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| ID | Type | Description | Link |
|---|---|---|---|
| KC25MIDV0449 | Other Identifier | Seoul St. Mary's Hospital, The Catholic University of Korea |
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| Name | Class |
|---|---|
| Samsung Medical Center | OTHER |
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This study aims to evaluate the safety and effectiveness of Enavogliflozin 0.3 mg, an SGLT2 inhibitor, in patients with amyloid cardiomyopathy. Participants will take both the study drug and a placebo in two separate periods, with a wash-out period in between. The goal is to determine whether Enavogliflozin is safe and effective for treating amyloid cardiomyopathy.
Patients with a confirmed diagnosis of cardiac amyloidosis and symptomatic heart failure (NYHA class II-III) will be recruited.
Participants will be randomized to receive either the study drug or placebo and will undergo baseline and 12-week assessments, including the Kansas City Cardiomyopathy Questionnaire (KCCQ), 6-minute walk test, and cardiopulmonary exercise testing. Following a 4-week washout period, participants will cross over to the alternate treatment arm (study drug or placebo) and repeat the same assessments at baseline and 12 weeks.
The primary endpoint will be the change in KCCQ Clinical Summary Score (KCCQ-CSS).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Enavogliflozin | Experimental | Administer Enavogliflozin 0.3 mg |
|
| Placebo | Placebo Comparator | Administer placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Enavogliflozin 0.3mg | Drug | Participants will receive Enavogliflozin 0.3 mg orally once daily for 12 weeks. The drug is administered in tablet form and is taken during the first or second treatment period of a crossover design. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in KCCQ-CSS from baseline to Week 12 | Change in the Clinical Summary Score of the Kansas City Cardiomyopathy Questionnaire (KCCQ-CSS) from baseline to 12 weeks after treatment initiation. The KCCQ-CSS is a patient-reported outcome measure that assesses symptom frequency and physical limitation in patients with heart failure. Scores range from 0 to 100, with higher scores indicating better symptom status and physical function (i.e., better health status). A higher change from baseline reflects an improvement in clinical status. | Baseline and Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in 6-Minute Walk Distance | Change in distance walked in 6 minutes from baseline to Week 12. Higher distance indicates better exercise capacity. | Baseline and Week 12 |
| Change in log-transformed NT-proBNP levels |
| Measure | Description | Time Frame |
|---|---|---|
| Change in maximal oxygen consumption (VOâ‚‚ max) | Change in VOâ‚‚ max measured by cardiopulmonary exercise testing (CPET) from baseline to Week 12. Higher values indicate better cardiorespiratory fitness. | Baseline and Week 12 |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jong-Chan Youn, MD, PhD | Contact | 82-2-3147-9853 | jong.chan.youn@gmail.com | |
| Maljoung Ahn, RN | Contact | 82-2-3147-9853 | cmccasc@gmail.com |
| Name | Affiliation | Role |
|---|---|---|
| Jong-Chan Youn, MD, PhD | Seoul St. Mary's Hospital, The Catholic University of Korea | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Samsung Medical Center | Recruiting | Seoul | 06351 | South Korea |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38866445 | Result | Porcari A, Cappelli F, Nitsche C, Tomasoni D, Sinigiani G, Longhi S, Bordignon L, Masri A, Serenelli M, Urey M, Musumeci B, Cipriani A, Canepa M, Badr-Eslam R, Kronberger C, Chimenti C, Zampieri M, Allegro V, Razvi Y, Patel R, Ioannou A, Rauf MU, Petrie A, Whelan C, Emdin M, Metra M, Merlo M, Sinagra G, Hawkins PN, Solomon SD, Gillmore JD, Fontana M. SGLT2 Inhibitor Therapy in Patients With Transthyretin Amyloid Cardiomyopathy. J Am Coll Cardiol. 2024 Jun 18;83(24):2411-2422. doi: 10.1016/j.jacc.2024.03.429. | |
| 37621153 |
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After publication of first manuscript and trial results, the de-identified data will be shared by permission of principle investigator, when asked.
After publication of first manuscript and trial results, the de-identified data will be shared by permission of principle investigator, when asked.
After publication of first manuscript and trial results, the de-identified data will be shared by permission of principle investigator, when asked.
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| ID | Term |
|---|---|
| D028227 | Amyloid Neuropathies, Familial |
| ID | Term |
|---|---|
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D017772 | Amyloid Neuropathies |
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| ID | Term |
|---|---|
| C000729921 | Enavogliflozin |
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Prospective, multi-center, double-blind, randomized controlled, crossover, superiority trial
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| Placebo | Drug | Participants will receive placebo orally once daily for 12 weeks. The drug is administered in tablet form and is taken during the first or second treatment period of a crossover design. |
|
Change in log-transformed NT-proBNP levels after 12 weeks of treatment compared to baseline
| Baseline and Week 12 |
| Change in Body Weight | Change in body weight in kilograms from baseline to Week 12 | Baseline and Week 12 |
| Change in Body Fat Percentage | Change in body fat percentage from baseline to Week 12 | Baseline and Week 12 |
| Change in Extracellular Water Ratio | Change in the ratio of extracellular water to total body water from baseline to Week 12 | Baseline and Week 12 |
| Incidence of Worsening Heart Failure Events or Death | Occurrence of worsening heart failure events (defined as emergency outpatient visit or unplanned hospitalization due to heart failure) or death | From baseline to Week 12 during each treatment period (excluding the 4-week washout period) |
| Seoul St. Mary's Hospital, The Catholic University of Korea | Recruiting | Seoul | 06591 | South Korea |
|
| Result |
| Packer M, Butler J, Zeller C, Pocock SJ, Brueckmann M, Ferreira JP, Filippatos G, Usman MS, Zannad F, Anker SD. Blinded Withdrawal of Long-Term Randomized Treatment With Empagliflozin or Placebo in Patients With Heart Failure. Circulation. 2023 Sep 26;148(13):1011-1022. doi: 10.1161/CIRCULATIONAHA.123.065748. Epub 2023 Aug 24. |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028226 | Amyloidosis, Familial |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000686 | Amyloidosis |
| D057165 | Proteostasis Deficiencies |