Porphyrias
Conditions
Keywords
Porphyrias, Variegate Porphyria, Hereditary Coproporphyria, Acute Intermittent Porphyria
Brief summary
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
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.
Interventions
OTHERWeb-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.
Sponsors
University of Texas
Wake Forest University Health Sciences
University of Miami
University of Washington
Icahn School of Medicine at Mount Sinai
University of Alabama at Birmingham
University of California, San Francisco
University of Utah
National Institutes of Health (NIH)
University of South Florida
Study design
Observational model
OTHER
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Acute Attack Form | 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 | 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 | 5 years from study start | Designed to assess medications used by category and effects of each. |
Countries
United States
Outcome results
None listed