Clinical trial NCT03559062: A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
Official title: A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2018-06-15
Enrollment: 67
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).


Eligibility criteria: Key Inclusion Criteria:

* Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
* Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
* Participants with a screening LCI2.5 result ≥7.5.
* Participants who are able to swallow tablets.

Key Exclusion Criteria:

* Clinically significant cirrhosis with or without portal hypertension.
* Colonization with organisms associated with a more rapid decline in pulmonary status.
* Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
timeFrame: From baseline through Week 8
Secondary outcomes:
measure: Absolute Change in Sweat Chloride At Week 8
timeFrame: From baseline at Week 8
measure: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
timeFrame: From baseline through Week 8
measure: Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
timeFrame: From first dose of study drug up to safety follow-up visit (up to Week 12)
Other outcomes:
Locations:
city: New Lambton Heights
country: Australia
facility: Hunter Medical Research Institute (HMRI)
city: Perth
country: Australia
facility: Princess Margaret Hospital for Children
city: South Brisbane
country: Australia
facility: Lady Cilento Children's Hospital
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
city: Brussels
country: Belgium
facility: Universitair Ziekenhuis Brussel - Campus Jette
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
city: Copenhagen
country: Denmark
facility: University of Copenhagen Rigshospitalet
city: Bordeaux Cedex
country: France
facility: Groupe Hospitalier Pellegrin - Hôpital des Enfants
city: Paris
country: France
facility: Hôpital Necker - Enfants Malades
city: Essen
country: Germany
facility: Universitaetsklinikum Essen
city: Frankfurt
country: Germany
facility: Klinikum der Johann Wolfgang Goethe-Universitaet
city: Giessen
country: Germany
facility: Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Heidelberg
country: Germany
facility: Universitaetsklinikum Heidelberg
city: Jena
country: Germany
facility: Universitaetsklinikum Jena
city: Koeln
country: Germany
facility: Universitaetsklinikum Koeln
city: Tuebingen
country: Germany
facility: Universitaetsklinikum Tuebingen
city: Dublin
country: Ireland
facility: Our Lady's Children's Hospital
city: Limerick
country: Ireland
facility: University Hospital Limerick
city: Dziekanow Lesny
country: Poland
facility: Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
city: Bern
country: Switzerland
facility: Inselspital - Universitaetsspital Bern
city: Zuerich
country: Switzerland
facility: Kinderspital Zuerich
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Sick Children
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
city: London
country: United Kingdom
facility: Royal Brompton Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospital City Campus
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
References:
citation: Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.