Porphyria Clinical Trial
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.
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.
;
Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT00004331 -
Studies in Porphyria I: Characterization of Enzyme Defects
|
N/A | |
| Completed |
NCT00004788 -
Study of Nutritional Factors in Porphyria
|
N/A | |
| Completed |
NCT00004330 -
Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks
|
N/A | |
| Completed |
NCT00004789 -
Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
|
Phase 1/Phase 2 | |
| Completed |
NCT00004398 -
Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria
|
Phase 1 | |
| Completed |
NCT00004397 -
Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria
|
Phase 1 |