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

This study has been completed.
Sponsor:
Collaborator:
University of Texas
Information provided by:
National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:
NCT00004396
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: December 2003
  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: Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment

Resource links provided by NLM:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

No publications provided

ClinicalTrials.gov Identifier: NCT00004396     History of Changes
Other 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
Health Authority: United States: Federal Government

Keywords provided by National Center for Research Resources (NCRR):
inborn errors of metabolism
porphyria
rare disease

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

ClinicalTrials.gov processed this record on August 27, 2014