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Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

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ClinicalTrials.gov Identifier: NCT03906214
Recruitment Status : Recruiting
First Posted : April 8, 2019
Last Update Posted : April 8, 2019
Sponsor:
Collaborators:
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)
Information provided by (Responsible Party):
University of South Florida

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.

Condition or disease Intervention/treatment
Porphyrias Other: Web-based Survey

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.


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Study Type : Observational
Estimated Enrollment : 2000 participants
Observational Model: Other
Time Perspective: Other
Official Title: Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria
Actual Study Start Date : April 3, 2019
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : April 2024



Intervention Details:
  • 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.


Primary Outcome Measures :
  1. 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.

  2. 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.

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



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03906214


Contacts
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Contact: Callyn Kirk, MSPH 813-396-9244 Callyn.Kirk@epi.usf.edu

Locations
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United States, Florida
University of South Florida Data Management Coordinating Center Recruiting
Tampa, Florida, United States, 33612
Contact: Callyn Kirk, MSPH         
Principal Investigator: Jeffrey Krischer, PhD         
Sponsors and Collaborators
University of South Florida
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)
Investigators
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Study Chair: D. Montgomery Bissell, MD University of California, San Francisco
Study Chair: Bruce Wang, MD University of California, San Francisco

Publications:
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Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT03906214     History of Changes
Other Study ID Numbers: PC 7208
First Posted: April 8, 2019    Key Record Dates
Last Update Posted: April 8, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University of South Florida:
Porphyrias
Variegate Porphyria
Hereditary Coproporphyria
Acute Intermittent Porphyria

Additional relevant MeSH terms:
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Porphyrias
Porphyria, Erythropoietic
Porphyrias, Hepatic
Coproporphyria, Hereditary
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases