A Prospective, Open-label, Platform Study for Long-term Follow-up of Participants Using Study Intervention in Pulmonary Hypertension Parent Studies
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 280
- 试验地点
- 67
- 主要终点
- Frequency of Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of the study is to enable participants with pulmonary hypertension (PH) currently treated with study intervention(s) in a clinical study (parent studies [NCT03422328, NCT03904693,NCT04565990, NCT02932410, NCT03492177, and NCT04175600]), to continue to benefit from the intervention after closure of the parent study in case they have no alternative means of access to the study intervention. This study will allow assessment of the long-term safety of each study intervention.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must sign an informed consent form (ICF) (or their legally designated representative must sign) indicating that participant understands the purpose of, and procedures required for, the study and is willing to participate in the study
- •Participant treated with oral macitentan or selexipag or fixed dose combination (FDC) of macitentan 10 milligrams (mg) and tadalafil 40 mg at the end of a sponsor parent study and: a) the indication of the parent study is included in the intervention-specific appendices (ISA) (pulmonary arterial hypertension [PAH]; b) participant has completed the parent study; c) no alternative means of access to study intervention (or equivalent approved therapy) have been identified; d) participant may continue to benefit from treatment with the study intervention; e) Participant is at least 18 years old for macitentan/tadalafil FDC, and at least 2 years old for macitentan or selexipag
- •A female participant of childbearing potential must: a) have a negative urine or serum pregnancy test prior to first intake of study intervention; b) agree to perform monthly urine pregnancy test up to the end of the safety follow-up period; c) If heterosexually active, agree to follow contraceptive methods until 30 days after the last intake of the study intervention. For pediatric female participants: It is the responsibility of the investigator to ensure appropriate counselling, including consultation with a specialist (if needed), to the participant and/or parent(s)/ legally designated representative (LDR)(s) on the acceptable method of contraception
排除标准
- •Participants prematurely discontinued from the study intervention in their parent study
- •Female participant being pregnant, or breastfeeding, or planning to become pregnant while enrolled in this study
- •Planned or current treatment with another investigational treatment
- •Macitentan-specific:
- •Known allergies, hypersensitivity, or intolerance to macitentan or its excipients
- •Hemoglobin less than (<) 80 grams per liter (g/L)
- •Serum aspartate (AST) and/or alanine aminotransferases (ALT) greater than (>) 3* upper limit of normal (ULN)
- •Known and documented severe hepatic impairment that is, Child-Pugh Class C. For participants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should be fully assessed and documented in the source documents at screening
- •Selexipag-specific:
- •Known allergies, hypersensitivity, or intolerance to selexipag or its excipients
- •Suspected or known pulmonary veno-occlusive disease (PVOD)
- •Uncontrolled thyroid disease
- •Severe coronary heart disease or unstable angina, myocardial infarction within the last 6 months, decompensated cardiac failure (if not under close medical supervision), severe arrhythmia, cerebrovascular events (for example, transient ischemic attack, stroke) within the last 3 months, or congenital or acquired valvular defects with clinically relevant myocardial function disorders not related to pulmonary hypertension (PH)
- •Known and documented severe hepatic impairment that is, Child-Pugh Class C. For participants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should be fully assessed and documented in the source documents at screening
- •Children only: (a) Current suspicion of intussusception or ileus or gastrointestinal obstruction, per the investigator's judgment; (b) hemoglobin or hematocrit <75 percent (%) of the lower limit of normal range
- •Macitentan/tadalafil FDC-specific:
- •Known allergies, hypersensitivity, or intolerance to macitentan or tadalafil or their excipients
- •Hemoglobin <80 g/L
- •Serum aspartate (AST) and/or alanine aminotransferases (ALT) >3* ULN range
- •Known and documented severe hepatic impairment that is, Child-Pugh Class C. For participants with hepatic impairment, Child-Pugh Class should be fully assessed and documented in the source documents at screening
- •Severe renal impairment (estimated glomerular filtration rate [eGF]/creatinine clearance <30 milliliter per minute [mL/min])
研究组 & 干预措施
Selexipag
Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive study drug selexipag orally during the course of the study. Study visits are scheduled every 6 months to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.
干预措施: Selexipag (Drug)
Macitentan/Tadalafil FDC
Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive drug Macitentan and Tadalafil fixed dose combination (FDC) orally during the course of the study. Study visits are scheduled every 6 months to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.
干预措施: Macitentan/Tadalafil FDC (Drug)
Macitentan
Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive study drug macitentan orally during the course of the study. For adult participants study visits will be scheduled every 6 months and for pediatric participants study visits will be scheduled every 3 months. The study includes on-site visits to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.
干预措施: Macitentan (Drug)
结局指标
主要结局
Frequency of Treatment Emergent Adverse Events (TEAEs)
时间窗: Baseline until End of Study (EOS) (up to 84 months)
An adverse event (AE) is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. TEAEs are defined as a treatment-emergent AE is any AE temporally associated with the use of study treatment (from start of treatment in the PLATYPUS protocol until 30 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.
Frequency of TEAEs Leading to Discontinuation
时间窗: Baseline until EOS (up to 84 months)
Frequency of TEAEs leading to discontinuation of study intervention will be reported. An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. TEAEs are defined as a treatment-emergent AE is any AE temporally associated with the use of study treatment (from start of treatment in the PLATYPUS protocol until 30 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.
Frequency of Serious Adverse Events (SAEs)
时间窗: Baseline until EOS (up to 84 months)
SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product and is medically important.
Frequency of Deaths
时间窗: Baseline until EOS (up to 84 months)
Frequency of deaths will be reported.
次要结局
未报告次要终点