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

This study has been completed.

Sponsor:

ClinicalTrials.gov Identifier:

NCT00004396

First Posted: October 19, 1999

Last Update Posted: December 9, 2005

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

Collaborator:

University of Texas

Information provided by:

National Center for Research Resources (NCRR)

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 2


Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: porphyria

MedlinePlus related topics: Porphyria

Genetic and Rare Diseases Information Center resources: Porphyria Congenital Erythropoietic Porphyria Acute Intermittent Porphyria

U.S. FDA Resources

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

Information from the National Library of Medicine

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Ages Eligible for Study:	18 Years and older (Adult, Senior)
Sexes Eligible for Study:	All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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


ClinicalTrials.gov Identifier:	NCT00004396 History of Changes
Other Study ID Numbers:	199/13185 UTMB-96-476 UTMB-96-318 UTMB-FDR000710 UTMB-FDR001459
First Submitted:	October 18, 1999
First Posted:	October 19, 1999
Last Update Posted:	December 9, 2005
Last Verified:	December 2003

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


inborn errors of metabolism porphyria rare disease

Additional relevant MeSH terms:


Porphyrias Porphyria, Erythropoietic Metabolic Diseases Skin Diseases, Genetic Genetic Diseases, Inborn	Skin Diseases Tin mesoporphyrin Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action

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