- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05882136
Acute Intermittent Porphyria Related Abnormalities in Cardiovascular System (AIPRACUS)
Assessment of the Structure and Function of Heart and Selected Cardiovascular Risk Factors in Patients With Acute Intermittent Porphyria
Study Overview
Status
Conditions
Detailed Description
Porphyrias are heterogeneous group of the disorders of heme biosynthesis. Acute intermittent porphyria (AIP) is the most common acute hepatic porphyria, caused by the mutations in the gene encoding hydroxymethylbilane synthase. Clinical symptoms i.e. abdominal pain, nausea, vomiting, paresis or paralysis, coma and/or mental abnormalities may be induced by many porphyrinogenic factors, such as drugs, alcohol, starvation or stress. The symptoms are often accompanied by tachycardia and elevated blood pressure. Due to the non-specific clinical picture, AIP is often diagnosed too late and causes a threat to the patients' lives.
There is a scarcity of data regarding the changes in cardiovascular system in patients with AIP. The aim of this study is to assess the structure and function of heart in patients with this disease. The prevalence of hypertension, cardiac arrhythmias and selected cardiovascular risk factors in patients with AIP will also be evaluated.
This is a case-control study with prospective observation of the subgroup of patients examined during the exacerbations of AIP.
Specific goals:
- assessment of the cardiac morphology and function,
- assessment of the concentrations of markers of myocardial injury (troponin T) and heart failure (NT-proBNP),
- assessment of the prevalence of cardiac arrhythmias and electrocardiographic abnormalities,
- assessment of the blood pressure profiles,
- assessment of selected cardiovascular risk factors,
- assessment of quality of life,
- assessment of clinical and biochemical factors associated with the pathological findings in patients with AIP.
Study Type
Enrollment (Actual)
Contacts and Locations
Study Contact
- Name: Krzysztof Jaworski, MD
- Phone Number: +48223434259
- Email: kjaworski@ikard.pl
Study Contact Backup
- Name: Rafal Dabrowski, MD, PhD
- Phone Number: +48223434409
- Email: rdabrowski@ikard.pl
Study Locations
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Warsaw, Poland, 02-776
- Institute of Hematology and Transfusion Medicine
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Warsaw, Poland, 04-628
- National Institute of Cardiology
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
Inclusion Criteria:
- acute intermittent porphyria (AIP),
- age 18-65 years,
- at least one hospitalization due to exacerbation of AIP.
Exclusion Criteria:
- previous myocardial infarction,
- heart failure of established (other than porphyria) etiology,
- severe heart valve disease,
- congenital heart defects,
- history of myocarditis,
- pacemaker,
- hyperthyroidism / hypothyroidism (except for adequate thyroid hormone replacement therapy),
- chronic advanced lung diseases,
- lack of consent to participate in the study.
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
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Patients with acute intermittent porphyria (AIP)
Patients aged 18-65 (men and women) with acute intermittent porphyria and at least one episode of exacerbation of this disease requiring admission to the hospital
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Control group
People recruited from the general population and matched by age, sex and body mass index.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of participants with heart failure and left ventricular dysfunction.
Time Frame: day 1 and up to 2 years in case of the exacerbation of AIP
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Heart failure and left ventricular dysfunction assessed by symptoms and echocardiography.
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day 1 and up to 2 years in case of the exacerbation of AIP
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Number of participants with hypertension.
Time Frame: day 1 and up to 2 years in case of the exacerbation of AIP
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Arterial hypertension diagnosed by ambulatory 24-hour blood pressure monitoring.
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day 1 and up to 2 years in case of the exacerbation of AIP
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Rate of cardiac arrhythmias.
Time Frame: day 1 and up to 2 years in case of the exacerbation of AIP
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Cardiac arrhythmias observed in 24-hour ECG monitoring.
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day 1 and up to 2 years in case of the exacerbation of AIP
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of participants with increased concentrations of markers of heart failure.
Time Frame: day 1 and up to 2 years in case of the exacerbation of AIP
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Concentration of N-terminal pro-B-type natriuretic peptide (NT-proBNP) > 125 pg/mL in serum.
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day 1 and up to 2 years in case of the exacerbation of AIP
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Number of participants with increased concentrations of markers of myocardial injury.
Time Frame: day 1 and up to 2 years in case of the exacerbation of AIP
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Concentration of high sensitivity troponin T > 14 ng/L in serum.
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day 1 and up to 2 years in case of the exacerbation of AIP
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Number of participants with chronic kidney disease.
Time Frame: day 1 and up to 2 years in case of the exacerbation of AIP
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Renal function defined by estimated glomerular filtration rate based on the concentrations of creatinine in serum.
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day 1 and up to 2 years in case of the exacerbation of AIP
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Number of participants with dyslipidemia.
Time Frame: day 1
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Dyslipidemia defined as the concentrations of total cholesterol > 190 mg/dL, low density lipoprotein (LDL) cholesterol > 115 mg/dL, high density lipoprotein (HDL) cholesterol < 48 mg/dL (female), < 40 mg/dL (male), triglycerides > 150 mg/dL.
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day 1
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Number of participants with diabetes.
Time Frame: day 1
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Diabetes in anamnesis or the concentration of glycated hemoglobin > 6.5%.
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day 1
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Level of quality of life.
Time Frame: day 1
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Physical and mental components defined by the Short Form 36 survey (SF36).
Answers are weighted and transformed into scores in a scale ranging from 0 (the lowest possible level of functioning) to 100 (no restrictions).
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day 1
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Krzysztof Jaworski, MD, National Institute of Cardiology, Warsaw, Poland
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2.39/VII/18
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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