacks of porphyria. II. Evaluate the efficacy and safety of heme arginate in preventing frequent exacerbations of acute porphyria in up to 15 patients. III. Estimate the lowest effective dose of heme arginate (0.3, 1.0, or 3.0 mg/kg) in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission. IV. Evaluate the safety and efficacy of tin mesoporphyrin used in combination with heme arginate in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
59

participants targeted

Target at P50-P75 for phase_1

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

July 1, 1993

Completed
6.7 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
Last Updated

June 24, 2005

Status Verified

December 1, 2001

First QC Date

February 24, 2000

Last Update Submit

June 23, 2005

Conditions

Porphyria

Keywords

inborn errors of metabolismacute porphyriarare disease

Interventions

heme arginateDRUG
tin mesoporphyrinDRUG

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Well-documented acute porphyria, i.e.: Acute intermittent porphyria Variegate porphyria Hereditary coproporphyria --Patient Characteristics-- * No pregnant women

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

Sponsors & Collaborators

MeSH Terms

Conditions

PorphyriasMetabolism, Inborn ErrorsPorphyria, Acute IntermittentRare Diseases

Interventions

heme arginatetin mesoporphyrin

Condition Hierarchy (Ancestors)

Metabolic DiseasesNutritional and Metabolic DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPorphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticSkin DiseasesSkin and Connective Tissue DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Karl Elmo Anderson

    University of Texas

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

July 1, 1993

Last Updated

June 24, 2005

Record last verified: 2001-12