Porphyrias, HepaticAIPRACUSNCT05882136
Acute Intermittent Porphyria Related Abnormalities in Cardiovascular System
An observational study of Porphyrias, Hepatic, run by National Institute of Cardiology, Warsaw, Poland at 2 sites in Poland.
Trial summary
This study aims to assess the changes in the cardiovascular system in patients with acute intermittent porphyria (AIP). Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Porphyrias, Hepatic
- How many people
- 180 participants
- Where
- 2 sitesPoland
- Study dates
- 5 Apr 2019to 28 Feb 2024
- 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 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: The patients with acute intermittent porphyria (AIP) meeting the eligibility criteria and people from the general population, matched by age, sex and body mass index.
Who may be able to join
- acute intermittent porphyria (AIP),
- age 18-65 years,
- at least one hospitalization due to exacerbation of AIP.
Who cannot join
- previous myocardial infarction,
- heart failure of established (other than porphyria) etiology,
- severe heart valve disease,
- congenital heart defects,
- history of myocarditis,
- pacemaker,
Show all 9Show fewer
- hyperthyroidism / hypothyroidism (except for adequate thyroid hormone replacement therapy),
- chronic advanced lung diseases,
- lack of consent to participate in the study.
Trial locations and contacts
2 sites in 1 country.
Poland2 sites
Institute of Hematology and Transfusion Medicine
Warsaw, 02-776
National Institute of Cardiology
Warsaw, 04-628
About this study
Official title: Assessment of the Structure and Function of Heart and Selected Cardiovascular Risk Factors in Patients With Acute Intermittent Porphyria
This study aims to assess the changes in the cardiovascular system in patients with acute intermittent porphyria (AIP).
Read the 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.
- Conditions
- Porphyrias, Hepatic
- Keywords
- acute intermittent porphyria, myocardial injury, heart failure, hypertension, cardiac arrhythmia, cardiovascular risk factors, lipid profile, quality of life
- Medical subject headings
- Porphyrias, Hepatic, Porphyria, Acute Intermittent, Heart Failure, Hypertension, Arrhythmias, Cardiac
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 | |
| Control group | People recruited from the general population and matched by age, sex and body mass index. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with heart failure and left ventricular dysfunction.
Heart failure and left ventricular dysfunction assessed by symptoms and echocardiography.
Measured: day 1 and up to 2 years in case of the exacerbation of AIP
Number of participants with hypertension.
Arterial hypertension diagnosed by ambulatory 24-hour blood pressure monitoring.
Measured: day 1 and up to 2 years in case of the exacerbation of AIP
Rate of cardiac arrhythmias.
Cardiac arrhythmias observed in 24-hour ECG monitoring.
Measured: day 1 and up to 2 years in case of the exacerbation of AIP
Secondary outcomes (6)
Number of participants with increased concentrations of markers of heart failure.
Concentration of N-terminal pro-B-type natriuretic peptide (NT-proBNP) > 125 pg/mL in serum.
Measured: day 1 and up to 2 years in case of the exacerbation of AIP
Number of participants with increased concentrations of markers of myocardial injury.
Concentration of high sensitivity troponin T > 14 ng/L in serum.
Measured: day 1 and up to 2 years in case of the exacerbation of AIP
Number of participants with chronic kidney disease.
Renal function defined by estimated glomerular filtration rate based on the concentrations of creatinine in serum.
Measured: day 1 and up to 2 years in case of the exacerbation of AIP
Number of participants with dyslipidemia.
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.
Measured: day 1
Number of participants with diabetes.
Diabetes in anamnesis or the concentration of glycated hemoglobin > 6.5%.
Measured: day 1
Level of quality of life.
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).
Measured: day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2023
- Study start5 Apr 2019
- Primary completion28 Feb 2024
- Study completion28 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Porphyrias, HepaticStudies of the same condition, open and under way.
- 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 180 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Cardiology, Warsaw, Poland
- Sponsor type
- University, hospital or other
- Collaborators
- Institute of Hematology and Transfusion Medicine, Warsaw
- Responsible party
- Principal investigator, Krzysztof Jaworski, National Institute of Cardiology, Warsaw, Poland
- Investigators
- Krzysztof Jaworski, MD, Principal investigator, National Institute of Cardiology, Warsaw, Poland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05882136
- Sponsor's protocol ID
- 2.39/VII/18
- Acronym
- AIPRACUS
- Status last verified
- Mar 2024
- First submitted
- 3 May 2023
- Last updated
- 2 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Jaworski K, Wasilewski R, Windyga J, Kowalik I, Szwed H, Lipniacka A, Niedolistek M, Was J, Januszewicz A, Kreutz R, Messerli F, Dabrowski R. Hypertension and blood pressure profiles in patients with acute intermittent porphyria: a prospective case-control study. Eur J Intern Med. 2026 May 8:106920. doi: 10.1016/j.ejim.2026.106920. Online ahead of print.
