NCT00004396

Brief Summary

OBJECTIVES: I. Compare the efficacy of heme arginate, singly or in combination with tin mesoporphyrin, in lowering porphyrin precursors in patients with asymptomatic acute intermittent porphyria. II. Evaluate and compare the safety and tolerability of these treatment regimens in this patient population.

Trial Health

80
On Track

Trial Health Score

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

Enrollment
32

participants targeted

Target at P25-P50 for phase_2

Geographic Reach
1 country

2 active sites

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

September 1, 1997

Completed
2.1 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2003

First QC Date

October 18, 1999

Last Update Submit

June 23, 2005

Conditions

Porphyria

Keywords

inborn errors of metabolismporphyriarare 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-- * Documented acute intermittent porphyria in remission for at least 1 month * Increased excretion of porphyrin precursors * Absence of neurovisceral symptoms due to porphyria for at least 1 month --Prior/Concurrent Therapy-- * At least 1 month since prior heme preparation therapy * Concurrent medication for coexisting condition is allowed, as long as dose and pattern of administration is held constant during study --Patient Characteristics-- * Renal: Urinary porphobilinogen excretion of at least 20 mg every 24 hours * Other: Not pregnant Fertile female patients must use effective contraception during and at least 6 months before study At least 1 month since any symptoms of disease No concurrent acute hemorrhagic disorder such as: Gastrointestinal bleeding Intracerebral hemorrhage No known hypersensitivity to heme arginate, tin mesoporphyrin, another heme preparation, or related heme analogue No other condition that may increase risk to patient

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Sponsors & Collaborators

Study Sites (2)

Rockefeller University Hospital

New York, New York, 10021-6399, United States

Location

University of Texas Medical Branch

Galveston, Texas, 77555-0209, United States

Location

MeSH Terms

Conditions

PorphyriasMetabolism, Inborn ErrorsRare Diseases

Interventions

heme arginatetin mesoporphyrin

Condition Hierarchy (Ancestors)

Metabolic DiseasesNutritional and Metabolic DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Karl Elmo Anderson

    University of Texas

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

September 1, 1997

Last Updated

June 24, 2005

Record last verified: 2003-12

Locations

US(2)