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Набира участници Фаза 1/2 NCT05668741

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

Фаза 1/2 – комбинирано ранно изпитване
Заболявания: Cystic Fibrosis

Спонсор: Vertex Pharmaceuticals Incorporated

Налично на: БГ
Обобщение
The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
Кой може да участва
Key Inclusion Criteria: * Body mass index is less than (\<) 30.0 kilograms per meter square (kg/m\^2) * A total body weight greater than (\>) 50 kg * Stable CF disease * CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy o Example mutations include but are not limited to, mutations that do not produce CFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice mutations (e.g., 621+1G-\>T) * Forced expiratory volume in 1 second (FEV1) value for SAD: greater than or equal to (≥)40 percent (%), MAD: ≥ 50% to less than or equal to (≤) 90% Key Exclusion Criteria: * History of uncontrolled asthma within a year prior to screening * History of solid organ or hematological transplantation * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) * Arterial oxygen saturation on room air less than (\<) 94% at screening Other protocol defined Inclusion/Exclusion criteria may apply.
Места на провеждане 10
Австралия (1)
The Alfred Hospital - Pulmonology
Melbourne
Белгия (1)
Universitair Ziekenhuis Gent
Ghent
Канада (1)
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary
Германия (1)
Ruhrlandklinik
Essen
Италия (1)
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico - Pulmonology
Genova
Нидерландия (1)
UMCU - Department of Pulmonology and Tuberculosis
Utrecht
WITHDRAWN
Испания (1)
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona
Швеция (1)
Karolinska University Hospital - Pulmonology
Stockholm
Великобритания (1)
Papworth Hospital NHS Foundation Trust
Cambridge
COMPLETED
САЩ (1)
University of Alabama at Birmingham - Child Health Research Unit
Birmingham , Alabama
Технически детайли
Статус
Набира участници
Фаза
Фаза 1/2
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Минимална възраст
18 Years
Максимална възраст
65 Years
Здрави доброволци
Не
Начална дата
27.02.2023
Крайна дата
12.05.2027
Регистрационен номер
NCT05668741
Източник
anzctr
Запитване за медицински туризъм

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