A Prospective, Randomized, Multicenter Clinical Trial on the Effect of a Home-Based Multicomponent Exercise Program Combined With Nutritional Supplementation in Patients With Transthyretin Cardiac Amyloidosis
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 102
- 主要终点
- Change in Handgrip Strength
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The study is open-label. Outcome assessors are blinded to group allocation.
入排标准
- 年龄范围
- 70 Years 至 —（Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥70 years.
- •Confirmed diagnosis of transthyretin cardiac amyloidosis (TTR-CA) based on positive bone scintigraphy with diphosphonates (Perugini grade 2 or 3) and absence of monoclonal protein.
- •Clinical stability during the 4 weeks prior to enrollment.
- •Ability to understand the study procedures and provide written informed consent.
排除标准
- •Light-chain (AL) amyloidosis or other non-TTR amyloidosis variants.
- •Absolute medical contraindication to moderate-intensity exercise.
- •Severe comorbid conditions with an estimated life expectancy <6 months.
- •Severe cognitive impairment (Mini-Mental State Examination score <20).
- •Concurrent participation in another clinical trial or structured exercise program.
- •Known allergy or intolerance to creatine, beta-hydroxy-beta-methylbutyrate (HMB), or microcrystalline cellulose.
- •Severe renal impairment requiring dialysis.
研究组 & 干预措施
Usual Care
Participants receive usual medical care for transthyretin cardiac amyloidosis, including routine cardiology follow-up and general lifestyle recommendations, without structured exercise or nutritional supplementation.
干预措施: Usual Care (Other)
Exercise + Fiber Supplementation (Active Control)
Participants perform a home-based multicomponent exercise program and receive daily supplementation with microcrystalline cellulose, used as a nutritionally inert control supplement.
干预措施: Home-Based Multicomponent Exercise Program (Behavioral)
Exercise + Fiber Supplementation (Active Control)
Participants perform a home-based multicomponent exercise program and receive daily supplementation with microcrystalline cellulose, used as a nutritionally inert control supplement.
干预措施: Fiber Supplementation (Microcrystalline Cellulose) (Dietary Supplement)
Exercise + Creatine and HMB Supplementation
Participants perform a home-based multicomponent exercise program and receive daily supplementation with creatine monohydrate (3 g/day) and β-hydroxy-β-methylbutyrate (HMB, 3 g/day).
干预措施: Home-Based Multicomponent Exercise Program (Behavioral)
Exercise + Creatine and HMB Supplementation
Participants perform a home-based multicomponent exercise program and receive daily supplementation with creatine monohydrate (3 g/day) and β-hydroxy-β-methylbutyrate (HMB, 3 g/day).
干预措施: Creatine and HMB Supplementation (Dietary Supplement)
结局指标
主要结局
Change in Handgrip Strength
时间窗: Baseline and 12 weeks
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.
Change in 6-Minute Walk Test Distance
时间窗: Baseline and 12 weeks
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.
次要结局
- Change in Fat Mass(Baseline and 12 weeks)
- Change in Skeletal Muscle Mass(Baseline and 12 weeks)
- Change in Fat-Free Mass(Baseline and 12 weeks)
- Change in Phase Angle(Baseline and 12 weeks)
- Change in Frailty Status Assessed by the FRAIL Scale(Baseline and 12 weeks)
- All-Cause Mortality(Up to 6 months)
- Change in Short Physical Performance Battery (SPPB) Score(Baseline and 12 weeks)
- Change in Clinical Frailty Scale (CFS) Score(Baseline and 12 weeks)
- Change in Barthel Index Score(Baseline and 12 weeks)
- Change in SARC-F Score(Baseline and 12 weeks)
- Change in Minnesota Living With Heart Failure Questionnaire Score(Baseline and 12 weeks)
- Change in Charlson Comorbidity Index(Baseline and 12 weeks)
- Incidence of Heart Failure Hospitalizations(Up to 6 months)
- Incidence of Non-Heart Failure Hospitalizations(Up to 6 months)
- Incidence of Emergency Department Visits(Up to 6 months)
- Incidence of Intervention-Related Adverse Events(Up to 6 months)
- Rate of Supplement Discontinuation(Up to 6 months)
- Exercise Program Adherence(Up to 12 weeks)
- Supplement Adherence(Up to 12 weeks)