Transthyretin cardiac amyloidosis (TTR-CA) is a heart disease that mainly affects older adults and often leads to reduced physical capacity, muscle weakness, frailty, and a decline in quality of life. While current medical treatments can slow disease progression, they do not fully address functional limitations or muscle deterioration. The EFICAC-TTR study is a prospective, randomized, multicenter clinical trial designed to evaluate whether a combined non-pharmacological intervention can improve physical function in patients aged 70 years or older with confirmed TTR-CA. A total of 102 participants will be randomly assigned to one of three groups: (1) usual medical care, (2) a home-based multicomponent exercise program combined with fiber supplementation, or (3) the same exercise program combined with creatine monohydrate and β-hydroxy-β-methylbutyrate (HMB) supplementation. The exercise program is adapted to each participant's functional level and is performed at home. The main outcomes of the study are changes in walking capacity, measured by the 6-minute walk test, and muscle strength, assessed by handgrip strength after 12 weeks. Secondary outcomes include changes in body composition, frailty, quality of life, and clinical events, while mechanistic biomarkers are assessed as exploratory outcomes. This study aims to determine whether combining exercise with nutritional supplementation can safely improve functional capacity and overall health in older adults with transthyretin cardiac amyloidosis.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
102
Standard clinical management for transthyretin cardiac amyloidosis according to routine cardiology practice.
A 12-week home-based multicomponent exercise program adapted from the Vivifrail model, including strength, balance, mobility, and endurance exercises tailored to individual functional capacity.
Daily oral supplementation with microcrystalline cellulose, used as a nutritionally inert control supplement to match supplementation procedures.
Daily oral supplementation with creatine monohydrate (3 g/day) and β-hydroxy-β-methylbutyrate (HMB, 3 g/day) for 12 weeks.
Change in Handgrip Strength
Change in maximal isometric handgrip strength measured in kilograms (kg) using a calibrated handheld dynamometer (best of three attempts for the dominant hand), expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in 6-Minute Walk Test Distance
Change in walking capacity assessed by the 6-minute walk test, measured as the difference in total distance walked (meters) between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in Fat Mass
Change in total body fat mass measured in kilograms (kg) using multifrequency bioelectrical impedance analysis, expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in Skeletal Muscle Mass
Change in skeletal muscle mass measured in kilograms (kg) using multifrequency bioelectrical impedance analysis, expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in Fat-Free Mass
Change in fat-free mass measured in kilograms (kg) using multifrequency bioelectrical impedance analysis, expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in Phase Angle
Change in phase angle measured in degrees (°) using multifrequency bioelectrical impedance analysis, expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in Frailty Status Assessed by the FRAIL Scale
Change in frailty status assessed by the FRAIL scale, a 5-item questionnaire with scores ranging from 0 to 5, where higher scores indicate greater frailty, expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
All-Cause Mortality
All-cause mortality, defined as death from any cause during the follow-up period.
Time frame: Up to 6 months
Change in Short Physical Performance Battery (SPPB) Score
Change in Short Physical Performance Battery (SPPB) total score, ranging from 0 to 12 points, where higher scores indicate better physical performance, expressed as the difference between baseline and 12 weeks.
Time frame: Baseline and 12 weeks
Change in Clinical Frailty Scale (CFS) Score
Change in frailty severity assessed by the Clinical Frailty Scale (CFS), a 9-point ordinal scale ranging from 1 (very fit) to 9 (terminally ill), where higher scores indicate greater frailty.
Time frame: Baseline and 12 weeks
Change in Barthel Index Score
Change in functional independence assessed by the Barthel Index, scored from 0 to 100, where higher scores indicate greater independence.
Time frame: Baseline and 12 weeks
Change in SARC-F Score
Change in sarcopenia risk assessed by the SARC-F questionnaire, with scores ranging from 0 to 10, where higher scores indicate greater sarcopenia risk.
Time frame: Baseline and 12 weeks
Change in Minnesota Living With Heart Failure Questionnaire Score
Change in health-related quality of life assessed by the Minnesota Living With Heart Failure Questionnaire (MLHFQ), with total scores ranging from 0 to 105, where higher scores indicate worse quality of life.
Time frame: Baseline and 12 weeks
Change in Charlson Comorbidity Index
Change in comorbidity burden assessed using the Charlson Comorbidity Index, a weighted index that accounts for the number and severity of comorbid conditions, where higher scores indicate greater comorbidity burden.
Time frame: Baseline and 12 weeks
Incidence of Heart Failure Hospitalizations
Number of hospitalizations due to heart failure occurring during the follow-up period.
Time frame: Up to 6 months
Incidence of Non-Heart Failure Hospitalizations
Hospital admissions due to causes other than heart failure during the follow-up period.
Time frame: Up to 6 months
Incidence of Emergency Department Visits
Number of emergency department visits during follow-up.
Time frame: Up to 6 months
Incidence of Intervention-Related Adverse Events
Number and type of adverse events related to exercise and/or supplementation, classified according to severity and relatedness, collected throughout the follow-up period.
Time frame: Up to 6 months
Rate of Supplement Discontinuation
Proportion of participants who discontinue study supplements.
Time frame: Up to 6 months
Exercise Program Adherence
Proportion (%) of prescribed exercise sessions completed, with adequate adherence defined as completion of at least 80% of prescribed sessions.
Time frame: Up to 12 weeks
Supplement Adherence
Proportion (%) of planned supplement doses consumed, with optimal compliance defined as 90-110%.
Time frame: Up to 12 weeks
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