NCT05882136

Brief Summary

This study aims to assess the changes in the cardiovascular system in patients with acute intermittent porphyria (AIP).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
180

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Apr 2019

Longer than P75 for all trials

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 5, 2019

Completed
4.1 years until next milestone

First Submitted

Initial submission to the registry

May 3, 2023

Completed
28 days until next milestone

First Posted

Study publicly available on registry

May 31, 2023

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 28, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 28, 2024

Completed
Last Updated

April 2, 2024

Status Verified

March 1, 2024

Enrollment Period

4.9 years

First QC Date

May 3, 2023

Last Update Submit

March 30, 2024

Conditions

Porphyrias, Hepatic

Keywords

acute intermittent porphyriamyocardial injuryheart failurehypertensioncardiac arrhythmiacardiovascular risk factorslipid profilequality of life

Outcome Measures

Primary Outcomes (3)

  • Number of participants with heart failure and left ventricular dysfunction.

    Heart failure and left ventricular dysfunction assessed by symptoms and echocardiography.

    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.

    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.

    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.

    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.

    day 1 and up to 2 years in case of the exacerbation of AIP

  • Number of participants with chronic kidney disease.

    day 1 and up to 2 years in case of the exacerbation of AIP

  • Number of participants with dyslipidemia.

    day 1

  • Number of participants with diabetes.

    day 1

  • +1 more secondary outcomes

Study Arms (2)

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.

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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.

You may qualify if:

  • acute intermittent porphyria (AIP),
  • age 18-65 years,
  • at least one hospitalization due to exacerbation of AIP.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Institute of Hematology and Transfusion Medicine

Warsaw, 02-776, Poland

Location

National Institute of Cardiology

Warsaw, 04-628, Poland

Location

Related Publications (1)

  • Jaworski 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.

    PMID: 39115302DERIVED

MeSH Terms

Conditions

Porphyrias, HepaticPorphyria, Acute IntermittentHeart FailureHypertensionArrhythmias, Cardiac

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesPorphyriasMetabolic DiseasesNutritional and Metabolic DiseasesHeart DiseasesCardiovascular DiseasesVascular DiseasesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Krzysztof Jaworski, MD

    National Institute of Cardiology, Warsaw, Poland

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

May 3, 2023

First Posted

May 31, 2023

Study Start

April 5, 2019

Primary Completion

February 28, 2024

Study Completion

February 28, 2024

Last Updated

April 2, 2024

Record last verified: 2024-03

Locations

PL(2)