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

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Primary Purpose

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

heme arginate

tin mesoporphyrin

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

NCT ID

NCT00004396

First Posted

October 18, 1999

Last Updated

June 23, 2005

Sponsor

National Center for Research Resources (NCRR)

Collaborators

University of Texas

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

Porphyria

About this trial

Eligibility Criteria

Sites / Locations

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

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