N-of-1 for Beta-Blockers in Cardiac Amyloidosis

Launched by WEILL MEDICAL COLLEGE OF CORNELL UNIVERSITY · Aug 17, 2021

Keywords

ClinConnect Summary

This trial will test whether stopping beta-blockers helps people with transthyretin cardiac amyloidosis feel better. In twenty older adults (65+), researchers will run two study periods, each up to 6 weeks. In the first period they stay on their usual beta-blocker; in the second period they gradually reduce the dose by 50% each week until they stop or reach a lowest safe dose. The study will measure how this change affects exercise ability (using a peak oxygen test), and how patients feel in daily life, health, and quality of life.

Eligible participants are ambulatory adults 65 and older with confirmed TTR cardiac amyloidosis who are already taking a beta-blocker and do not have other strong reasons to keep taking it. The study will be done at Weill Cornell Medicine in New York, and involves basic tests and questionnaires at several points (baseline, during each period, and after the end). After Period 2, participants decide with their doctors whether to continue or stop the beta-blocker long-term. The trial is a single-arm N-of-1 design, focusing on the individual’s experience, with results to help understand deprescribing beta-blockers in this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Ambulatory adults age ≥65 years with TTR-cardiac amyloidosis (confirmed by PYP scan or biopsy)
• • 2. Taking beta-blocker
• Exclusion Criteria:
• 1. Other compelling indication(s) for beta-blockers:
• • 1. Angina
• • 2. Acute coronary syndrome, myocardial infarction or coronary artery bypass surgery in prior 3 years
• • 3. Atrial arrhythmia with hospitalization for rapid ventricular response, prior 1 year
• • 4. Heart rate \>100 bpm within the prior 3 months
• • 5. Atrial arrhythmia with ventricular rate \>90 beats per minute within the prior 3 months
• • 6. Systolic blood pressure readings \>160 mmHg within the prior 1 month, unless classified as white coat hypertension/effect (e.g., migraine prevention, anxiety symptom management, hyperthyroidism, essential tumor reduction)
• • 2. Clinical instability (N-of-1 trials are appropriate for stable conditions only)
• • 1. Decompensated heart failure
• • 2. Hospitalized in the past 30 days
• • 3. Medication changes or procedures in the prior 14 days that could confound observations/data at Principal Investigator discretion
• • 4. Anticipated medication changes or procedures in subsequent 3 months that could confound observations/data at PI discretion
• • 5. Clinical instability from other medical issues
• • 3. Estimated life expectancy \<6 months
• • 4. Moderate-severe dementia or psychiatric disorder precluding informed consent
• • 5. Language barrier that will preclude informed consent and ability to comprehend study procedures
• • 6. Non-compliance or inability to complete study procedures
• • 7. Enrollment in a clinical trial not approved for co-enrollment
• • 8. Any condition that, in Principal Investigator or treating physician's opinion, makes the patient unsuitable for study participation