A Phase 3 Non-randomized, Non-controlled, Open Label Clinical Study to Evaluate the Efficacy and Safety of MK-7962 (Sotatercept) add-on to Background Therapy in Japanese Participants With Pulmonary Arterial Hypertension (PAH)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 46
- 试验地点
- 17
- 主要终点
- Change From Pulmonary Vascular Resistance (PVR) From Baseline at Week 24
研究概览
简要总结
This local Phase 3 study is planned to confirm the efficacy and safety in Japanese PAH participants. The primary population of this study is Japanese PAH participants with World Health Organization Functional Class (WHO FC) II or III while the study includes PAH participants with WHO FC I or IV as other populations. There are no hypotheses for this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnostic right heart catheterization (RHC) at any time prior to screening confirming the diagnosis of World Health Organization (WHO) pulmonary arterial hypertension (PAH) Group 1 in any of the following subtypes:
- •Idiopathic PAH
- •Heritable PAH
- •Drug/toxin-induced PAH
- •PAH associated with connective tissue disease
- •PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair
- •PAH classified as WHO functional class (FC) I or symptomatic PAH classified as WHO FC II to IV
- •On stable doses of background PAH therapy and diuretics (if applicable) for at least 90 days prior to screening
排除标准
- •Diagnosis of PH WHO Groups 2, 3, 4, or 5
- •Diagnosis of the following PAH Group 1 subtypes:
- •Human immunodeficiency virus (HIV)-associated PAH
- •PAH associated with portal hypertension
- •Schistosomiasis-associated PAH
- •PAH with features of significant venous/capillary pulmonary veno-occlusive disease/pulmonary capillary hemangiomatosis (PVOD/PCH) involvement
- •Is on the waiting list for lung transplant
- •Pregnant or breastfeeding women
- •History of full or partial pneumonectomy
- •Pulmonary function test (PFT) values of forced vital capacity (FVC) < 60% predicted at the screening visit or within 6 months prior to the screening visit.
- •Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to the screening visit or planned initiation during the study.
- •History of more than mild obstructive sleep apnea that is untreated
- •Known history of portal hypertension or chronic liver disease, including hepatitis B and/or hepatitis C (with evidence of recent infection and/or active virus replication), defined as mild to severe hepatic impairment.
- •History of restrictive, constrictive, or congestive cardiomyopathy.
- •History of atrial septostomy within 180 days prior to the screening visit.
- •Personal or family history of long QT syndrome (LQTS) or sudden cardiac death.
- •Left ventricular ejection fraction (LVEF) < 45% on historical Echocardiogram (ECHO) within 6 months prior to the screening visit.
- •Any symptomatic coronary disease events within 6 months prior to the screening visit.
- •Cerebrovascular accident within 3 months prior to the screening visit.
- •Significant (≥ 2+ regurgitation) mitral regurgitation or aortic regurgitation valvular disease, mitral stenosis and more than mild aortic valve stenosis.
- •Prior exposure to sotatercept or luspatercept or history of allergic or anaphylactic reaction or hypersensitivity to recombinant proteins or excipients in investigational product
- •Received intravenous inotropes (e.g., dobutamine, dopamine, norepinephrine, vasopressin) within 30 days prior to the screening visit
- •Currently enrolled in or have completed any other investigational product study within 30 days
- •Weight at the screening is over 85 kg
研究组 & 干预措施
Sotatercept
Participants on background PAH therapy will receive sotatercept subcutaneous (SC) injections at a starting dose of 0.3 mg/kg with a target dose of 0.7 mg/kg every 3 weeks up to 24 weeks. Thereafter, participants may choose to receive the sotatercept treatment at same dose and schedule in the extension treatment period from Week 24 until up to 6 months after sotatercept becomes locally commercially available and reimbursed in Japan.
干预措施: Sotatercept (Biological)
结局指标
主要结局
Change From Pulmonary Vascular Resistance (PVR) From Baseline at Week 24
时间窗: Baseline and Week 24
PVR was the resistance against blood flow from the pulmonary artery to the left atrium. PVR was measured in dyn\*sec/cm\^5 by right heart catheterization (RHC). RHC was performed during the screening period (baseline) and Week 24. Per protocol, the change in PVR from baseline at Week 24 was reported for the primary treatment period.
Number of Participants Who Experienced an Adverse Event (AE)
时间窗: Up to ~24 weeks
An AE was any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Per protocol, the number of participants who experienced an AE were reported for the primary treatment period.
Number of Participants Who Discontinued Study Intervention Due to AEs
时间窗: Up to ~24 weeks
An AE was any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Per protocol, the number of participants who discontinued study treatment due to AEs were reported for the primary treatment period.
次要结局
- Change From Baseline in Six-Minute Walk Distance (6MWD) at Week 24(Baseline and Week 24)
- Percentage of Participants With Improvement in World Health Organization Functional Class (WHO FC) at Week 24(Baseline and Week 24)
- Change From Baseline in N-terminal proB-type Natriuretic Peptide (NT-proBNP) at Week 24(Baseline and Week 24)