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

This study has been completed.
Sponsor:
Collaborator:
University of Texas
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004789
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 2001
  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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00004789

Sponsors and Collaborators
University of Texas
Investigators
Study Chair: Karl Elmo Anderson University of Texas
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004789     History of Changes
Other Study ID Numbers: 199/11887, UTMB-398
Study First Received: February 24, 2000
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

Additional relevant MeSH terms:
Porphyrias
Porphyria, Erythropoietic
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Skin Diseases, Metabolic
Skin Diseases
Metabolic Diseases
Skin Diseases, Genetic
Tin mesoporphyrin
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 30, 2014