Trial Outcomes & Findings for A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF) (NCT NCT04969224)

Last Updated: 2025-09-24

Results Overview

Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

82 participants

Primary outcome timeframe

Baseline, Week 8 through Week 12

Results posted on

2025-09-24

Participant Flow

This study was conducted in participants with cystic fibrosis, aged 12 years and older, who are heterozygous for the F508del mutation and the minimal function mutation (F/MF) genotypes.

Participant milestones

Participant milestones
Measure	ELX/TEZ/IVA Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/ tezacaftor (TEZ) 100 mg qd/ ivacator (IVA) 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks.
Overall Study STARTED	82
Overall Study COMPLETED	80
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	ELX/TEZ/IVA Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/ tezacaftor (TEZ) 100 mg qd/ ivacator (IVA) 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks.
Overall Study Adverse Event	1
Overall Study Enrolled but never dosed	1

Baseline Characteristics

A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ELX/TEZ/IVA n=81 Participants Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period approximately 13 weeks.
Age, Continuous	25.7 years STANDARD_DEVIATION 9.6 • n=5 Participants
Sex: Female, Male Female	37 Participants n=5 Participants
Sex: Female, Male Male	44 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	78 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	81 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 8 through Week 12

Population: The Full Analysis Set (FAS) will include all enrolled participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome.

Outcome measures

Outcome measures
Measure	ELX/TEZ/IVA n=80 Participants Participants received ELX 200 mg qd/ TEZ 100 mg qd/ IVA150 mg q12h in the treatment period approximately 13 weeks.
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12	91.7 percent reduction Interval 89.2 to 93.6

SECONDARY outcome

Timeframe: Baseline, Week 8 through Week 12

Population: FAS. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome.

Outcome measures

Outcome measures
Measure	ELX/TEZ/IVA n=80 Participants Participants received ELX 200 mg qd/ TEZ 100 mg qd/ IVA150 mg q12h in the treatment period approximately 13 weeks.
Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12	637.56 step count per day Interval 298.16 to 976.96

Adverse Events

ELX/TEZ/IVA

Serious events: 2 serious events

Other events: 56 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	ELX/TEZ/IVA n=81 participants at risk Participants received ELX 200 mg qd/ TEZ 100 mg qd/ IVA150 mg q12h in the treatment period approximately 13 weeks.
Gastrointestinal disorders Pancreatitis	1.2% 1/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Psychiatric disorders Anxiety	1.2% 1/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.

Other adverse events

Other adverse events
Measure	ELX/TEZ/IVA n=81 participants at risk Participants received ELX 200 mg qd/ TEZ 100 mg qd/ IVA150 mg q12h in the treatment period approximately 13 weeks.
Gastrointestinal disorders Abdominal pain	9.9% 8/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Gastrointestinal disorders Abdominal pain upper	8.6% 7/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Gastrointestinal disorders Diarrhoea	13.6% 11/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Infections and infestations COVID-19	16.0% 13/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Infections and infestations Nasopharyngitis	16.0% 13/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Infections and infestations Upper respiratory tract infection	8.6% 7/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Nervous system disorders Headache	17.3% 14/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Cough	7.4% 6/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Nasal congestion	6.2% 5/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	9.9% 8/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Rash	6.2% 5/81 • Day 1 up to Week 17 Safety Set included all participants who had received at least 1 dose of study drug.

Additional Information

Medical Monitor

Vertex Pharmaceuticals Incorporated

Phone: 617-341-6777

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place