심근병증을 동반한 트랜스티레틴 아밀로이드증 환자가 타파미디스에서 아코라미디스로 전환 시 트랜스티레틴 혈중 농도 변화에 대한 연구 (ACO-SWITCH)
타파미디스로 이전 치료를 받은 변이형 또는 야생형 트랜스티레틴 아밀로이드증 심근병증(ATTR-CM) 성인 환자에서 아코라미디스 투여 시 혈청 트랜스티레틴(TTR) 수준 변화를 평가하기 위한 전향적, 단일군, 4상 임상시험
Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (variant ATTR-CM).
The study drug, acoramidis, works by attaching itself to the TTR protein, making TTR more stable and less likely to break down and form amyloid (clumps). This helps to slow down the progression of the disease, improve heart function, and increase the TTR levels in the blood. Acoramidis is an approved treatment for wild-type or variant ATTR-CM in Europe and the United States.
Tafamidis is another drug that stabilizes TTR and prevents it from breaking down.
In this study, acoramidis will be studied in participants with ATTR--CM who were previously treated with tafamidis. The main purpose of this study is to assess the change in blood TTR levels after participants are switched from tafamidis to acoramidis. This will be studied to understand if acoramidis causes an increase in blood TTR levels beyond the levels achieved with tafamidis.
For this, the researchers will measure the change in the levels of TTR protein in participants' blood after 6 months of the treatment with acoramidis, or earlier if a participant stops the treatment before reaching that six-month mark.
All participants will continue taking tafamidis during the screening period. In the treatment period of the study, participants will take acoramidis as two tablets twice daily by mouth, for up to 6 months.
At the start of this study, the study doctors will review each participant's medical history and check their overall health. The study doctors will perform electrocardiograms (ECG), and measure blood pressure and heart rate. Researchers will also take blood and urine samples from the participants to measure levels of TTr, NT-proBNP, hs-TnT, hs-CRP, RBP4, eGFR, creatinine, cystatin-C, UACR, and TSH at the start of the study, and at various time points thereafter (during the study) to assess heart, kidney and thyroid function.
There will be a total of 9 study check-ins. Participants will visit the study site twice: at screening and at the end of treatment period. A study nurse will visit the participant's home 6 times, at the start of treatment, Weeks 1, 2, 3 and 4, then again at 3 months. The final check-in will be done by phone.
The study doctors will monitor the health of the participants regularly for any medical problems during follow-up visits. Participants will know the treatment they will receive during the study. Each participant could be in the study for about 8 months.
연구 개요
연구 유형
등록 (추정된)
단계
- 4단계
연락처 및 위치
연구 연락처
- 이름: Bayer Clinical Trials Contact
- 전화번호: 18888422937
- 이메일: clinical-trials-contact@bayer.com
연구 장소
-
-
-
Oslo, 노르웨이, 0424
- 아직 모집하지 않음
- Oslo Universitetssykehus HF, Rikshospitalet
-
-
-
-
-
Berlin, 독일, 10365
- 빼는
- Sana Klinikum Lichtenberg - Klinik für Innere Medizin II, Schwerpunkt Kardiologie
-
Hamburg, 독일, 20246
- 빼는
- Universitätsklinikum Hamburg Eppendorf (UKE)-Universitären Herz- und Gefäßzentrum Hamburg, Klinik für Kardiologie
-
-
Baden-Wurttemberg
-
Ulm, Baden-Wurttemberg, 독일, 89081
- 빼는
- Universitaetsklinikum Ulm | Klinik fuer Innere Medizin II
-
-
Hesse
-
Darmstadt, Hesse, 독일, 64287
- 아직 모집하지 않음
- KVZ Kardiovaskulaeres Zentrum Darmstadt GmbH
-
-
Mecklenburg-Vorpommern
-
Greifswald, Mecklenburg-Vorpommern, 독일, 17475
- 빼는
- Universitaetsmedizin Greifswald KöR- Klinik und Poliklinik für Innere Medizin B
-
-
North Rhine-Westphalia
-
Bonn, North Rhine-Westphalia, 독일, 53127
- 빼는
- Universitätsklinikum Bonn - Herzzentrum des Universitätsklinikums Bonn
-
-
-
-
Brussels Capital
-
Brussels, Brussels Capital, 벨기에, 1070
- 아직 모집하지 않음
- Hôpital Erasme/Erasmus Ziekenhuis
-
-
West Flanders
-
Bruges, West Flanders, 벨기에, 8000
- 아직 모집하지 않음
- AZ St-Jan Brugge-Oostende A.V.
