Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

NCT03906214 | Completed

• 1 other identifier: PC 7208
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

Porphyrias

Keywords

Porphyrias; Variegate Porphyria; Hereditary Coproporphyria; Acute Intermittent Porphyria

Outcome Measures

Primary Outcomes (3)

• 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

• 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

• Use of Medications of Interest Form

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

  5 years from study start

Interventions

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• Unable to provide informed consent and complete forms

Sponsors & Collaborators

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

Study Sites (1)

University of South Florida Data Management Coordinating Center

Tampa, Florida, 33612, United States

Location

Related 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. doi: 10.7326/0003-4819-142-6-200503150-00010.

  PMID: 15767622 BACKGROUND

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

  PMID: 4868613 BACKGROUND

• 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.

  PMID: 17763398 BACKGROUND

• 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.

  PMID: 1244784 BACKGROUND

• 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.

  PMID: 17578481 BACKGROUND

MeSH Terms

Conditions

Porphyrias; Porphyria, Variegate; Coproporphyria, Hereditary; Porphyria, Acute Intermittent

Condition Hierarchy (Ancestors)

Metabolic Diseases; Nutritional and Metabolic Diseases; Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• D. Montgomery Bissell, MD

  University of California, San Francisco

  STUDY CHAIR

• Bruce Wang, MD

  University of California, San Francisco

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2019
• First Posted: April 8, 2019
• Study Start: April 3, 2019
• Primary Completion: September 13, 2019
• Study Completion: September 13, 2019
• Last Updated: October 16, 2019

Record last verified: 2019-04

Locations

US (1)