Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

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.

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Key information

Conditions

Porphyrias Congenital, Hereditary, and Neonatal Diseases and Abnormalities Coproporphyria, Hereditary Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Porphyria, Acute Intermittent Porphyria, Variegate Porphyrias, Hepatic Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

University of South Florida Data Management Coordinating Center

Tampa, Florida, 33612, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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):

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

Exclusion criteria

Unable to provide informed consent and complete forms

Treatment and study plan

Web-based Survey

Other

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.

Primary outcomes

Acute Attack Form

Time frame: 5 years from study start

Designed to capture PBG levels, characteristics of the acute attack, timing of medication administration, and pharmacological information on the medication.
Uneventful Use of Possible Risky Medication Form

Time frame: 5 years from study start

Designed to capture details of medication in question, ALA/PGB results during use of medication in questions, concomitant medication use, diet history, and alcohol use.
Use of Medications of Interest Form

Time frame: 5 years from study start

Designed to assess medications used by category and effects of each.

Sponsors and collaborators

Lead sponsor

University of South Florida

Other

Collaborators

Icahn School of Medicine at Mount Sinai
National Institutes of Health (NIH)
University of Alabama at Birmingham
University of California, San Francisco
University of Miami
University of Texas
University of Utah
University of Washington
Wake Forest University Health Sciences

Registry information

Important dates

Study start: 2019
Primary completion: 2019
Study completion: 2019
First posted: Apr 8, 2019
Registry last updated: Oct 16, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed