Clinical trial NCT04969224: A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
Official title: A Phase 3b Open-label Study Evaluating the Effects of Elexacaftor/Tezacaftor/Ivacaftor on Cough and Physical Activity in Cystic Fibrosis Subjects 12 Years of Age and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2021-07-20
Enrollment: 82
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.


Eligibility criteria: Key Inclusion Criteria:

* Forced expiratory volume in 1 second (FEV1) value ≥30% and ≤90% predicted
* Heterozygous for CF transmembrane conductance regulator gene (CFTR) F508del mutation and a minimal function mutation (F/MF genotypes)

Key Exclusion Criteria:

* Clinically significant liver cirrhosis
* Solid organ or hematological transplantation
* Non-ambulatory status
* Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12
timeFrame: Baseline, Week 8 through Week 12
Secondary outcomes:
measure: Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12
timeFrame: Baseline, Week 8 through Week 12
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Chermside
country: Australia
facility: The Prince Charles Hospital
city: Melbourne, VIC
country: Australia
facility: Alfred Hospital
city: Nedlands
country: Australia
facility: Institute for Respiratory Health
city: Nedlands
country: Australia
facility: Telethon Kids Institute
city: Parkville, VIC
country: Australia
facility: The Royal Children's Hospital
city: South Brisbane
country: Australia
facility: Mater Adult Hospital
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Brussels
country: Belgium
facility: Cliniques Universitaires de Bruxelles Hopital Erasme
city: Brussels
country: Belgium
facility: Universitair Ziekenhuis Brussel - Campus Jette
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
city: Calgary
country: Canada
facility: University of Calgary Medical Clinic of the Foothills Medical Centre
city: Edmonton
country: Canada
facility: University of Alberta Hospital, Edmonton Clinic
city: Halifax
country: Canada
facility: Queen Elizabeth II Health Sciences Center
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Sabadell
country: Spain
facility: Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
Study officials:
References: