PorphyriasNCT03906214
Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria
An observational study of Porphyrias, run by University of South Florida at 1 site in United States.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Porphyrias
- What is being tested?
- Web-based Survey
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Apr 2019to 13 Sep 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium.
Who may be able to join
- 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
Who cannot join
- Unable to provide informed consent and complete forms
Trial locations and contacts
1 site in 1 country.
United States1 site
University of South Florida Data Management Coordinating Center
Tampa, Florida, 33612
About this study
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.
Read the 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.
- Conditions
- Porphyrias
- Being tested
- Web-based Survey
- Keywords
- Porphyrias, Variegate Porphyria, Hereditary Coproporphyria, Acute Intermittent Porphyria
- Medical subject headings
- Porphyrias, Porphyria, Variegate, Coproporphyria, Hereditary, Porphyria, Acute Intermittent
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Web-based SurveyOther
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
Acute Attack Form
Designed to capture PBG levels, characteristics of the acute attack, timing of medication administration, and pharmacological information on the medication.
Measured: 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.
Measured: 5 years from study start
Use of Medications of Interest Form
Designed to assess medications used by category and effects of each.
Measured: 5 years from study start
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2019
- Study start3 Apr 2019
- Primary completion13 Sep 2019
- Study completion13 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for PorphyriasStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- 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)
- Responsible party
- Sponsor
- Investigators
- D. Montgomery Bissell, MD, Study chair, University of California, San FranciscoBruce Wang, MD, Study chair, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03906214
- Sponsor's protocol ID
- PC 7208
- Status last verified
- Apr 2019
- First submitted
- 4 Apr 2019
- Last updated
- 16 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 15767622de Matteis F. Disturbances of liver porphyrin metabolism caused by drugs. Pharmacol Rev. 1967 Dec;19(4):523-57. No abstract available.
PubMed 4868613Seth 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.
PubMed 17763398Slavin 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.
PubMed 1244784Thunell 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.
PubMed 17578481
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria
- ClinicalTrials.gov ID
- NCT03906214
- Acronym / sponsor ID
- PC 7208
- Conditions
- Porphyrias
- Keywords
- Porphyrias, Variegate Porphyria, Hereditary Coproporphyria, Acute Intermittent Porphyria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- 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)
- Study start
- 3 Apr 2019 (actual)
- Primary completion
- 13 Sep 2019 (actual)
- Study completion
- 13 Sep 2019 (actual)
- First posted
- 8 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be recruited through the RDCRN Contact Registry for the Porphyrias Consortium.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (1 listed)
- Unable to provide informed consent and complete forms
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Web-based SurveyParticipants 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. | Other | – |
Primary outcome
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.
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.
Use of Medications of Interest Form
Time frame: 5 years from study start
Designed to assess medications used by category and effects of each.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2019.
- Background: PubMed 15767622Anderson 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.
- Background: PubMed 4868613de Matteis F. Disturbances of liver porphyrin metabolism caused by drugs. Pharmacol Rev. 1967 Dec;19(4):523-57. No abstract available.
- Background: PubMed 17763398Seth 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.
- Background: PubMed 1244784Slavin 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.
- Background: PubMed 17578481Thunell 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
D. Montgomery Bissell, MD study chair
University of California, San Francisco
Bruce Wang, MD study chair
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Porphyrias, run by University of South Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Acute Attack Form (time frame: 5 years from study start). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03906214, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2019.