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Clinical Trial Results:
A Phase 2, Randomized, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Filgotinib in Subjects with Active Noninfectious Uveitis

Summary
EudraCT number	2017-001485-17
Trial protocol	GB
Global end of trial date	22 Apr 2021

Results information
Results version number	v1(current)
This version publication date	11 Jan 2022
First version publication date	11 Jan 2022
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

GS-US-432-4097

ISRCTN number

US NCT number

NCT03207815

WHO universal trial number (UTN)

Sponsor organisation name

Gilead Sciences

Sponsor organisation address

333 Lakeside Drive, Foster City, CA, United States, 94404

Public contact

Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com

Scientific contact

Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

22 Apr 2021

Is this the analysis of the primary completion data?

Yes

Primary completion date

29 Dec 2020

Global end of trial reached?

Yes

Global end of trial date

22 Apr 2021

Was the trial ended prematurely?

Yes

Main objective of the trial

The primary objective of this study is to evaluate the efficacy of filgotinib versus placebo for the treatment of the signs and symptoms of noninfectious uveitis as measured by the percentage of participants failing treatment for active noninfectious uveitis by Week 24.

Protection of trial subjects

The protocol and consent/assent forms were submitted by each investigator to a duly constituted Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. All revisions to the consent/assent forms (if applicable) after initial IEC/IRB approval were submitted by the investigator to the IEC/IRB for review and approval before implementation in accordance with regulatory requirements. This study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.

Background therapy

Evidence for comparator

Actual start date of recruitment

26 Jul 2017

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Australia: 1

Country: Number of subjects enrolled

Canada: 2

Country: Number of subjects enrolled

Germany: 1

Country: Number of subjects enrolled

Israel: 1

Country: Number of subjects enrolled

New Zealand: 1

Country: Number of subjects enrolled

United Kingdom: 6

Country: Number of subjects enrolled

United States: 62

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Participants were enrolled at study sites in the United States, the United Kingdom, Canada, Australia, Germany, Israel, and New Zealand. The first participant was screened on 26 July 2017. The last study visit occurred on 22 April 2021.

Screening details

116 participants were screened.

Period 1 title

Overall Study (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Filgotinib

Arm description

Participants received filgotinib 200 milligrams (mg) tablet orally, once daily for up to 52 weeks along with a standardized prednisone burst of 60 mg/day at Day 1/Baseline followed by a protocol-defined mandatory taper schedule up to Week 15.

Arm type

Experimental

Investigational medicinal product name

Filgotinib

Investigational medicinal product code

Other name

GS-6034, GLPG0634

Pharmaceutical forms

Film-coated tablet

Routes of administration

Oral use

Dosage and administration details

200 mg administered once daily

Investigational medicinal product name

Prednisone

Investigational medicinal product code

Other name

Pharmaceutical forms

Film-coated tablet

Routes of administration

Oral use

Dosage and administration details

60 mg administered once on Day 1/Baseline

Placebo

Arm description

Participants received placebo to match filgotinib tablet orally, once daily for up to 52 weeks along with a standardized prednisone burst of 60 mg/day at Day 1/Baseline followed by a protocol-defined mandatory taper schedule up to Week 15.

Arm type

Placebo

Investigational medicinal product name

Placebo to match filgotinib

Investigational medicinal product code

Other name

Pharmaceutical forms

Film-coated tablet

Routes of administration

Oral use

Dosage and administration details

Administered once daily

Investigational medicinal product name

Prednisone

Investigational medicinal product code

Other name

Pharmaceutical forms

Film-coated tablet

Routes of administration

Oral use

Dosage and administration details

60 mg administered once on Day 1/Baseline

Number of subjects in period 1 ^[1]	Filgotinib	Placebo
Started	37	35
Completed	30	29
Not completed	7	6
Withdrew Consent	1	-
Adverse Event	1	2
Investigator's Discretion	1	1
Pregnancy/Partner Pregnancy	1	-
Protocol Violation	2	-
Lost to follow-up	1	2
Lack of efficacy	-	1

Notes
[1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
Justification: 2 participants who were enrolled but not treated were not included in the disposition table.

