2023-504861-22-00招募中2 期
A Phase 2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sotatercept (MK-7962) in Children from 1 to Less Than 18 Years of Age With PAH on Standard of Care
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 22
- 试验地点
- 13
- 主要终点
- Serum Trough Concentration (Ctrough) of Sotatercept
研究概览
简要总结
- To evaluate the safety and tolerability of sotatercept over 24 weeks of treatment
- To evaluate the PK of sotatercept over 24 weeks of treatment
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Documented, historic diagnostic right heart catheterization (RHC) any time before Screening confirming the diagnosis of PAH WHO Group 1 in any of the following subtypes: • Idiopathic pulmonary arterial hypertension (IPAH) • Heritable PAH • Drug/toxin-induced PAH • PAH associated with connective tissue disease • PAH-congenital heart disease (CHD) with shunt closure >6 months before Screening and subsequently confirmed by RHC before Screening • PAH with coincidental shunt.
- •Must be on a stable dose(s) of background PAH therapy (phosphdiesterase-5 (PDE5) inhibitors, endothelin receptor antagonists (ERAs), soluble guanylate cyclase stimulators (sGCS), or prostanoids [including subcutaneous and intravenous])
- •If male, agree to the following during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention: • Abstains from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agrees to remain abstinent or • Uses contraception unless confirmed to be azoospermic (vasectomized or secondary to medical cause, documented from the site personnel's review of the participant's medical records, medical examination, or medical history interview) as detailed below: o Uses a male condom plus partner use of an additional contraceptive method when having penile-vaginal intercourse with a woman of childbearing potential (WOCBP) who is not currently pregnant Note: Men with a pregnant or breastfeeding partner must agree to remain abstinent from penile-vaginal intercourse or use a male condom during each episode of penile-vaginal penetration.
- •If female, must be either not a WOCBP or use a contraceptive method that is highly effective or be abstinent from heterosexual intercourse during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention
- •If male, agrees to refrain from donating blood or sperm for the duration of the study and for 16 weeks (112 days) after the last dose of study intervention
- •If female, agrees to refrain from donating blood, eggs, or ovum for the duration of the study and for at least 16 weeks (112 days) after the last dose of study intervention
排除标准
- •History of left-sided heart disease, including valvular disease (eg, moderate or greater mitral or aortic regurgitation or stenosis), left ventricular outflow tract obstruction, and/or left heart failure (eg, restrictive or dilated cardiomyopathy)
- •History of known pericardial constriction
- •Family history of sudden cardiac death or long QT syndrome
- •Any current or prior history of symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain) within 6 months before Screening
- •Cerebrovascular accident within 3 months before Screening
- •Prior exposure to sotatercept or luspatercept or has had an allergic reaction to any of their excipients
- •Severe (as based on the opinion of the investigator) congenital or developmental abnormalities of the lung, thorax, and/or diaphragm
- •History of Eisenmenger syndrome, Potts shunt, or atrial septostomy
- •Unrepaired or residual cardiac shunt
- •Diagnosis of pulmonary veno-occlusive diseases, pulmonary capillary hemangiomatosis, or overt signs of capillary and/or venous involvement
- •PAH associated with portal hypertension
- •Known visceral (lung, liver, or brain) arteriovenous malformation(s)
- •History of full or partial pneumonectomy
- •Untreated more than mild obstructive sleep apnea
结局指标
主要结局
Serum Trough Concentration (Ctrough) of Sotatercept
Serum Trough Concentration (Ctrough) of Sotatercept
Percentage of Participants Who Discontinue Study Drug Due to an AE
Percentage of Participants Who Discontinue Study Drug Due to an AE
Area Under the Curve at Steady State (AUCss) of Sotatercept
Area Under the Curve at Steady State (AUCss) of Sotatercept
Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept
Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept
Percentage of Participants Who Experience at Least 1 Adverse Event (AE)
Percentage of Participants Who Experience at Least 1 Adverse Event (AE)
Laboratory Parameter (Hematology): Concentration of Hemoglobin
Laboratory Parameter (Hematology): Concentration of Hemoglobin
Laboratory Parameter (Hematology): Hematocrit
Laboratory Parameter (Hematology): Hematocrit
Laboratory Parameter (Hematology): RBC Count
Laboratory Parameter (Hematology): RBC Count
Laboratory Parameter (Hematology): Reticulocyte Count
Laboratory Parameter (Hematology): Reticulocyte Count
Laboratory Parameter (Hematology): Platelet Count
Laboratory Parameter (Hematology): Platelet Count
Blood Pressure (BP)
Blood Pressure (BP)
Titer of Anti-drug Antibody (ADA) to Sotatercept
Titer of Anti-drug Antibody (ADA) to Sotatercept
次要结局
- Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2)
- Mean Change from Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)
- Mean Change from Baseline in Pulmonary Artery Systolic Pressure (PASP)
- Mean Change from Baseline in Right Ventricular Fractional Area Change (RVFAC)
- Mean Change from Baseline in Eccentricity Index
- Mean Change from Baseline in Right Ventricular (RV) Function (Cohorts 1 and 2)
- Mean Change from Baseline on Cardiac Output (Cohorts 1 and 2)
- Mean Change from Baseline in Pulmonary Arterial Pressure (PAP) (Cohorts 1 and 2)
- Mean Change from Baseline in Pediatric Quality of Life (PedsQL) Generic Score
- Mean Change from Baseline in N-terminal Prohormone B-type Natriuretic Peptide (NT-proBNP)
- Percentage of Participants who Either Improved or Maintained Their World Health Organization Functional Class (WHO FC)
研究者
Paul Reyfman
Scientific
Merck Sharp & Dohme LLC
研究点 (13)
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