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Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004789
First Posted: February 25, 2000
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:
Office of Rare Diseases (ORD)
  Purpose

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.


Condition Intervention Phase
Porphyria Drug: heme arginate Drug: tin mesoporphyrin Phase 1 Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 59
Study Start Date: July 1993
Detailed Description:

PROTOCOL OUTLINE: This project involves 4 studies: an unblinded multicenter treatment study, an unblinded prevention study, a controlled dose-ranging study, and a controlled drug combination dose-ranging study.

In the treatment study, participants with acute attacks of porphyria receive intravenous heme arginate daily for 4 days. Treatment may be extended to 7 days in patients with severe attacks and slow recovery; these data are analyzed separately.

In the prevention study, participants receive weekly infusions of heme arginate for 6 months. Treatment may be extended in selected cases; these data are analyzed separately. If an acute attack occurs, patients are treated with a standard course of heme arginate. Participants are followed weekly for 6 months after preventive treatment.

In the dose-ranging study, 4 groups of 3 patients are randomly assigned to standard and low-dose heme arginate infusions, administered daily for 4 days. After a washout of 4-12 weeks, patients are crossed to a second dose. In 2 of the 4 groups, patients cross between heme arginate and a normal saline control.

In the combination dose-ranging study, 4 groups of 3 patients are randomly assigned to 1 of 2 doses of tin mesoporphyrin. After a 4-12 week washout, these patients are crossed to a standard or low-dose heme arginate infusion. After a second 4-12 week washout, patients are given a combination infusion of tin mesoporphyrin followed by heme arginate.

All participants are given a special diet to prevent fluctuations in porphyrin precursors.

  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--

  • Well-documented acute porphyria, i.e.: Acute intermittent porphyria Variegate porphyria Hereditary coproporphyria

--Patient Characteristics--

  • No pregnant women
  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): NCT00004789


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: NCT00004789     History of Changes
Other Study ID Numbers: 199/11887
UTMB-398
First Submitted: February 24, 2000
First Posted: February 25, 2000
Last Update Posted: December 9, 2005
Last Verified: December 2001

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
acute 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