Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
Primary Purpose
Porphyria
Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
heme arginate
tin mesoporphyrin
Sponsored by
About this trial
This is an interventional treatment trial for Porphyria focused on measuring inborn errors of metabolism, acute porphyria, rare disease
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Well-documented acute porphyria, i.e.: Acute intermittent porphyria Variegate porphyria Hereditary coproporphyria --Patient Characteristics-- No pregnant women
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004789
First Posted
February 24, 2000
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Texas
1. Study Identification
Unique Protocol Identification Number
NCT00004789
Brief Title
Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
Study Type
Interventional
2. Study Status
Record Verification Date
December 2001
Overall Recruitment Status
Completed
Study Start Date
July 1993 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Texas
4. Oversight
5. Study Description
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 (0.3, 1.0, or 3.0 mg/kg) 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Porphyria
Keywords
inborn errors of metabolism, acute porphyria, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Enrollment
59 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
heme arginate
Intervention Type
Drug
Intervention Name(s)
tin mesoporphyrin
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Well-documented acute porphyria, i.e.: Acute intermittent porphyria Variegate porphyria Hereditary coproporphyria
--Patient Characteristics--
No pregnant women
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karl Elmo Anderson
Organizational Affiliation
University of Texas
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
We'll reach out to this number within 24 hrs