Baseline characteristics

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Reporting group title

Filgotinib

Reporting group description

Reporting group title

Placebo

Reporting group description

Reporting group values	Filgotinib	Placebo	Total
Number of subjects	37	35	72
Age categorical
Units: Subjects
Age continuous
Units: years
arithmetic mean (standard deviation)	48 ± 15.1	43 ± 15.7	-
Gender categorical
Units: Subjects
Female	23	20	43
Male	14	15	29
Race
Units: Subjects
Asian	4	0	4
Black or African American	2	8	10
Native Hawaiian or Pacific Islander	1	0	1
White	29	26	55
Other	1	1	2
Ethnicity
Not Permitted = local regulators did not allow collection of ethnicity information.
Units: Subjects
Hispanic or Latino	4	10	14
Not Hispanic or Latino	32	25	57
Not Permitted	1	0	1

End points

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Reporting group title

Filgotinib

Reporting group description

Reporting group title

Placebo

Reporting group description

Primary: Percentage of Participants Failing Treatment for Active NonInfectious Uveitis by Week 24

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End point title

Percentage of Participants Failing Treatment for Active NonInfectious Uveitis by Week 24

End point description

Treatment failure was a participant meeting at least 1 of these criteria in at least 1 eye: New active, inflammatory lesions relative to Day 1/Baseline (all visits starting Wk 6); Inability to achieve ≤Grade 0.5+ (at Wk 6)/2-step increase (change of Grade 0 to Grade 2+/Grade 0.5+ to Grade 3+) (all visits after Wk 6) relative to best state (RBS) achieved in Anterior Chamber (AC) cell grade (Standardization of Uveitis Nomenclature [SUN] criteria)[AC cell grades range from 0 (0 cells) to 4+ (>50 cells),higher scores=severe uveitis]; Inability to achieve ≤Grade 0.5+ (at Wk 6)/2-step increase (all visits after Wk 6) RBS achieved in Vitreous Haze (VH) grade (National Eye Institute [NEI]/SUN criteria)[VH grades range from 0(no evident VH) to 4+(optic nerve head is obscured),higher scores=severe uveitis]; Worsening of best corrected visual acuity (BCVA) by ≥15 letters RBS achieved (all visits starting Wk 6),measured by an eye chart,fewer correct letters=severe uveitis. Evaluable Analysis Set.

End point type

Primary

End point timeframe

Week 6 through Week (Wk) 24

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: percentage of participants
number (confidence interval 95%)	37.5 (19.2 to 55.8)	67.6 (50.5 to 84.8)

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0064 ^[1]

Method

Cochran-Mantel-Haenszel

Parameter type

Difference in Treatment Failure Rate

Point estimate

-30.1

Confidence interval

level

95%

sides

2-sided

lower limit

-56.2

upper limit

-4.1

Notes
[1] - P-value was estimated from the Cochran-Mantel-Haenszel (CMH) test, adjusted for the stratification factors.

Secondary: Time to Treatment Failure on or After Week 6

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End point title

Time to Treatment Failure on or After Week 6

End point description

Treatment failure was a participant meeting at least 1 of these criteria in at least 1 eye: New active, inflammatory lesions relative to Day 1/Baseline (all visits starting Wk 6); Inability to achieve ≤Grade 0.5+ (at Wk 6) or 2-step increase (change of Grade 0 to Grade 2+/Grade 0.5+ to Grade 3+) (all visits after Wk 6) relative to best state (RBS) achieved in AC cell grade (SUN criteria) [AC cell grades range from 0 (0 cells) to 4+ (>50 cells), higher scores=severe uveitis]; Inability to achieve ≤Grade 0.5+ (at Wk 6) or 2-step increase (all visits after Wk 6) RBS achieved in VH grade (NEI/SUN criteria) [VH grades range from 0 (no evident VH) to 4+ (optic nerve head is obscured), higher scores=severe uveitis]; Worsening of BCVA by ≥15 letters RBS achieved (all visits starting Wk 6), measured by an eye chart, fewer correct letters=severe uveitis. Participants in the Evaluable Analysis Set were analyzed. 9999=Not Available as the calculated percentiles of event rate were not reached.

