Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

October 15, 2019 updated by: University of South Florida
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Actual)

60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Tampa, Florida, United States, 33612
        • University of South Florida Data Management Coordinating Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
5 years from study start
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.
5 years from study start
Use of Medications of Interest Form
Time Frame: 5 years from study start
Designed to assess medications used by category and effects of each.
5 years from study start

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of South Florida

Collaborators

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

Investigators

  • Study Chair: D. Montgomery Bissell, MD, University of California, San Francisco
  • Study Chair: Bruce Wang, MD, University of California, San Francisco

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 3, 2019

Primary Completion (Actual)

September 13, 2019

Study Completion (Actual)

September 13, 2019

Study Registration Dates

First Submitted

April 4, 2019

First Submitted That Met QC Criteria

April 4, 2019

First Posted (Actual)

April 8, 2019

Study Record Updates

Last Update Posted (Actual)

October 16, 2019

Last Update Submitted That Met QC Criteria

October 15, 2019

Last Verified

April 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PC 7208

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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