ClinicalTrials.gov record NCT05797857
Exercise Training in Transthyretin Cardiac Amyloidosis
Personalized Exercise Training to Improve Functional Capacity in Transthyretin Cardiac Amyloidosis
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This single-group interventional study tests a 12-week personalized, supervised exercise program for people with transthyretin cardiac amyloidosis. It measures aerobic capacity, lower-extremity function, and quality of life before and after the program. Exercise sessions are two supervised 60-minute sessions per week.
What it is studying. The study tests whether a supervised, personalized 12-week exercise program (two 60-minute sessions per week) changes aerobic capacity (peak VO2), lower-extremity function, and health-related quality of life in people with transthyretin cardiac amyloidosis. As a related aim, it compares skeletal muscle performance between people with ATTR-CM and people with non-amyloid heart failure.
What the researchers hope to learn. To determine if targeted exercise training changes peak VO2, Kansas City Cardiomyopathy Questionnaire scores, and short physical performance battery scores in transthyretin cardiac amyloidosis, and to clarify skeletal muscle contribution to exercise intolerance.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Other
- Exercise Training
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
Exclusion criteria
- 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
- High risk for non-adherence as determined by screening evaluation
- Inability or unwillingness to comply with the study requirements
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
Heart failure (HF) affects over 5 million adults over the age of 65. Cardiac transthyretin amyloidosis (ATTR-CM) is a cause of HF in ~10% of older adults and leads to significant morbidity and mortality. Exercise intolerance is traditionally attributed to cardiac dysfunction but the contribution of other systems to this has not been studied. Musculoskeletal involvement is common in ATTR-CM and occur 5-10 years prior to onset of HF. Tafamidis, a transthyretin stabilizer, is the only approved treatment for ATTR-CM. It slows disease progression, prolongs life, and reduces HF hospitalizations. However, it does not improve functional capacity- no therapeutic intervention has been shown to do so in ATTR-CM. The idea behind this project is that skeletal muscle dysfunction from amyloidosis and HF severely limits exercise capacity and, thus, quality of life in ATTR-CM, and that targeted exercise training will improve quality of life by improving skeletal muscle performance and aerobic capacity. Cardiopulmonary exercise testing (CPET) and the short physical performance battery (SBBP), including a leg extensor muscle power assessment will be used to achieve the following specific aims; 1) to compare skeletal muscle performance in ATTR-CM and non-amyloid HF; and 2) to determine improvements in aerobic capacity and quality of life due to 12 weeks of supervised exercise training in patients with ATTR-CM. To achieve the second aim, we will use a personalized exercise intervention.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants take part in a supervised, personalized exercise program: two 60‑minute training sessions per week for 12 weeks. Study visits include baseline, a 4‑week check (questionnaire), and a 12‑week assessment with cardiopulmonary exercise testing (CPET) to measure peak VO2, physical function tests (SPPB and leg extensor power), and quality‑of‑life questionnaires (KCCQ). CPET or other assessments could reveal findings that require further evaluation and might affect participation. You must be willing and able to attend sessions, complete testing, and follow study instructions.
How the study is structured. Interventional, single-group (all participants receive the same program), unmasked study. Participants follow a personalized supervised exercise training program for 12 weeks. Key measurements are cardiopulmonary exercise testing (peak VO2) at baseline and 12 weeks, and quality-of-life and lower-extremity function assessments at baseline, 4 weeks (KCCQ), and 12 weeks.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on wild-type ATTR amyloidosis
This trial is also listed on this site under ATTRv amyloidosis.
Important dates
- Study start
- Jul 10, 2024
- Primary completion
- Jun 30, 2029 (estimated)
- Study completion
- Jun 30, 2030 (estimated)
- Target enrollment
- 40 participants (estimated)
- Last updated on registry
- Mar 18, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Brigham and Women's Hospital
- Principal investigator
- Sarah Cuddy, MD, Cardiologist
Brigham and Women's Hospital
Participating sites (1)
- Brigham and Women's HospitalRecruiting
Boston, Massachusetts, 02115 · United States
- Sarah Cuddy, MD · 6177328410 · scuddy1@bwh.harvard.edu
Terms used on this page · explanations by MedicalUpdates.ai
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- control arm
- The group the study treatment is compared with. It may receive a placebo, an existing treatment (active comparator), a sham procedure, or no intervention.
- enrolment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- biomarkers
- Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
- estimated glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- life expectancy
- Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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