2024-518899-31-00招募中3 期
CLEOPATTRA: Effects of NNC6019-0001 versus placebo on cardiovascular outcomes in participants with transthyretin amyloid cardiomyopathy (ATTR-CM).
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 165
- 试验地点
- 21
- 主要终点
- Number of occurrences of the composite endpoint consisting of: • CV death • Recurrent CV events (CV hospitalisation and urgent HF visits )
研究概览
简要总结
To demonstrate superiority of NNC6019-0001 versus placebo, both added to SoCa, in reducing CV death and morbidity in participants with ATTRwt‑CM or ATTRv‑CM
研究设计
- 分配方式
- Randomized
- 主要目的
- Follow-up
- 盲法
- Double (Investigator, Analyst, Monitor, Subject, Carer)
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Male or female.
- •Age 18 years or above at the time of signing the informed consent.
- •Have an established diagnosis of ATTR-CM, (ATTRwt or ATTRv), with cardiac amyloid infiltration, increased left ventricular (LV) wall thickness, and HF. Note: Target ATTRv recruitment is approximately 15% of the study population. a. Cardiac amyloid infiltration demonstrated by: i. Cardiac biopsy positive for TTR amyloid, OR, ii. Grade 2 or 3 cardiac uptake at PYP/DPD/HMDP scintigraphy with single-photon emission computed tomography (SPECT/CT) combined with an extracardiac biopsy positive for TTR amyloid, OR, iii. Grade 2 or 3 cardiac uptake at PYP/DPD/HMDP scintigraphy with SPECT/CT combined with normal serum free light chain ratio, and negative serum and urine protein electrophoresis with immunofixation (SPIE & UPIE). Notes: o Non-invasive diagnostic pathway will be confirmed by a centralised expert review. o Bone tracer scintigraphy will be conducted using 99m-technetium (Tc)-labelled pyrophosphate (99mTc-PYP)/99mTc-labelled 3,3-diphosphono-1,2-propanodicarboxylic acid (99mTc DPD)/99mTc-labeled hydroxymethylene diphosphonate (99mTc-HMDP). b. Increased LV wall thickness, as assessed by centralised review of echocardiography, showing interventricular septal wall thickness ≥12 mm. c. Chronic HF (New York Heart Classification [NYHA] I-IV) requiring ongoing treatment with a loop diuretic with: i. At least 1 documented hospitalisation for HF, OR ii. History of HF manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath, signs of pulmonary congestion on x-ray or auscultation, or peripheral oedema).
- •Expected to be on stable CV medical therapy (defined as no greater than 50% dose adjustment and no categorical changes of medications), with the exception of diuretics, 4 weeks prior to the randomisation visit.
- •NT-proBNP concentration ≥"CCI" pg/mL at screening. Note: Participants with NT-proBNP levels between "CCI" and "CCI" pg/mL may be enrolled until a cap of 35% of the total study population is reached.
- •Completed >50 meters on the 6MWT at screening.
排除标准
- •Known or suspected hypersensitivity to study intervention(s) or related products.
- •Prior solid organ transplant or planned solid organ transplant during the study.
- •Left ventricular ejection fraction (LVEF) <30% as assessed by centralised review of echocardiography.
- •Presence or history of malignant neoplasm (other than basal or squamous cell skin cancer, in situ carcinomas of the cervix, or carcinoma in situ/high-grade prostatic intraepithelial neoplasia (PIN), low-risk prostate cancer or on stable therapy for prostate cancer) within 3 years before screening.
- •End stage renal disease (estimated glomerular filtration rate (eGFR) <15 ml/min/1.73m2 at screening, or chronic/intermittent haemodialysis or peritoneal dialysis).
- •Current or previous participation (dosing with active treatment) in a study for an investigational ATTR depleting drug or ATTR gene editing therapy.
- •Total bilirubin >3 × upper limit of normal (ULN) at screening.
- •Current diagnosis or history of amyloid light chain, other non-ATTR amyloidosis or known leptomeningeal amyloidosis, or multiple myeloma.
- •HF not primarily caused by ATTR-CM, for example, due to hypertension, valvular heart disease, or ischemic heart disease in the opinion of the investigator.
- •Currently hospitalised or hospitalised within 14 days prior to screening.
- •Currently treated with positive inotropic medication.
- •Uncorrected, severe, haemodynamically significant, left-sided heart valve disease. Note: pre existing echocardiogram up to 2 years old may be used.
- •Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularisation, cardiac device implantation, cardiac valve repair, or major surgery within 60 days of screening.
结局指标
主要结局
Number of occurrences of the composite endpoint consisting of: • CV death • Recurrent CV events (CV hospitalisation and urgent HF visits )
Number of occurrences of the composite endpoint consisting of: • CV death • Recurrent CV events (CV hospitalisation and urgent HF visits )
次要结局
- Secondary Confirmatory: Change in KCCQ-CSS
- Change in 6MWD
- Number of occurrences of CV events (CV hospitalisation and urgent HF visits)
- Time to occurrence of CV death
- Time to occurrence of all-cause death
- Time to first occurrence of composite CKD endpoint: • CV death • Onset of persistent decline eGFR ≥ 30% • Onset of persistent eGFR < 15 mL/min/1.73m2 • Initiation of chronic kidney replacement therapy (dialysis or kidney transplant)
- Change in KCCQ-OSS
研究者
EU Submission Hub
Scientific
Novo Nordisk A/S
研究点 (21)
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