EUCTR2019-002835-27-DK进行中（未招募）1 期
A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) - CARDIO –TTRANSFORM
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 1,400
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
- •Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
- •Willing to be genetically tested for mutations in the transthyretin (TTR) gene during screening, if it was not done before
- •Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid [DPD-Tc], 99m Tc-pyrophosphate [PYP-Tc], or 99mTc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
- •End-diastolic interventricular septum thickness of > 12 mm on Screening echocardiogram
- •Medical history of heart failure (HF) secondary to hereditary or wild-type ATTR-CM with at least: A) prior hospitalization for HF, which may include hospitalization for arrhythmia or pacemaker/ICD (implantable cardioverter defibrillator) placement, OR B) symptoms and signs of volume overload or elevated intracardiac pressure that either requires or required treatment with diuretics other than mineralocorticoid receptor antagonists (MRA) for clinical stabilization
- •Screening N-terminal prohormone of brain natriuretic peptide (NTproBNP) = 600 pg/mL (= 70 pmol/L) by central lab. For patients in atrial fibrillation at screening the NT-proBNP values = 1200 pg/mL (= 140 pmol/L)
- •New York Heart Association (NYHA) class I-III
- •6MWT between = 100 meters
- •If on medical treatment for HF on stable dosage regimen for 2 weeks prior to randomization
- •Willingness to adhere to vitamin A supplement per protocol
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 350
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1050
排除标准
- •Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
- •Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
- •Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations in immunoglobulin free light chain (FLC) ratio, unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For patients with CKD and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist, Investigator or Medical Monitor if the risks associated with the biopsy outweigh the benefits
- •Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
- •Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or RNA therapeutic (including siRNA; does not apply to COVID-19 mRNA vaccinations)
- •Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or nondihydropyridine calcium-channel blocker (e.g.,verapamil, diltiazem).
- •Patients receiving any of these agents must respect a Wash-out Period of 14 days before randomization.
- •Abnormal thyroid function tests with clinical significance per Investigator judgement.
- •Contraindication for immunosuppressive therapy, per Investigator's discretion
- •Known history of or positive test for human immunodeficiency virus (HIV) (as evidenced by positive tests for HIV antibody and HIV RNA), hepatitis C (as evidenced by positive tests for HCV antibody and HCV RNA) or hepatitis B (as evidenced by a positive test for hepatitis B surface antigen)
- •History of bleeding, diathesis or coagulopathy (e.g., liver cirrhosis, hematologic malignancy, antiphospholipid antibody syndrome, congenital disorders such as hemophilia A, B, and Von Willebrand disease)
研究者
相似试验
进行中（未招募）
1 期
A study evaluating the efficacy and safety of Etrasimod in the treatment of patients with moderately to severely active Ulcerative Colitislcerative ColitisMedDRA version: 20.1Level: LLTClassification code 10045365Term: Ulcerative colitisSystem Organ Class: 100000004856MedDRA version: 20.1Level: LLTClassification code 10045366Term: Ulcerative colitis, unspecifiedSystem Organ Class: 100000004856EUCTR2018-003985-15-PTArena Pharmaceuticals Inc.433
进行中（未招募）
1 期
A study to test efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravisEUCTR2019-000968-18-CZCB Biopharma SR240
进行中（未招募）
1 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Niraparib Maintenance Treatment in Patients with Advanced Ovarian Cancer Following Response on Front-Line Platinum-Based ChemotherapyHomologous recombination deficiency advanced ovarian cancerMedDRA version: 20.0Level: PTClassification code 10033128Term: Ovarian cancerSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)EUCTR2015-000952-11-ITTESARO, INCORPORATED733
进行中（未招募）
1 期
A Clinical trial to evaluate safety, and effectiveness of Selonsertib in Subjects with Compensated Cirrhosis due to Nonalcoholic Steatohepatitis (NASH)EUCTR2016-004148-13-PTGilead Sciences, Inc.800
招募中
1 期
Placebo-controlled Study Comparing Niraparib Plus Pembrolizumab Versus Placebo Plus Pembrolizumab as Maintenance Therapy in Participants with Advanced/Metastatic Non-Small Cell Lung Cancerung Cancer, Non-Small CellMedDRA version: 21.1Level: PTClassification code: 10029521Term: Non-small cell lung cancer stage IIIB Class: 100000004864MedDRA version: 21.1Level: PTClassification code: 10029522Term: Non-small cell lung cancer stage IV Class: 100000004864MedDRA version: 21.1Level: PTClassification code: 10061873Term: Non-small cell lung cancer Class: 100000004864CTIS2023-508443-40-00Glaxosmithkline Research & Development Limited644