End point type

Secondary

End point timeframe

Week 6 through Week 52

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: weeks
median (inter-quartile range (Q1-Q3))	9999 (24.1 to 9999)	22.0 (12.1 to 47.0)

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0014 ^[2]

Method

Stratified Log-Rank Test

Parameter type

Stratified Hazard Ratio

Point estimate

0.309

Confidence interval

level

95%

sides

2-sided

lower limit

0.144

upper limit

0.663

Notes
[2] - P-value was derived from the log rank test stratified by the stratification factors.

Secondary: Change in Vitreous Haze (VH) Grade in Each Eye (NEI/SUN Criteria), From Best State Achieved Prior to Week 6 to Week 52 or End of Treatment (EOT) Visit or Early Termination (ET)

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End point title

Change in Vitreous Haze (VH) Grade in Each Eye (NEI/SUN Criteria), From Best State Achieved Prior to Week 6 to Week 52 or End of Treatment (EOT) Visit or Early Termination (ET)

End point description

Grading of VH was based on the publication from the NEI which has also been adapted by the SUN working group. VH grades range from 0 (no evident VH) to 4+ (optic nerve head is obscured), with higher scores indicating greater severity of uveitis. A negative change (cha) from the best value obtained prior to Week 6 indicates improvement. Participants in the Evaluable Analysis Set with available data were analyzed.

End point type

Secondary

End point timeframe

Prior to Week 6; Up to Week 52 or EOT or ET (maximum: 53 weeks)

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: score
arithmetic mean (standard deviation)
Left Eye: Best State Prior to Wk 6 N=32,33	0.3 ± 0.40	0.2 ± 0.33
Right Eye: Best State Prior to Wk 6	0.3 ± 0.36	0.3 ± 0.45
Lef Eye Cha From Best State at Wk52/EOT/ET N=32,33	0.1 ± 0.79	0.3 ± 0.75
Rig Eye Cha From Best State at Wk52/EOT/ET N=32,33	0.1 ± 0.62	0.2 ± 0.66

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.355 ^[3]

Method

Repeated Measure ANCOVA

Parameter type

Least Squares Mean Treatment Difference

Point estimate

-0.1

Confidence interval

level

95%

sides

2-sided

lower limit

-0.4

upper limit

0.2

Variability estimate

Standard error of the mean

Dispersion value

0.14

Notes
[3] - P-value was estimated using a repeated measure Analysis of Covariance (ANCOVA) model which included treatment, eye, interaction of treatment and eye, stratification factors and best state value.

Secondary: Change in Anterior Chamber (AC) Cell Grade in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

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End point title

Change in Anterior Chamber (AC) Cell Grade in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

End point description

The number of AC cells observed within a 1 mm × 1 mm slit beam were recorded for each eye. The reported number was used to determine the grade according to the SUN criteria. AC cell grades range from 0 (0 cells in field) to 4+ (>50 cells in field), with higher scores indicating more cells visible in the AC and greater severity of uveitis. A negative change from the best state value obtained prior to Week 6 indicates improvement. Participants in the Evaluable Analysis Set were analyzed.

End point type

Secondary

End point timeframe

Prior to Week 6; Up to Week 52 or EOT or ET (maximum: 53 weeks)

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: score
arithmetic mean (standard deviation)
Left Eye: Best State Prior to Wk 6	0.0 ± 0.09	0.1 ± 0.19
Right Eye: Best State Prior to Wk 6	0.0 ± 0.12	0.1 ± 0.25
Left Eye: Change From Best State at Wk 52/EOT/ET	0.2 ± 0.59	0.6 ± 0.87
Right Eye: Change From Best State at Wk 52/EOT/ET	0.2 ± 0.53	0.7 ± 1.05

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0145 ^[4]

Method

Repeated Measure ANCOVA

Parameter type

LS Mean Treatment Difference

Point estimate

-0.4

Confidence interval

level

95%

sides

2-sided

lower limit

-0.8

upper limit

-0.1

Variability estimate

Standard error of the mean

Dispersion value

0.18

Notes
[4] - P-value was estimated using a repeated measure ANCOVA model which included treatment, eye, interaction of treatment and eye, stratification factors and best state value.

