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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
National Center for Research Resources (NCRR)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Porphyria focused on measuring inborn errors of metabolism, acute porphyria, rare disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

    First Posted
    February 24, 2000
    Last Updated
    June 23, 2005
    Sponsor
    National Center for Research Resources (NCRR)
    Collaborators
    University of Texas
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    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

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