Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

Porphyrias Clinical Trial

Official title:

Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03906214
• Other study ID #: PC 7208
• Status: Completed
• Start date: April 3, 2019
• Est. completion date: September 13, 2019

Study information

• Verified date: April 2019
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: September 13, 2019
• Est. primary completion date: September 13, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Porphyria, Erythropoietic
• Porphyrias

Intervention

Other:
• 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.

Locations

Country	Name	City	State
United States	University of South Florida Data Management Coordinating Center	Tampa	Florida

Sponsors (10)

Lead Sponsor	Collaborator
University of South Florida	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

Country where clinical trial is conducted

United States, 

References & Publications (5)

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. Review. Erratum in: Ann Intern Med. 2005 Aug 16;143(4):316. — View Citation

de Matteis F. Disturbances of liver porphyrin metabolism caused by drugs. Pharmacol Rev. 1967 Dec;19(4):523-57. Review. — View Citation

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. Review. — View Citation

Slavin SA, Christoforides C. Thiopental administration in acute intermittent porphyria without adverse effect. Anesthesiology. 1976 Jan;44(1):77-9. — View Citation

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. Epub 2007 Jun 19. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Acute Attack Form	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
Primary	Uneventful Use of Possible Risky Medication Form	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
Primary	Use of Medications of Interest Form	Designed to assess medications used by category and effects of each.	5 years from study start