Trial Outcomes & Findings for Effect of Andecaliximab on FEV1 in Adults With Cystic Fibrosis (NCT NCT02759562)
NCT ID: NCT02759562
Last Updated: 2018-08-17
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Baseline; Week 8
Results posted on
2018-08-17
Participant Flow
Participants were enrolled at study sites in Australia and Europe. The first participant was screened on 04 November 2016. The last study visit occurred on 21 July 2017. The study was terminated after 6 participants were enrolled in Part 1 of the study. Therefore, Part 2 was not initiated.
26 participants were screened.
Participant milestones
| Measure |
Andecaliximab
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|
|
Double-Blind Treatment Period (8 Weeks)
STARTED
|
3
|
3
|
|
Double-Blind Treatment Period (8 Weeks)
COMPLETED
|
3
|
3
|
|
Double-Blind Treatment Period (8 Weeks)
NOT COMPLETED
|
0
|
0
|
|
Open-Label Treatment Period
STARTED
|
2
|
2
|
|
Open-Label Treatment Period
COMPLETED
|
0
|
0
|
|
Open-Label Treatment Period
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
Andecaliximab
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|
|
Open-Label Treatment Period
Withdrew Consent
|
1
|
0
|
|
Open-Label Treatment Period
Study Terminated by Sponsor
|
1
|
2
|
Baseline Characteristics
Effect of Andecaliximab on FEV1 in Adults With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Andecaliximab
n=3 Participants
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
n=3 Participants
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
46.0 years
STANDARD_DEVIATION 20.42 • n=5 Participants
|
43.3 years
STANDARD_DEVIATION 10.02 • n=7 Participants
|
44.7 years
STANDARD_DEVIATION 14.46 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
France
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Pre-bronchodilator Forced expiratory volume in 1 second (FEV1) % predicted
|
49.7 percent
STANDARD_DEVIATION 8.70 • n=5 Participants
|
59.1 percent
STANDARD_DEVIATION 19.26 • n=7 Participants
|
54.4 percent
STANDARD_DEVIATION 14.33 • n=5 Participants
|
|
Post-bronchodilator FEV1 % predicted
|
52.1 percent
STANDARD_DEVIATION 5.90 • n=5 Participants
|
64.6 percent
STANDARD_DEVIATION 20.58 • n=7 Participants
|
58.3 percent
STANDARD_DEVIATION 15.17 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline; Week 8Population: Full Analysis Set: all randomized participants who took at least 1 dose of study drug.
Outcome measures
| Measure |
Andecaliximab
n=3 Participants
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
n=3 Participants
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|
|
Absolute Change in Pre-bronchodilator FEV1 Percent Predicted From Baseline to Week 8
|
-2.10 percent
Standard Deviation 4.446
|
2.90 percent
Standard Deviation 3.461
|
SECONDARY outcome
Timeframe: Baseline; Week 8Population: Full Analysis Set
Outcome measures
| Measure |
Andecaliximab
n=3 Participants
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
n=3 Participants
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|
|
Absolute Change in Post-bronchodilator FEV1 Percent Predicted From Baseline to Week 8
|
1.01 percent
Standard Deviation 4.534
|
-1.45 percent
Standard Deviation 1.655
|
SECONDARY outcome
Timeframe: Baseline; Week 8Population: Full Analysis Set
Outcome measures
| Measure |
Andecaliximab
n=3 Participants
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
n=3 Participants
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|
|
Relative Change in Pre-bronchodilator FEV1 Percent Predicted From Baseline to Week 8
|
-3.85 percent change
Standard Deviation 9.009
|
4.41 percent change
Standard Deviation 4.196
|
SECONDARY outcome
Timeframe: Baseline; Week 8Population: Full Analysis Set
Outcome measures
| Measure |
Andecaliximab
n=3 Participants
Double-Blind Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
Placebo
n=3 Participants
Double-Blind Period: Placebo administered via subcutaneous injection weekly for 8 doses
Open-Label Period: Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|
|
Relative Change in Post-bronchodilator FEV1 Percent Predicted From Baseline to Week 8
|
2.28 percent change
Standard Deviation 9.323
|
-1.98 percent change
Standard Deviation 1.749
|
Adverse Events
Andecaliximab (Double-Blind)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Placebo (Double-Blind)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Andecaliximab (Open-Label)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Andecaliximab (Double-Blind)
n=3 participants at risk
Andecaliximab 600 mg administered via subcutaneous injection weekly for 8 weeks
|
Placebo (Double-Blind)
n=3 participants at risk
Placebo administered via subcutaneous injection weekly for 8 doses
|
Andecaliximab (Open-Label)
n=4 participants at risk
Andecaliximab 600 mg administered via subcutaneous injection weekly for up to 16 weeks
|
|---|---|---|---|
|
Congenital, familial and genetic disorders
Cystic fibrosis
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Gastrointestinal disorders
Gastritis
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
General disorders
Injection site bruising
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
General disorders
Injection site erythema
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
General disorders
Injection site haematoma
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
General disorders
Injection site pain
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
General disorders
Injection site pruritus
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
General disorders
Injection site rash
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
25.0%
1/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Infections and infestations
Oral herpes
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
25.0%
1/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Infections and infestations
Rhinitis
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Investigations
Bacterial test positive
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Nervous system disorders
Seizure
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
25.0%
1/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
50.0%
2/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
33.3%
1/3 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
0.00%
0/4 • Baseline up to the last dose date plus 30 days (maximum exposure: 140 days)
Safety Analysis Set
|
Additional Information
Gilead Clinical Study Information Center
Gilead Sciences
Phone: 1-833-445-3230 (GILEAD-0)
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee * The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or * The study has been completed at all study sites for at least 2 years
- Publication restrictions are in place
Restriction type: OTHER