Exercise Training in Transthyretin Cardiac Amyloidosis

NCT05797857 | Recruiting

• 1 other identifier: 2022p002754
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

Transthyretin cardiac amyloidosis causes debilitating heart failure in older adults. The proposed research will develop a personalized exercise training program to improve functional capacity in patients on optimal treatment for transthyretin cardiac amyloidosis. This is a vital next step to improve functional capacity and quality of life of people suffering from transthyretin cardiac amyloidosis.

Conditions

Amyloid Cardiomyopathy; Transthyretin Cardiac Amyloidosis

Outcome Measures

Primary Outcomes (1)

• peak oxygen consumption (VO2)

  CPET performed at baseline and 12-weeks, following the exercise intervention will be used to measure aerobic capacity, peak VO2. The change in peak VO2 from baseline to 12 weeks is the primary outcome measure. An increase of \> 1.0 ml/kg/min is considered a clinically meaningful increase

  12 weeks

Secondary Outcomes (2)

• Kansas City Cardiomyopathy Questionnaire

  12 weeks

• Lower extremity function

  12 weeks

Study Arms (1)

Intervention arm

EXPERIMENTAL

Other: Exercise Training

Interventions

Exercise Training OTHER

A supervised, personalized exercise training program, which will consist of two 60-minute exercise training sessions per week.

Intervention arm

Eligibility Criteria

• Age: 60 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis and typing of ATTR-CM by endomyocardial biopsy or by Grade 2 or Grade 3 pyrophosphate (PYP) positivity (exception: nonamyloid control arm in aim 1).
• Diagnosis of heart failure, with prior or current need of diuretics and increased N-terminal prohormone B-natureitic peptide (BNP) (≥450 pg/ml).
• Peak VO2 \<80% predicted, indicating impaired aerobic capacity (for aim 2 only).
• Taking tafamidis (for aim 2 only)
• Able to walk 4 meters (with or without the use of an assistive device) and independent with basic activities of daily living at the time of enrolment.
• Adequate clinical stability has been achieved in the judgment of the investigator to allow participation in study assessments and the intervention.
• Signed informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study.

You may not qualify if:

• Acute myocardial infarction (Note: given that cardiac biomarkers such as troponin are frequently elevated in ATTR-CM patients, the diagnosis of acute myocardial infarction should be based on clinical diagnosis, not biomarkers alone)
• \>70% obstructive coronary artery disease
• Severe aortic valve stenosis
• Already actively participating in formal, facility-based cardiac exercise
• Already engaging in regular moderate to vigorous exercise conditioning defined as \> 30 minutes per day, ≥ twice per week consistently during the previous 6 weeks
• Ventricular assist device
• Light chain amyloidosis or other form of non-ATTR amyloidosis
• Advanced chronic kidney disease defined as estimated glomerular filtration rate \<20 mL/min/1.73m2
• Any organ transplantation
• Terminal illness other than HF with life expectancy \< 1 year
• Pacemaker or implantable cardioverter-defibrillato (ICD) with heart rate limits \< expected heart rates for exercise and unable to be reprogrammed
• Neuropathy due to transthyretin (TTR) mutation
• Impairment from stroke, injury or other medical disorder that precludes participation in the intervention
• Abnormal cardiopulmonary exercise testing (CPET) finding that requires further investigation and management
• Dementia that precludes ability to participate in exercise and follow study protocols

Sponsors & Collaborators

• Brigham and Women's Hospital: lead

Study Sites (1)

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

Exercise

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Cardiologist

Study Record Dates

• First Submitted: March 22, 2023
• First Posted: April 4, 2023
• Study Start: July 10, 2024
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): June 30, 2030
• Last Updated: March 18, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)