Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

NCT ID: NCT03906214

Last Updated: 2019-10-16

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

Total Enrollment

60 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-04-03

Study Completion Date

2019-09-13

Brief Summary

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This is an observational study collecting patient/caregiver reports on suspected medication/drug-induced acute porphyria attacks, as well as safe use of drugs previously labeled "unsafe" or with unknown risk. Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium. The study will be advertised on the Consortium website and through the American Porphyria Foundation's social media network.

Detailed Description

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Contact Registrants will be sent an email invitation describing the study. A unique link will be generated for each registrant and included in the email invitation. After 2 weeks, a second email invitation will be sent to the registrants who have not yet participated. After 1 month, a third email invitation will be sent to the registrants who have not yet participated. We may send additional emails periodically (e.g. every 6-12 months) to subjects enrolled to remind them to complete new surveys if they have taken any other medications of interest or have experienced any new reactions/attacks.

If the registrant wants to participate in the study, he/she will follow the survey link in the email invitation, which directs him/her to an IRB-approved online consent form. If the participant consents to participate, he/she will be directed to online forms to report general information (ex. age, sex, test results, family history), suspected medication-related acute attacks, and "uneventful use of possibly risky medication". If a patient has multiple medication-related acute attacks, the patient/caregiver will be able to submit multiple report forms that will be linked. In addition to the report forms, patients will be asked to review a list of "Medications of Interest" and report current or past use.

Conditions

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Porphyrias

Study Design

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Observational Model Type

OTHER

Study Time Perspective

OTHER

Interventions

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Web-based Survey

Participants will be directed to online forms to report general information (ex. age, sex, test results, family history), suspected medication-related acute attacks, and "uneventful use of possibly risky medication". If a patient has multiple medication-related acute attacks, the patient/caregiver will be able to submit multiple report forms that will be linked. In addition to the report forms, patients will be asked to review a list of "Medications of Interest" and report current or past use.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Able to read and write in English
* Enrolled in the RDCRN PC Contact Registry
* Self-reported patient or caregiver of patient with acute porphyria

There are no restrictions on age, sex or ethnicity. However, the following criteria will be applied to those reports that are considered evaluable (analyzed and scored):

1. Medication name must be specified
2. The data elements required in the scoring system must be complete

Exclusion Criteria

* Unable to provide informed consent and complete forms
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Texas

OTHER

Sponsor Role collaborator

Wake Forest University Health Sciences

OTHER

Sponsor Role collaborator

University of Miami

OTHER

Sponsor Role collaborator

University of Washington

OTHER

Sponsor Role collaborator

Icahn School of Medicine at Mount Sinai

OTHER

Sponsor Role collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role collaborator

University of California, San Francisco

OTHER

Sponsor Role collaborator

University of Utah

OTHER

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

University of South Florida

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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D. Montgomery Bissell, MD

Role: STUDY_CHAIR

University of California, San Francisco

Bruce Wang, MD

Role: STUDY_CHAIR

University of California, San Francisco

Locations

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University of South Florida Data Management Coordinating Center

Tampa, Florida, United States

Site Status

Countries

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United States

References

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Anderson KE, Bloomer JR, Bonkovsky HL, Kushner JP, Pierach CA, Pimstone NR, Desnick RJ. Recommendations for the diagnosis and treatment of the acute porphyrias. Ann Intern Med. 2005 Mar 15;142(6):439-50. doi: 10.7326/0003-4819-142-6-200503150-00010.

Reference Type BACKGROUND
PMID: 15767622 (View on PubMed)

de Matteis F. Disturbances of liver porphyrin metabolism caused by drugs. Pharmacol Rev. 1967 Dec;19(4):523-57. No abstract available.

Reference Type BACKGROUND
PMID: 4868613 (View on PubMed)

Seth AK, Badminton MN, Mirza D, Russell S, Elias E. Liver transplantation for porphyria: who, when, and how? Liver Transpl. 2007 Sep;13(9):1219-27. doi: 10.1002/lt.21261.

Reference Type BACKGROUND
PMID: 17763398 (View on PubMed)

Slavin SA, Christoforides C. Thiopental administration in acute intermittent porphyria without adverse effect. Anesthesiology. 1976 Jan;44(1):77-9. doi: 10.1097/00000542-197601000-00021. No abstract available.

Reference Type BACKGROUND
PMID: 1244784 (View on PubMed)

Thunell S, Pomp E, Brun A. Guide to drug porphyrogenicity prediction and drug prescription in the acute porphyrias. Br J Clin Pharmacol. 2007 Nov;64(5):668-79. doi: 10.1111/j.0306-5251.2007.02955.x. Epub 2007 Jun 19.

Reference Type BACKGROUND
PMID: 17578481 (View on PubMed)

Other Identifiers

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PC 7208

Identifier Type: -

Identifier Source: org_study_id

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