-
-
-
-
Lower Austria
-
Sankt Pölten, Lower Austria, 오스트리아, 1210
- 빼는
- Universitätsklinikum St. Pölten - Lilienfeld - Klinische Abteilung für Innere Medizin 3
-
-
State of Vienna
-
Vienna, State of Vienna, 오스트리아, 3500
- 아직 모집하지 않음
- Klinik Ottakring - 3. Medizinische Abteilung mit Kardiologie, internistischer Intensivmedizin und Ambulanz
-
Vienna, State of Vienna, 오스트리아, 1090
- 모병
- Medizinische Universität Wien- Universitätsklinik für Innere Medizin II, Klinische Abteilung für Kardiologie
-
Vienna, State of Vienna, 오스트리아, 1100
- 모병
- Klinik Favoriten - 5.Medizinische Abteilung-Kardiologie
-
-
Styria
-
Vienna, Styria, 오스트리아, 1030
- 모병
- Medizinische Universität Graz- Klinische Abteilung für Kardiologie
-
-
Upper Austria
-
Braunau am Inn, Upper Austria, 오스트리아, 5280
- 모병
- Krankenhaus St. Josef Braunau | Innere Medizin I
-
-
Vorarlberg
-
Feldkirch, Vorarlberg, 오스트리아, 6800
- 빼는
- VIVIT - Landeskrankenhaus Feldkirch - Innere Medizin I, Kardiologie, Angiologie, Endokrinologie, Diabetologie, Intensivmedizin
-
-
-
-
-
Naples, 이탈리아, 80131
- 아직 모집하지 않음
- Azienda Ospedaliera Dei Colli_Monaldi - Malattie Rare e Genetiche Cardiovascolari
-
-
Apulia
-
Lecce, Apulia, 이탈리아, 73100
- 아직 모집하지 않음
- Ospedale Vito Fazzi Lecce_Cardiologia e UTIC
-
-
Emilia-Romagna
-
Bologna, Emilia-Romagna, 이탈리아, 40138
- 아직 모집하지 않음
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola_Cardiologia
-
-
Lombardy
-
Bergamo, Lombardy, 이탈리아, 24127
- 모병
- Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII - Cardiologia 1
-
Milan, Lombardy, 이탈리아, 20138
- 모병
- Centro Cardiologico Monzino S.p.A. - Cardiologia Peri-Operatoria e Imaging Cardiovascolare
-
Pavia, Lombardy, 이탈리아, 27100
- 아직 모집하지 않음
- Fondazione IRCCS Policlinico San Matteo-SC Medicina Generale 2 - Centro Amiloidosi Sistemiche e Malattie ad Alta Complessità
-
-
Tuscany
-
Florence, Tuscany, 이탈리아, 50134
- 모병
- Careggi University Hospital_cardio intensive care unit
-
Pisa, Tuscany, 이탈리아, 56124
- 모병
- Fondazione Toscana Gabriele Monasterio_Pisa - Cardiologia e Medicina Cardiovascolare
-
Siena, Tuscany, 이탈리아, 53100
- 모병
- Azienda Ospedaliera Universitaria Senese - cardiologia
-
-
Veneto
-
Padova, Veneto, 이탈리아, 35128
- 모병
- Azienda Ospedale-Università di Padova-UOC Cardiologia
-
-
참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Participants must be 18 to 90 years of age inclusive, at the time of signing the informed consent.
- Diagnosis of ATTR-CM; disease defining examination, i.e., Single Photon Emission Computed Tomography (SPECT) or SPECT/Computed Tomography (CT) or biopsy, within 24 months prior to Visit 1 (V1).
- Participants must currently be treated with tafamidis and have used tafamidis for at least the previous 3 months prior to V1 and have adhered to tafamidis therapy.
- New York Heart Association (NYHA) class ≤ II at V1.
- Estimate glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73m^2 at V1.
- N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) > 300 and ≤ 7000 pg/mL at V1.
Exclusion Criteria:
- Prior liver or heart transplantation or planned within the next 12 months.
- Current or planned use of ventricular assist device.
- Active cancer or other disease that decreases the life expectancy to less than one year.
- Heart failure due to ischemic heart disease.
- Myocardial infarction, cardiovascular (CV) surgery, or unstable angina within the last 90 days prior to V1.
- Confirmed diagnosis of light-chain amyloidosis.
- Dialysis or severe renal impairment as reflected by Urinary Albumin Creatinine Ratio (UACR) > 300 mg/g at V1.
- Major surgery within 90 days prior to V1.
- Recent initiation of Sodium-Glucose-Cotransporter-2 inhibitors (SGLT2i) within 3 months before V1.
- Initiation of treatment with a diuretic or increase in diuretic dose within 3 months before V1.
- Treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis.
- Recent CV hospitalization within 3 months before V1.
- Known hypersensitivity to acoramidis or to any of the excipients.
- A condition that, as judged by the investigator, would preclude compliance with the study protocol, such as a history of substance abuse, alcoholism, or a psychiatric condition.
- Known or suspected liver disorder and bile secretion/flow (cholestasis, also history of it).
