A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option

Официальное название

A Prospective, Open-label, Platform Study for Long-term Follow-up of Participants Using Study Intervention in Pulmonary Hypertension Parent Studies

Обзор

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.

Вмешательства

• Препарат Macitentan
  Adult participants will receive oral dose of macitentan 10 milligrams (mg) tablet once daily. Children greater than or equal to (\>=) 2 year to less than (\<) 18 years will be given an oral macitentan dose tailored to their body weight, ensuring an equivalent level of systemic exposure as in adults.
• Препарат Selexipag
  Adult Participant will receive oral dose of selexipag tablet twice daily at the dose strength corresponding to their maintenance dose at the end of their parent study. Available strengths: 200, 400, 600, 800, 1000, 1200, 1400 and 1600 micrograms (µg). Children with body weight category of \>=50 kg will use the tablets at the required dose strength as described for adults. Children with a body weight \< 50 kg will receive tablets for pediatric use (dose strengths: 100 and 150 mcg), twice daily to
• Препарат Macitentan/Tadalafil FDC
  Participants will receive oral FDC of macitentan 10 mg and tadalafil 40 mg once daily during the course of the study as already received in the parent studies.

Первичные конечные точки

• Frequency of Treatment Emergent Adverse Events (TEAEs) [Срок оценки: Baseline until End of Study (EOS) (up to 84 months)]
• Frequency of TEAEs Leading to Discontinuation [Срок оценки: Baseline until EOS (up to 84 months)]
• Frequency of Serious Adverse Events (SAEs) [Срок оценки: Baseline until EOS (up to 84 months)]
• Frequency of Deaths [Срок оценки: Baseline until EOS (up to 84 months)]

Критерии участия

Критерии включения

• 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

Критерии исключения

General:

• 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 \[mL/min\])

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Польша · 8 центров

• Klinika Kardiologii Z Oddzialem Intensywnego Nadzoru Kardiologicznego UM W Bialymstoku — Bialystok
• Szpital Uniwersytecki nr 2 im dr Jana Biziela w Bydgoszczy, Klinika Kardiologii — Bydgoszcz
• SPSK nr 7 SUM w Katowicach Gornoslaskie Centrum Medyczne im Prof Leszka Gieca — Katowice
• Oddzial Kardiologii Wojewodzki Szpital Specjalistyczny im W Bieganskiego — Lodz
• Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ — Lublin
• SPSK2 PUM Klinika Kardiologii — Szczecin
• Wojewodzki Szpital Specjalist Osrodek Badawczo Rozwojowy — Wroclaw
• Wojewodzki Szpital Specjalistyczny we Wroclawiu — Wroclaw

Китай · 7 центров

• Beijing Anzhen Hospital 1 — Пекин
• Beijing Anzhen Hospital — Пекин
• The Second Xiangya Hospital of Central South Hospital — Чанша
• Jiangsu Province Hospital — Нанкин
• Qingdao Women and Children's Hospital — Qingdao
• Childrens Hospital of Shanghai — Шанхай
• The First Affiliated Hospital of Xian Jiaotong University — Сиань

South Korea · 7 центров

• Chungnam National University Hospital — Daejeon
• Gachon University Gil Medical Center — Incheon
• Seoul National University Hospital 1 — Seoul
• Seoul National University Hospital — Seoul
• Asan Medical Center — Seoul
• Samsung Medical Center — Seoul
• The Catholic University of Korea Seoul St Marys Hospital — Seoul

Россия · 5 центров

• Scientific and Research Institution of Cardiovascular Diseases Complex Problems — Kemerovo
• E.Meshalkin National Medical Research Center of the Ministry of Health of the Russian Fede — Novosibirsk
• Federal State Budgetary Institution — Saint Petersburg
• Institute of Cardiology of Tomsk National Research Medical Center of Rus Academy of Scienc — Tomsk
• Regional Clinical Hospital No1 — Tyumen

Тайвань · 5 центров

• Kaohsiung Veterans General Hospital — Kaohsiung City
• National Cheng Kung University Hospital — Tainan
• National Taiwan University Hospital — Taipei
• Taipei Veterans General Hospital — Taipei
• Chang-Gung Memorial Hospital, LinKou Branch — Taoyuan

Вьетнам · 3 центра

• Hanoi Heart Hospital — Hanoi
• Hanoi Medical University Hospital — Hanoi
• Children's Hospital 1 — Ho Chi Minh City

Беларусь · 2 центра

• The Republican Scientific-Practical Center ''Cardiology'' — Minsk
• Minsk Regional Clinical Hospital Of The Red Banner Of Labor — Minsk

ЮАР · 2 центра

• Abdullah, IA — Durban
• Dr Kalla — Lenasia

Украина · 2 центра

• Municipal Inst. Of Dnipropetrovsk Region. Council — Dnipro
• State Institute Of Phthisiology And Pulmonology N.A. F.G. Yanovskiy Of Ams Ukraine — Kyiv

Бельгия · 1 центр

• UZ Leuven — Leuven

Болгария · 1 центр

• University Multiprofile Hospital for Active Treatment- UMHAT Sveta Anna AD — Sofia

Венгрия · 1 центр

• Gottsegen György Országos Kardiológiai Intézet — Budapest

Таиланд · 1 центр

• Maharaj Nakorn Chiang Mai hospital Faculty of Medicine — Chiang Mai

Идентификаторы

NCT: NCT05179876 · CR109121 · NOPRODPAPUH3001 · 2021-002297-11 · 2023-506791-27-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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