search
Back to results

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

Primary Purpose

Porphyria

Status
Completed
Phase
Phase 2
Locations
United States
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, porphyria, rare disease

Eligibility Criteria

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

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

Sites / Locations

  • Rockefeller University Hospital
  • University of Texas Medical Branch

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
October 18, 1999
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Texas
search

1. Study Identification

Unique Protocol Identification Number
NCT00004396
Brief Title
Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias
Study Type
Interventional

2. Study Status

Record Verification Date
December 2003
Overall Recruitment Status
Completed
Study Start Date
September 1997 (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. 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.
Detailed Description
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.

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, porphyria, rare disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Enrollment
32 (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-- 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karl Elmo Anderson
Organizational Affiliation
University of Texas
Official's Role
Study Chair
Facility Information:
Facility Name
Rockefeller University Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10021-6399
Country
United States
Facility Name
University of Texas Medical Branch
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555-0209
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

We'll reach out to this number within 24 hrs