- Abnormal liver function tests at V1, defined as ALT (GPT) or AST (GOT) ≥ 3 x ULN or total bilirubin ≥ 3 x ULN at V1.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Acoramidis
Participants will receive acoramidis 712 mg orally BID |
356 mg film-coated tablets. The recommended dose of acoramidis is 712 mg (two tablets, 356 mg) orally, twice daily, corresponding to a total daily dose of 1424 mg.
다른 이름들:
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment
기간: From baseline to month 6 or premature discontinuation of treatment
|
TTR = Transthyretin |
From baseline to month 6 or premature discontinuation of treatment
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
TEAE 및 중대한 TEAE가 발생한 참가자 수
기간: 연구 약물의 첫 투여 시점부터 연구 약물의 최종 투여 후 30일 또는 연구 종료 시점 중 먼저 도래하는 시점까지
|
연구 약물의 첫 투여 시점부터 연구 약물의 최종 투여 후 30일 또는 연구 종료 시점 중 먼저 도래하는 시점까지
|
|
|
6개월 기준 크레아티닌 변화
기간: 기준 시점부터 6개월까지
|
기준 시점부터 6개월까지
|
|
|
기저선 대비 6개월 시점의 Cystatin C 변화
기간: 기저선부터 6개월까지
|
기저선부터 6개월까지
|
|
|
Change in serum TTR level from baseline to week 1, 2, 3, 4 and at month 3.
기간: At baseline, week 1, 2, 3, 4 and at month 3
|
TTR = Transthyretin |
At baseline, week 1, 2, 3, 4 and at month 3
|
|
Change from baseline to month 6 of NT-proBNP
기간: From baseline to month 6
|
NT-proBNP = N-terminal pro-B-type Natriuretic Peptide |
From baseline to month 6
|
|
Change from baseline to month 6 of hs-TnT
기간: From baseline to month 6
|
hs-TNT = high-sensitivity Troponin T |
From baseline to month 6
|
|
Change from baseline to month 6 of RBP4
기간: From baseline to month 6
|
RBP4 = Retinol Binding Protein 4 |
From baseline to month 6
|
|
Signs of cardiac remodeling from baseline to month 6
기간: From baseline to month 6
|
These signs will be measured by echocardiogram |
From baseline to month 6
|
|
Change from baseline to month 6 in 6MWD
기간: From baseline to month 6
|
The 6-Minute Walk distance (6MWD) measures the total distance that a participant could walk in 6 minutes. |
From baseline to month 6
|
|
Change from baseline to month 6 in the eGFR
기간: From baseline to month 6
|
eGFR = estimated glomerular filtration rate |
From baseline to month 6
|
|
Change from baseline to month 6 in UACR
기간: From baseline to month 6
|
UACR = Urinary Albumin Creatinine Ratio |
From baseline to month 6
|
|
Change from baseline to month 6 in TSH
기간: From baseline to month 6
|
TSH = Thyroid Stimulating Hormone |
From baseline to month 6
|
|
Change from baseline to month 6 in KCCQ-OS score
기간: From baseline to month 6
|
The Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS) is 23-item questionnaire developed to measure health status and health-related quality of life (QoL) in individuals with heart failure. Items include heart failure symptoms, impact on physical and social functions, and how heart failure impacts QoL. |
From baseline to month 6
|
|
Change from baseline to month 6 of EQ-5D-5L index score
기간: From baseline to month 6
|
The EuroQol-5-Dimension-5-Level (EQ-5D-5L) includes two parts. In the first part, participants are asked to rate their current health state on 5 dimensions (mobility, self-care, usual activities, pain or discomfort, and anxiety or depression) with each dimension having five levels of function (1-no problem, 2-slight problem, 3-moderate problem, 4-severe problem, and 5-extreme problem). The second part is a self-rating of current health status on a Visual Analogue Scale (EQ VAS) with endpoints labeled "best imaginable health state" (score of 100) and "worst imaginable health state" (score of 0). The scores from the 5 dimensions are used to calculate the index score. |
From baseline to month 6
|
공동 작업자 및 조사자
스폰서
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- 23026
- 2025-521831-35-00 (레지스트리 식별자: CTIS (EU))
- 2025 (미국 NIH 보조금/계약)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Currently, there is no established plan for the sharing of Individual Patient Data (IPD) from this study. The availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA 'Principles for responsible clinical trial data sharing.' This pertains to the scope, timepoint, and process of data access.
As such, Bayer commits to considering requests from qualified researchers for patient- / study-level clinical trial data, and documents from clinical trials involving medicines and indications approved in the US and EU. However, this commitment does not reflect an active IPD sharing plan. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.
Researchers can use www.vivli.org to request access to IPD and documents from clinical studies to conduct research. Information on Bayer's criteria for listing studies is provided in the member section of the portal.
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .