Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

This study has been completed.

First Received: October 18, 1999 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	National Center for Research Resources (NCRR) University of Texas
Information provided by:	National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:	NCT00004396

Purpose

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.

Condition	Intervention	Phase
Porphyria	Drug: heme arginate Drug: tin mesoporphyrin	Phase II

Genetics Home Reference related topics: porphyria

MedlinePlus related topics: Porphyria

Drug Information available for: Heme arginate Tin mesoporphyrin

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Safety/Efficacy Study

Further study details as provided by National Center for Research Resources (NCRR):

Estimated Enrollment:	32
Study Start Date:	September 1997

Detailed Description:

PROTOCOL OUTLINE: This is an unblinded, dose ranging study. Patients receive heme arginate alone or in combination with tin mesoporphyrin.

Patients receive tin mesoporphyrin IV as a single dose. Heme arginate is administered as a single intravenous infusion.

Patients are entered in cohorts of 4. Subsequent cohorts of 4 patients each receive escalating doses of tin mesoporphyrin in combination with 1 of 2 different dosages of heme arginate. Subjects must maintain a constant diet.

Patients experiencing adverse reactions are followed as clinically indicated.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004396

Locations

United States, New York
Rockefeller University Hospital
New York, New York, United States, 10021-6399
United States, Texas
University of Texas Medical Branch
Galveston, Texas, United States, 77555-0209

Sponsors and Collaborators

National Center for Research Resources (NCRR)

University of Texas

Investigators

Study Chair:

Karl Elmo Anderson

University of Texas

More Information

No publications provided

Study ID Numbers:	199/13185, UTMB-96-476, UTMB-96-318, UTMB-FDR000710, UTMB-FDR001459
Study First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004396 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by National Center for Research Resources (NCRR):

inborn errors of metabolism
porphyria
rare disease

Study placed in the following topic categories:

Metabolic Diseases
Porphyrias
Skin Diseases
Acute Intermittent Porphyria
Rare Diseases
Porphyria, Acute Intermittent
Porphyria, Congenital Erythropoietic

Metabolism, Inborn Errors
Tin mesoporphyrin
Genetic Diseases, Inborn
Porphyria, Erythropoietic
Porphyria
Skin Diseases, Genetic
Metabolic Disorder

Additional relevant MeSH terms:

Metabolism, Inborn Errors
Tin mesoporphyrin
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Skin Diseases
Genetic Diseases, Inborn

Porphyrias
Skin Diseases, Metabolic
Enzyme Inhibitors
Porphyria, Erythropoietic
Skin Diseases, Genetic
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 07, 2009