Trial Outcomes & Findings for A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF) (NCT NCT05111145)

NCT ID: NCT05111145

Last Updated: 2023-07-11

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 86 participants
• Primary outcome timeframe: Day 1 up to Week 36
• Results posted on: 2023-07-11

Participant Flow

Participants from Parent Studies VX19-445-117 (NCT04599465) and VX20-445-126 (NCT04969224) were enrolled in this study. A total of 86 participants were enrolled in this study.

Participant milestones

Measure	ELX/TEZ/IVA Participants received elexacaftor (ELX) 200 mg once daily (qd)/tezacaftor (TEZ) 100 mg qd/ivacaftor (IVA) 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.
Overall Study STARTED	86
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	86

Reasons for withdrawal

Measure	ELX/TEZ/IVA Participants received elexacaftor (ELX) 200 mg once daily (qd)/tezacaftor (TEZ) 100 mg qd/ivacaftor (IVA) 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.
Overall Study Commercial drug is available for participant	76
Overall Study Participant enrolled in another qualified Vertex study	10

Baseline Characteristics

A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)

Baseline characteristics by cohort

Measure	ELX/TEZ/IVA n=86 Participants Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks.
Age, Continuous	25.3 years STANDARD_DEVIATION 9.9 • n=5 Participants
Sex: Female, Male Female	37 Participants n=5 Participants
Sex: Female, Male Male	49 Participants n=5 Participants
Race/Ethnicity, Customized White	79 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or other Pacific Islander	0 Participants n=5 Participants
Race/Ethnicity, Customized Other	0 Participants n=5 Participants
Race/Ethnicity, Customized Not collected per local regulations	7 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	11 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	68 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 up to Week 36

Population: Safety set included for all participants who received at least 1 dose of study drug.

Outcome measures

Measure	ELX/TEZ/IVA n=86 Participants Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) Participants with SAEs	4 Participants
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) Participants with TEAEs	61 Participants

Adverse Events

ELX/TEZ/IVA

Serious events: 4 serious events

Other events: 33 other events

Deaths: 0 deaths

Serious adverse events

Measure	ELX/TEZ/IVA n=86 participants at risk Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks.
Infections and infestations Gastroenteritis viral	1.2% 1/86 • Day 1 up to Week 36
Investigations Alanine aminotransferase increased	1.2% 1/86 • Day 1 up to Week 36
Investigations Aspartate aminotransferase increased	1.2% 1/86 • Day 1 up to Week 36
Metabolism and nutrition disorders Malnutrition	1.2% 1/86 • Day 1 up to Week 36
Psychiatric disorders Anxiety	1.2% 1/86 • Day 1 up to Week 36

Other adverse events

Measure	ELX/TEZ/IVA n=86 participants at risk Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks.
Infections and infestations COVID-19	18.6% 16/86 • Day 1 up to Week 36
Infections and infestations Infective pulmonary exacerbation of cystic fibrosis	12.8% 11/86 • Day 1 up to Week 36
Infections and infestations Nasopharyngitis	10.5% 9/86 • Day 1 up to Week 36
Infections and infestations Upper respiratory tract infection	7.0% 6/86 • Day 1 up to Week 36

Additional Information

Medical Monitor

Vertex Pharmaceuticals Incorporated

Phone: 617-341-6777

Email: medicalinfo@vrtx.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place