Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias
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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 |
| Estimated Enrollment: | 32 |
| Study Start Date: | September 1997 |
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 (Adult, Senior) |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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 LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00004396
| 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 | |
| Study Chair: | Karl Elmo Anderson | University of Texas |
More Information
| 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 Metabolic Diseases Skin Diseases, Genetic Genetic Diseases, Inborn |
Skin Diseases Tin mesoporphyrin Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |
ClinicalTrials.gov processed this record on September 30, 2016