Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria

This study has been completed.
Sponsor:
Collaborator:
University of Texas
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004397
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: June 2000
  Purpose

OBJECTIVES: I. Evaluate the effectiveness of tin mesoporphyrin in reducing the number of heme infusions needed to prevent acute attacks of porphyria.

II. Evaluate the safety and tolerability of tin mesoporphyrin in these patients.


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

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: 20
Study Start Date: January 1998
Estimated Study Completion Date: May 2000
Detailed Description:

PROTOCOL OUTLINE: This is an unblinded study. Patients start a preventive heme regimen and are followed for 6 months prior to tin mesoporphyrin. The frequency of attacks is recorded.

Patients receive tin mesoporphyrin once a week for 6 months. Patients must avoid sunlight during treatment and for 2 months thereafter. The frequency of heme infusions is reduced by half and the frequency of attacks of porphyria is monitored.

Patients are followed for 6 months after last treatment.

  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, variegate porphyria, or hereditary coproporphyria causing 6 or more attacks per year Fewer than 2 attacks in the preceding 6 months if already on preventive heme therapy --Prior/Concurrent Therapy-- See Disease Characteristics --Patient Characteristics-- Performance status: Not specified Life expectancy: Not specified Hematopoietic: Not specified Hepatic: Not specified Renal: Urinary porphobilinogen at least 20 mg/24 hours during acute symptoms Other: Not pregnant Fertile female patients must use effective contraception during and for 6 months prior to study No recurrent symptoms due to another illness No continuous (for longer than 1 month) symptoms of porphyria unless it is in addition to acute exacerbations No acute hemorrhagic disorder such as: Gastrointestinal bleeding Intracerebral hemorrhage No known hypersensitivity to tin mesoporphyrin No known hypersensitivity to any intravenous heme preparation No other condition which 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: NCT00004397

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

No publications provided

ClinicalTrials.gov Identifier: NCT00004397     History of Changes
Other Study ID Numbers: 199/13187, UTMB-97-117, UTMB-FDR001459
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
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 18, 2014