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

NCT00004397 | Completed Phase 1

• 3 other identifiers: 199/13187UTMB-97-117UTMB-FDR001459
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Porphyria

Keywords

inborn errors of metabolism; porphyria; rare disease

Interventions

heme arginate DRUG

tin mesoporphyrin DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Center for Research Resources (NCRR): lead
• University of Texas: collaborator

MeSH Terms

Conditions

Porphyrias; Metabolism, Inborn Errors; Rare Diseases

Interventions

heme arginate; tin mesoporphyrin

Condition Hierarchy (Ancestors)

Metabolic Diseases; Nutritional and Metabolic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Karl Elmo Anderson

  University of Texas

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: January 1, 1998
• Study Completion: May 1, 2000
• Last Updated: June 24, 2005

Record last verified: 2000-06