Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Официальное название

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older

Обзор

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

Вмешательства

• Препарат VNZ/TEZ/D-IVA
  Fixed-dose combination tablets or granules for oral administration.

Первичные конечные точки

• Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) [Срок оценки: From Baseline up to Week 100]
• Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) [Срок оценки: From Baseline up to Week 196]

Вторичные конечные точки (12)

• Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl) [Срок оценки: From Baseline Through Week 96]
• Part B: Absolute Change in Sweat Chloride (SwCl) [Срок оценки: From Baseline Through Week 192]
• Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) [Срок оценки: From Baseline Through Week 100]
• Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) [Срок оценки: From Baseline Through Week 196]
• Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx) [Срок оценки: From Baseline Through Week 100]
• Part B: Number of Pulmonary Exacerbation (PEx) [Срок оценки: From Baseline Through Week 196]
• Part A (All Cohorts): Number of CF- Related Hospitalizations [Срок оценки: From Baseline Through Week 100]
• Part B: Number of CF- Related Hospitalizations [Срок оценки: From Baseline Through Week 196]
• Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [Срок оценки: From Baseline Through Week 100]
• Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [Срок оценки: From Baseline Through Week 196]
• Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI) [Срок оценки: From Baseline Through Week 100]
• Part B: Absolute Change in Body Mass Index (BMI) [Срок оценки: From Baseline Through Week 196]

Критерии участия

Критерии включения

Parts A and B:

• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Part B:

-Meets at least 1 of the following criteria:

• Completed study drug treatment in Part A
• Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A

Критерии исключения

• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
• History of solid organ, hematological transplantation, or cancer
• History of drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 18 центров

• Children's Hospital of Orange County — Orange
• Stanford University - Palo Alto - Pulmonology — Palo Alto
• Children's Hospital of Colorado - Pulmonology — Aurora
• The Emory Clinic - Clifton Road - Pulmonology — Atlanta
• Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology — Chicago
• JW Riley Hospital for Children - Pulmonology — Indianapolis
• Boston Children's Hospital — Boston
• Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota — Minneapolis
• … и ещё 10 центров

Австралия · 4 центра

• The Kids Research Institute Australia — Nedlands
• Women's & Children's Hospital — North Adelaide
• The Royal Children's Hospital Melbourne — Parkville
• Children's Health Queensland Hospital and Health Service — South Brisbane

Германия · 3 центра

• Charite Paediatric Pulmonology Department — Berlin
• Universitatsklinikum Essen — Essen
• Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonat — Hanover

Канада · 2 центра

• Hospital for Sick Children - Pulmonology — Toronto
• British Columbia Children's Hospital — Vancouver

Франция · 2 центра

• Hopital Femme Mere-Enfant — Bron
• Hopital Necker Enfants Malades - Pulmonology — Paris

Швеция · 2 центра

• Sahlgrenska Universitetssjukhuset - Göteborg CF-center — Gothenburg
• Karolinska University Hospital - Pulmonology — Stockholm

Швейцария · 2 центра

• Inselspital Bern — Bern
• Kinderspital Zurich - Abteilung Pneumologie — Zurich

Великобритания · 2 центра

• Noah's Ark Children's Hospital for Wales — Cardiff
• Great Ormond Street Hospital for Children — London

Нидерланды · 1 центр

• Sophia Children's Hospital — Rotterdam

Новая Зеландия · 1 центр

• Starship Child Health — Grafton

Идентификаторы

NCT: NCT05844449 · VX22-121-106 · 2022-503081-74-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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