PubMed 42106273Jaworski K, Wasilewski R, Windyga J, Januszewicz A, Szwed H, Dabrowski R. Cardiovascular abnormalities in patients with acute intermittent porphyria: the AIPRACUS study. Pol Arch Intern Med. 2024 Aug 8;134(7-8):16820. doi: 10.20452/pamw.16820. Epub 2024 Aug 8. No abstract available.
PubMed 39115302
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of the Structure and Function of Heart and Selected Cardiovascular Risk Factors in Patients With Acute Intermittent Porphyria
- ClinicalTrials.gov ID
- NCT05882136
- Acronym / sponsor ID
- AIPRACUS / 2.39/VII/18
- Conditions
- Porphyrias, Hepatic
- Keywords
- acute intermittent porphyria, myocardial injury, heart failure, hypertension, cardiac arrhythmia, cardiovascular risk factors, lipid profile, quality of life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 180 participants (actual)
- Lead sponsor
- National Institute of Cardiology, Warsaw, PolandUniversity, hospital or other
- Collaborators
- Institute of Hematology and Transfusion Medicine, Warsaw
- Study start
- 5 Apr 2019 (actual)
- Primary completion
- 28 Feb 2024 (actual)
- Study completion
- 28 Feb 2024 (actual)
- First posted
- 31 May 2023 (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 study aims to assess the changes in the cardiovascular system in patients with acute intermittent porphyria (AIP).
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 design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
| Control group | – | –People recruited from the general population and matched by age, sex and body mass index. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- The patients with acute intermittent porphyria (AIP) meeting the eligibility criteria and people from the general population, matched by age, sex and body mass index.
Key inclusion criteria (3 listed)
- acute intermittent porphyria (AIP),
- age 18-65 years,
- at least one hospitalization due to exacerbation of AIP.
Key exclusion criteria (9 listed)
- previous myocardial infarction,
- heart failure of established (other than porphyria) etiology,
- severe heart valve disease,
- congenital heart defects,
- history of myocarditis,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Heart failure and left ventricular dysfunction assessed by symptoms and echocardiography.
Number of participants with hypertension.
Time frame: day 1 and up to 2 years in case of the exacerbation of AIP
Arterial hypertension diagnosed by ambulatory 24-hour blood pressure monitoring.
Rate of cardiac arrhythmias.
Time frame: day 1 and up to 2 years in case of the exacerbation of AIP
Cardiac arrhythmias observed in 24-hour ECG monitoring.
6 secondary outcomes
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
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
Number of participants with chronic kidney disease.
Time frame: day 1 and up to 2 years in case of the exacerbation of AIP
Number of participants with dyslipidemia.
Time frame: day 1
Number of participants with diabetes.
Time frame: day 1
Level of quality of life.
Time frame: day 1
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 2 Apr 2024.
- Derived from this study: PubMed 42106273Jaworski K, Wasilewski R, Windyga J, Kowalik I, Szwed H, Lipniacka A, Niedolistek M, Was J, Januszewicz A, Kreutz R, Messerli F, Dabrowski R. Hypertension and blood pressure profiles in patients with acute intermittent porphyria: a prospective case-control study. Eur J Intern Med. 2026 May 8:106920. doi: 10.1016/j.ejim.2026.106920. Online ahead of print.
- Derived from this study: PubMed 39115302Jaworski K, Wasilewski R, Windyga J, Januszewicz A, Szwed H, Dabrowski R. Cardiovascular abnormalities in patients with acute intermittent porphyria: the AIPRACUS study. Pol Arch Intern Med. 2024 Aug 8;134(7-8):16820. doi: 10.20452/pamw.16820. Epub 2024 Aug 8. No abstract available.
- Individual participant dataSharing planned: Undecided
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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.
Krzysztof Jaworski, MD principal investigator
National Institute of Cardiology, Warsaw, Poland
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Porphyrias, Hepatic, run by National Institute of Cardiology, Warsaw, Poland at 2 sites in Poland.
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 3 inclusion and 9 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 2 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Poland. 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?
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). 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 NCT05882136, which also lists 2 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 2 Apr 2024.