Observational Study on Cardiac Biomarkers Testing in Patients with Muscular Dystrophy Cardiomyopathy (KYMA)

Observational Study on Cardiac Biomarkers Testing in Patients with Muscular Dystrophy Cardiomyopathy and Assessing the Feasibility of Serial Cardiac Troponin Testing At 0 Hour and 1 Hour, As Well As the Mid-term Biovariability of Cardiac Troponin

The objective of this study is to evaluate acute changes of cardiac troponin (and other cardiac biomarkers) and mid-term biovariability in patients with cardiomyopathy associated with chronic skeletal muscle disease. The specific aims of the study are:

Firstly, to evaluate the feasibility of the ESC 0/1 hour protocol for rule-in and rule-out of a non-ST-segment elevation acute coronary syndrome (NSTE-ACS).

Secondly, a) to determine reference change values (RCV) to characterize physiological biovariability, b) to differentiate acute from chronic high-sensitivity cardiac troponin T (hs-cTnT) elevations.

Study Overview

• Status: Completed
• Conditions: Cardiomyopathies; Skeletal Muscle Disorder
• Intervention / Treatment: Other: No intervention is planned.

Detailed Description

In patients with skeletal muscle dystrophy, cardiac involvement is one of the limiting factors regarding mortality. Early detection of a dystrophy-associated cardiomyopathy is important, as heart failure therapy can slow adverse cardiac remodeling and alleviate heart failure symptoms. Cardiac biomarkers, especially high-sensitivity cardiac Troponin T (hs-cTnT) and to a lesser extent, high-sensitivity cardiac Troponin I (hs-cTnI), are chronically elevated in diseases with underlying structural heart disease. However, there is no reliable data on whether changes in the concentration of cardiac Troponin T or I are suitable for monitoring the cardiac disease progression. This can lead to delayed diagnosis and treatment of a relevant deterioration of the disease or even acute heart muscle damage in the context of acute myocardial infarction, pulmonary embolism, and myocarditis. Due to insufficient clinical data, the feasibility and safety of the ESC 0/1-hour protocol according to the ESC guidelines for the diagnosis of acute myocardial infarction in this patient population are uncertain. Furthermore, it is necessary to determine the normal biovariability of high-sensitivity cardiac Troponin T (hs-cTnT) and high-sensitivity cardiac Troponin I (hs-cTnI) to differentiate physiological concentration fluctuations of these cardiac biomarkers in this patient population from changes that represent an improvement or deterioration of heart function. Routine blood samples that will be collected at presentation, a second blood sample for study purposes will be collected after 1 hour for the first index visit. On the routine follow-up visit, 20 ml of blood will be collected for storage in addition to the routine blood test. Blood samples will be obtained by standard venous blood sampling. If venipuncture is unsuccessful or considered harmful by the patient and a suitable peripheral venous catheter is available, the venous catheter may be used for blood sampling to minimize damage. Assessment of parameters includes demographics and clinical characteristics (symptoms, history, medication, vital signs, risk scores etc.) and laboratory values with special focus on findings related to myocardial injury as indicated by high sensitivity cardiac troponin T (hs-cTnT) and hs-cTnI. Moreover, the laboratory panel comprises other cardiac biomarkers, electrolytes, renal (creatine, glomerular filtration rate) and liver function (GOT, GPT, LDH), C-reactive protein, D-dimer etc.

• Study Type: Observational
• Enrollment (Actual): 35

Contacts and Locations

Study Locations

• Germany
  • Baden-Württemberg
    • Heidelberg, Baden-Württemberg, Germany, 69120
      • University Hospital of Heidelberg

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

All consecutive patients with cardiomyopathy associated with chronic skeletal muscle disease presenting at Heidelberg University Hospital in the cardiomyopathy outpatients' clinic during a time period of 12 months are eligible for study inclusion.

Description

Inclusion Criteria:

• Written informed consent
• Diagnosed muscular dystrophy
• Patients >18 years of age

Exclusion Criteria:

• Patients with poor venous status
• Hemodialysis
• Lack of capacity to provide informed consent or refusal

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Prevalence of stable troponin kinetics in patients	The first main objective is to determine the prevalence of stable troponin kinetics defined as high-sensitivity Troponin T (hour 0) : high-sensitivity Troponin T (hour 1) < 5 ng/l at baseline presentation.	1 hour
Reference Change Values calculation for cardiac biomarkers	The second main objective is to calculate the Reference Change Values (RCV) of high-sensitivity Troponin T within the 6-month follow-up period.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Longitudinal follow-up of novel laboratory biomarkers for prognostic significance.	Thirdly, longitudinal follow-up of the patients will be conducted once a year to clarify the prognostic significance of novel laboratory biomarkers.	unlimited

Collaborators and Investigators

• Sponsor: University Hospital Heidelberg

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2023
• Primary Completion (Actual): December 31, 2024
• Study Completion (Actual): December 31, 2024

Study Registration Dates

• First Submitted: March 7, 2023
• First Submitted That Met QC Criteria: March 19, 2023
• First Posted (Actual): March 22, 2023

Study Record Updates

• Last Update Posted (Actual): March 30, 2025
• Last Update Submitted That Met QC Criteria: March 25, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Cardiovascular Diseases; Heart Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Muscular Disorders, Atrophic; Cardiomyopathies; Muscular Dystrophies; Muscular Diseases
• Other Study ID Numbers: KYMA

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No