Trial Outcomes & Findings for A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) (NCT NCT05033080)
NCT ID: NCT05033080
Last Updated: 2024-10-03
Results Overview
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
435 participants
Primary outcome timeframe
From Baseline Through Week 24
Results posted on
2024-10-03
Participant Flow
This study was conducted in cystic fibrosis (CF) participants aged 12 years or older. It was pre-specified in the protocol to combine the data from this study with study VX20-121-103 (NCT05076149) for selected outcome measures.
A total of 435 participants were enrolled in this study, of which 37 were included in the run-in period but were not dosed in treatment period. Therefore, results are presented for only 398 participants dosed in the treatment period.
Participant milestones
| Measure |
ELX/TEZ/IVA
Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
202
|
196
|
|
Overall Study
Pooled Analysis Set
|
491
|
480
|
|
Overall Study
COMPLETED
|
191
|
184
|
|
Overall Study
NOT COMPLETED
|
11
|
12
|
Reasons for withdrawal
| Measure |
ELX/TEZ/IVA
Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Overall Study
Adverse Event
|
4
|
4
|
|
Overall Study
Withdrawal of consent (not due to AE)
|
5
|
5
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Other non-compliance
|
0
|
1
|
|
Overall Study
Physician Decision
|
0
|
1
|
|
Overall Study
Other
|
1
|
1
|
Baseline Characteristics
Here, "Number Analyzed" signifies participants who were evaluable for this study specific baseline measure. This analysis set (N=394) included participants who received the dose and who had a data for this efficacy analysis.
Baseline characteristics by cohort
| Measure |
ELX/TEZ/IVA
n=202 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
n=196 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
Total
n=398 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
30.9 years
STANDARD_DEVIATION 11.4 • n=202 Participants
|
30.8 years
STANDARD_DEVIATION 10.5 • n=196 Participants
|
30.8 years
STANDARD_DEVIATION 11.0 • n=398 Participants
|
|
Sex: Female, Male
Female
|
83 Participants
n=202 Participants
|
80 Participants
n=196 Participants
|
163 Participants
n=398 Participants
|
|
Sex: Female, Male
Male
|
119 Participants
n=202 Participants
|
116 Participants
n=196 Participants
|
235 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
11 Participants
n=202 Participants
|
13 Participants
n=196 Participants
|
24 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
190 Participants
n=202 Participants
|
183 Participants
n=196 Participants
|
373 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
Not Collected per Local Regulations
|
1 Participants
n=202 Participants
|
0 Participants
n=196 Participants
|
1 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
White
|
197 Participants
n=202 Participants
|
191 Participants
n=196 Participants
|
388 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
1 Participants
n=202 Participants
|
4 Participants
n=196 Participants
|
5 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=202 Participants
|
1 Participants
n=196 Participants
|
1 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Participants
n=202 Participants
|
0 Participants
n=196 Participants
|
1 Participants
n=398 Participants
|
|
Race/Ethnicity, Customized
More than one race
|
3 Participants
n=202 Participants
|
0 Participants
n=196 Participants
|
3 Participants
n=398 Participants
|
|
Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
67.2 Percentage points
STANDARD_DEVIATION 14.6 • n=201 Participants • Here, "Number Analyzed" signifies participants who were evaluable for this study specific baseline measure. This analysis set (N=394) included participants who received the dose and who had a data for this efficacy analysis.
|
67.0 Percentage points
STANDARD_DEVIATION 15.3 • n=193 Participants • Here, "Number Analyzed" signifies participants who were evaluable for this study specific baseline measure. This analysis set (N=394) included participants who received the dose and who had a data for this efficacy analysis.
|
67.1 Percentage points
STANDARD_DEVIATION 15.0 • n=394 Participants • Here, "Number Analyzed" signifies participants who were evaluable for this study specific baseline measure. This analysis set (N=394) included participants who received the dose and who had a data for this efficacy analysis.
|
PRIMARY outcome
Timeframe: From Baseline Through Week 24Population: The Full Analysis Set (FAS) included all randomized participants who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during Treatment Period. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome measure.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
ELX/TEZ/IVA
n=193 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
n=187 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
|
0.3 percentage points
Interval -0.3 to 0.9
|
0.5 percentage points
Interval -0.1 to 1.1
|
SECONDARY outcome
Timeframe: From Baseline Through Week 24Population: FAS. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome measure.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
ELX/TEZ/IVA
n=194 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
n=185 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Absolute Change in Sweat Chloride (SwCl)
|
0.9 millimole per liter (mmol/L)
Interval -0.6 to 2.3
|
-7.5 millimole per liter (mmol/L)
Interval -9.0 to -6.0
|
SECONDARY outcome
Timeframe: From Baseline Through Week 24Population: The Pooled Full Analysis Set (PFAS) included all randomized participants from this study (VX20-121-102) and from Study VX20-121-103 who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during the Treatment Period. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
ELX/TEZ/IVA
n=479 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
n=465 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103)
|
76.6 percentage of participants
|
85.8 percentage of participants
|
SECONDARY outcome
Timeframe: From Baseline Through Week 24Population: PFAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure.
