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

NCT ID: NCT00004396

Last Updated: 2005-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

32 participants

Study Classification

INTERVENTIONAL

Study Start Date

1997-09-30

Brief Summary

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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

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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.

Conditions

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Porphyria

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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heme arginate

Intervention Type DRUG

tin mesoporphyrin

Intervention Type DRUG

Eligibility Criteria

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Inclusion 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Texas

OTHER

Sponsor Role collaborator

National Center for Research Resources (NCRR)

NIH

Sponsor Role lead

Principal Investigators

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Karl Elmo Anderson

Role: STUDY_CHAIR

University of Texas

Locations

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Rockefeller University Hospital

New York, New York, United States

Site Status

University of Texas Medical Branch

Galveston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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UTMB-96-476

Identifier Type: -

Identifier Source: secondary_id

UTMB-96-318

Identifier Type: -

Identifier Source: secondary_id

UTMB-FDR000710

Identifier Type: -

Identifier Source: secondary_id

UTMB-FDR001459

Identifier Type: -

Identifier Source: secondary_id

199/13185

Identifier Type: -

Identifier Source: org_study_id