Porphyria
Conditions
Keywords
inborn errors of metabolism, porphyria, rare disease
Brief summary
OBJECTIVES: I. Evaluate the efficacy of tin mesoporphyrin in patients with acute porphyria attacks who are also treated with a standard course of heme arginate. II. Evaluate the safety and tolerability of tin mesoporphyrin when administered to these patients.
Detailed description
PROTOCOL OUTLINE: This is an randomized, unblinded, multicenter study. Patients are randomized to receive heme arginate alone or both heme arginate and tin mesoporphyrin. Patients receive a single intravenous dose of tin mesoporphyrin immediately before the first dose of heme arginate. Heme arginate is administered daily for 4 days. Patients are followed at 3 and 6 days after treatment.
Interventions
DRUGheme arginate
Sponsors
University of Texas
National Center for Research Resources (NCRR)
Study design
Allocation
RANDOMIZED
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Documented acute intermittent porphyria, variegate porphyria, or hereditary coproporphyria No chronic or subacute symptoms (present for longer than 2 weeks) --Prior/Concurrent Therapy-- At least 3 weeks since prior treatment with heme arginate or tin mesoporphyrin --Patient Characteristics-- Renal: Urinary porphobilinogen at least 40 mg/24 hr Other: * Not pregnant * Fertile female patients must use effective contraception during and 6 months before study * No evidence that symptoms are due to another acute illness * No acute hemorrhagic disorder such as: Gastrointestinal bleeding Intracerebral hemorrhage * No known sensitivity to heme preparation or tin mesoporphyrin * No other medical condition that might increase risk to patient
Outcome results
None listed