Sweat samples were collected using an approved collection device.
Outcome measures
| Measure |
ELX/TEZ/IVA
n=479 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VX-121/TEZ/D-IVA
n=465 Participants
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103)
|
22.5 percentage of participants
|
30.5 percentage of participants
|
Adverse Events
ELX/TEZ/IVA
Serious events: 41 serious events
Other events: 184 other events
Deaths: 0 deaths
VNZ/TEZ/D-IVA
Serious events: 28 serious events
Other events: 177 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ELX/TEZ/IVA
n=202 participants at risk
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VNZ/TEZ/D-IVA
n=196 participants at risk
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Cardiac disorders
Arteriospasm coronary
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Congenital, familial and genetic disorders
Cerebrovascular arteriovenous malformation
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Constipation
|
0.99%
2/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Dysphagia
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Mechanical ileus
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Pancreatitis
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Subileus
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Hepatobiliary disorders
Cholestasis
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Appendicitis
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
COVID-19
|
1.5%
3/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Cellulitis
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Diverticulitis
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
11.4%
23/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
5.6%
11/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Influenza
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
1.5%
3/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Meningitis aseptic
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
1.0%
2/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Viral myocarditis
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Injury, poisoning and procedural complications
Alcohol poisoning
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Injury, poisoning and procedural complications
Postoperative ileus
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Alanine aminotransferase increased
|
0.99%
2/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Aspartate aminotransferase increased
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Blood alkaline phosphatase increased
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Pulmonary function test decreased
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Metabolism and nutrition disorders
Hyperphosphatasaemia
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Nervous system disorders
Epilepsy
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Nervous system disorders
Headache
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Nervous system disorders
Serotonin syndrome
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Nervous system disorders
Syncope
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
1.0%
2/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Psychiatric disorders
Behaviour disorder
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Psychiatric disorders
Depression
|
0.00%
0/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.51%
1/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Psychiatric disorders
Depression suicidal
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Psychiatric disorders
Mixed anxiety and depressive disorder
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Psychiatric disorders
Suicidal ideation
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
1.0%
2/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.99%
2/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
1.0%
2/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal polyps
|
0.50%
1/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
0.00%
0/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
Other adverse events
| Measure |
ELX/TEZ/IVA
n=202 participants at risk
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.
|
VNZ/TEZ/D-IVA
n=196 participants at risk
Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
5.0%
10/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
2.6%
5/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Abdominal pain
|
6.9%
14/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
5.1%
10/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
3.0%
6/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
6.1%
12/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Diarrhoea
|
7.4%
15/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
10.7%
21/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Gastrointestinal disorders
Nausea
|
8.4%
17/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
3.6%
7/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
General disorders
Fatigue
|
7.9%
16/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
9.2%
18/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
General disorders
Pyrexia
|
10.4%
21/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
12.2%
24/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
COVID-19
|
26.2%
53/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
25.0%
49/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
31.7%
64/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
27.0%
53/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Influenza
|
5.0%
10/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
8.7%
17/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Nasopharyngitis
|
17.3%
35/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
23.0%
45/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Respiratory tract infection
|
5.9%
12/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
4.1%
8/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Sinusitis
|
5.0%
10/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
5.1%
10/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Upper respiratory tract infection
|
13.4%
27/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
8.7%
17/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
8.9%
18/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
8.7%
17/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Alanine aminotransferase increased
|
5.0%
10/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
6.6%
13/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Aspartate aminotransferase increased
|
4.0%
8/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
6.1%
12/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Investigations
Blood creatine phosphokinase increased
|
11.4%
23/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
9.2%
18/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.4%
11/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
5.1%
10/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.9%
16/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
4.6%
9/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Nervous system disorders
Headache
|
10.9%
22/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
12.8%
25/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.3%
41/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
23.0%
45/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
5.0%
10/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
6.6%
13/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
11.9%
24/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
9.7%
19/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
11.4%
23/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
12.2%
24/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
2.5%
5/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
6.1%
12/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.9%
14/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
9.7%
19/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
2.5%
5/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
7.1%
14/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
10.4%
21/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
9.2%
18/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.5%
9/202 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
6.1%
12/196 • Day 1 up to Safety follow-up (up to 56 weeks)
Safety set include all participants who received at least 1 dose of study drug during the Treatment Period.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place