Trial Outcomes & Findings for A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation (NCT NCT02730208)
NCT ID: NCT02730208
Last Updated: 2019-10-23
Results Overview
The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
41 participants
Primary outcome timeframe
From Baseline at Week 72
Results posted on
2019-10-23
Participant Flow
A total of 41 participants were randomized and treated in the study.
Participant milestones
| Measure |
TEZ/IVA
Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
21
|
|
Overall Study
COMPLETED
|
20
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
TEZ/IVA
Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
|---|---|---|
|
Overall Study
Withdrawal of consent (not due to AE)
|
0
|
1
|
Baseline Characteristics
A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Baseline characteristics by cohort
| Measure |
TEZ/IVA
n=20 Participants
Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
n=21 Participants
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
Total
n=41 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
20.4 years
STANDARD_DEVIATION 7.5 • n=5 Participants
|
20.1 years
STANDARD_DEVIATION 9.3 • n=7 Participants
|
20.2 years
STANDARD_DEVIATION 8.4 • n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
20 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Total Brody/Cystic Fibrosis - Computed Tomography (CF-CT) Score
|
38.29 scores on a scale
STANDARD_DEVIATION 22.91 • n=5 Participants
|
43.68 scores on a scale
STANDARD_DEVIATION 33.96 • n=7 Participants
|
40.98 scores on a scale
STANDARD_DEVIATION 28.72 • n=5 Participants
|
PRIMARY outcome
Timeframe: From Baseline at Week 72Population: FAS included all participants who were randomized and received at least 1 dose of study drug.
The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
Outcome measures
| Measure |
TEZ/IVA
n=20 Participants
Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
n=21 Participants
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
|---|---|---|
|
Absolute Change in Total Brody/CF-CT Score
|
0.90 scores on a scale
Standard Error 2.09
|
2.38 scores on a scale
Standard Error 2.07
|
SECONDARY outcome
Timeframe: Day 1 up to Week 76Population: Safety set included all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
TEZ/IVA
n=20 Participants
Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
n=21 Participants
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with AEs
|
20 Participants
|
21 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
8 Participants
|
13 Participants
|
Adverse Events
TEZ/IVA
Serious events: 8 serious events
Other events: 19 other events
Deaths: 0 deaths
Placebo
Serious events: 13 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
TEZ/IVA
n=20 participants at risk
Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
n=21 participants at risk
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
25.0%
5/20 • Day 1 up to Week 76
|
28.6%
6/21 • Day 1 up to Week 76
|
|
Infections and infestations
Lung infection pseudomonal
|
10.0%
2/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Infections and infestations
Atypical mycobacterial lower respiratory tract infection
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Cardiac disorders
Atrioventricular block first degree
|
5.0%
1/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.0%
1/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Reproductive system and breast disorders
Testicular torsion
|
5.0%
1/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Hepatobiliary disorders
Hepatic cirrhosis
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Immune system disorders
Type I hypersensitivity
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Investigations
Bacterial test positive
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Investigations
Weight decreased
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
Other adverse events
| Measure |
TEZ/IVA
n=20 participants at risk
Participants received TEZ 100 mg/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
|
Placebo
n=21 participants at risk
Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
|
|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
25.0%
5/20 • Day 1 up to Week 76
|
42.9%
9/21 • Day 1 up to Week 76
|
|
Infections and infestations
Upper respiratory tract infection
|
20.0%
4/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
15.0%
3/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Infections and infestations
Nasopharyngitis
|
15.0%
3/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Infections and infestations
Gastroenteritis
|
10.0%
2/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Infections and infestations
Influenza
|
10.0%
2/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Infections and infestations
Pharyngitis
|
10.0%
2/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Infections and infestations
Rhinitis
|
0.00%
0/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
40.0%
8/20 • Day 1 up to Week 76
|
42.9%
9/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
20.0%
4/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
20.0%
4/20 • Day 1 up to Week 76
|
19.0%
4/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
10.0%
2/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
10.0%
2/20 • Day 1 up to Week 76
|
4.8%
1/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
5.0%
1/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
5.0%
1/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
General disorders
Pyrexia
|
15.0%
3/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
General disorders
Fatigue
|
5.0%
1/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
General disorders
Chest pain
|
0.00%
0/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Nervous system disorders
Migraine
|
10.0%
2/20 • Day 1 up to Week 76
|
0.00%
0/21 • Day 1 up to Week 76
|
|
Nervous system disorders
Headache
|
5.0%
1/20 • Day 1 up to Week 76
|
19.0%
4/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Abdominal pain
|
5.0%
1/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/20 • Day 1 up to Week 76
|
19.0%
4/21 • Day 1 up to Week 76
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/20 • Day 1 up to Week 76
|
14.3%
3/21 • Day 1 up to Week 76
|
|
Injury, poisoning and procedural complications
Sunburn
|
5.0%
1/20 • Day 1 up to Week 76
|
14.3%
3/21 • Day 1 up to Week 76
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Investigations
Fungal test positive
|
5.0%
1/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
|
Investigations
Bacterial test positive
|
0.00%
0/20 • Day 1 up to Week 76
|
23.8%
5/21 • Day 1 up to Week 76
|
|
Investigations
Blood alkaline phosphatase increased
|
0.00%
0/20 • Day 1 up to Week 76
|
9.5%
2/21 • Day 1 up to Week 76
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER