Ignite Creation Date:
2024-05-05 @ 10:17 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004789
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2000-02-24
Brief Title:
Phase III Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
Sponsor:
National Center for Research Resources NCRR
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2001-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
OBJECTIVES I Evaluate the efficacy and safety of heme arginate in the treatment of 20 patients with acute attacks of porphyria
II Evaluate the efficacy and safety of heme arginate in preventing frequent exacerbations of acute porphyria in up to 15 patients
III Estimate the lowest effective dose of heme arginate 03 10 or 30 mgkg in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission
IV Evaluate the safety and efficacy of tin mesoporphyrin used in combination with heme arginate in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission
II Evaluate the efficacy and safety of heme arginate in preventing frequent exacerbations of acute porphyria in up to 15 patients
III Estimate the lowest effective dose of heme arginate 03 10 or 30 mgkg in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission
IV Evaluate the safety and efficacy of tin mesoporphyrin used in combination with heme arginate in reducing porphyrin precursors in 12 stable patients with acute intermittent porphyria in remission
Detailed Description:
PROTOCOL OUTLINE This project involves 4 studies an unblinded multicenter treatment study an unblinded prevention study a controlled dose-ranging study and a controlled drug combination dose-ranging study
In the treatment study participants with acute attacks of porphyria receive intravenous heme arginate daily for 4 days Treatment may be extended to 7 days in patients with severe attacks and slow recovery these data are analyzed separately
In the prevention study participants receive weekly infusions of heme arginate for 6 months Treatment may be extended in selected cases these data are analyzed separately If an acute attack occurs patients are treated with a standard course of heme arginate Participants are followed weekly for 6 months after preventive treatment
In the dose-ranging study 4 groups of 3 patients are randomly assigned to standard and low-dose heme arginate infusions administered daily for 4 days After a washout of 4-12 weeks patients are crossed to a second dose In 2 of the 4 groups patients cross between heme arginate and a normal saline control
In the combination dose-ranging study 4 groups of 3 patients are randomly assigned to 1 of 2 doses of tin mesoporphyrin After a 4-12 week washout these patients are crossed to a standard or low-dose heme arginate infusion After a second 4-12 week washout patients are given a combination infusion of tin mesoporphyrin followed by heme arginate
All participants are given a special diet to prevent fluctuations in porphyrin precursors
In the treatment study participants with acute attacks of porphyria receive intravenous heme arginate daily for 4 days Treatment may be extended to 7 days in patients with severe attacks and slow recovery these data are analyzed separately
In the prevention study participants receive weekly infusions of heme arginate for 6 months Treatment may be extended in selected cases these data are analyzed separately If an acute attack occurs patients are treated with a standard course of heme arginate Participants are followed weekly for 6 months after preventive treatment
In the dose-ranging study 4 groups of 3 patients are randomly assigned to standard and low-dose heme arginate infusions administered daily for 4 days After a washout of 4-12 weeks patients are crossed to a second dose In 2 of the 4 groups patients cross between heme arginate and a normal saline control
In the combination dose-ranging study 4 groups of 3 patients are randomly assigned to 1 of 2 doses of tin mesoporphyrin After a 4-12 week washout these patients are crossed to a standard or low-dose heme arginate infusion After a second 4-12 week washout patients are given a combination infusion of tin mesoporphyrin followed by heme arginate
All participants are given a special diet to prevent fluctuations in porphyrin precursors
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UTMB-398 | None | None | None |