Secondary: Change in Logarithm of the Minimal Angle of Resolution (logMAR) Best Corrected Visual Acuity (BCVA) in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

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End point title

Change in Logarithm of the Minimal Angle of Resolution (logMAR) Best Corrected Visual Acuity (BCVA) in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

End point description

BCVA is the best possible vision that an eye can achieve with the set of glasses or contact lenses. A refraction test was performed to measure the appropriate lens strength to focus light on the retina. Using the appropriate corrective lenses based on that visit’s refraction, participant`s BCVA was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. In the ETDRS system, 15 letters is equal to a change in 3 lines of visual acuity. If the participant is unable to read letters on a testing chart, visual acuity is described as ranging from ability to count fingers, recognize hand movements, or light perception. The smaller BVCA score indicates greater severity of uveitis. A positive change from best state value obtained prior to Week 6 indicates improvement. Participants in the Evaluable Analysis Set were analyzed.

End point type

Secondary

End point timeframe

Prior to Week 6; Up to Week 52 or EOT or ET (maximum: 53 weeks)

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: logMAR
arithmetic mean (standard deviation)
Left Eye: Best State Prior to Wk 6	0.09 ± 0.195	0.07 ± 0.209
Right Eye: Best State Prior to Wk 6	0.09 ± 0.193	0.12 ± 0.280
Left Eye: Change From Best State at Wk 52/EOT/ET	0.03 ± 0.154	0.05 ± 0.112
Right Eye: Change From Best State at Wk 52/EOT/ET	-0.01 ± 0.116	0.07 ± 0.144

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0389 ^[5]

Method

Repeated Measure ANCOVA

Parameter type

LS Mean Treatment Difference

Point estimate

-0.05

Confidence interval

level

95%

sides

2-sided

lower limit

-0.1

upper limit

Variability estimate

Standard error of the mean

Dispersion value

0.025

Notes
[5] - P-value was estimated using a repeated measure ANCOVA model which included treatment, eye, interaction of treatment and eye, stratification factors and best state value.

Secondary: Log Change in Central Retinal Thickness in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

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End point title

Log Change in Central Retinal Thickness in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

End point description

Central retinal thickness is measured by optical coherence tomography (OCT). Central retinal thickness is defined as the thickness of the retina in the center of the foveal pit (1 mm subfield). The larger central retinal thickness value indicates greater severity of uveitis. A negative change (cha) from best state value obtained prior to Week 6 indicates improvement. Participants in the Evaluable Analysis Set with the available data were analyzed.

End point type

Secondary

End point timeframe

Prior to Week 6; Up to Week 52 or EOT or ET (maximum: 53 weeks)

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: log microns
arithmetic mean (standard deviation)
Left Eye: Best State Prior to Wk 6	2.45 ± 0.059	2.46 ± 0.097
Right Eye: Best State Prior to Wk 6	2.47 ± 0.056	2.44 ± 0.104
Lef Eye Cha From Best State at Wk52/EOT/ET N=32,32	0.01 ± 0.062	0.04 ± 0.080
Rig Eye Cha From Best State at Wk52/EOT/ET N=32,32	0.01 ± 0.049	0.03 ± 0.055

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.034 ^[6]

Method

Repeated Measure ANCOVA

Parameter type

LS Mean Treatment Difference

Point estimate

-0.02

Confidence interval

level

95%

sides

2-sided

lower limit

-0.05

upper limit

Variability estimate

Standard error of the mean

Dispersion value

0.011

Notes
[6] - P-value was estimated using a repeated measure ANCOVA model which included treatment, eye, interaction of treatment and eye, OCT machine, and best state value.

Secondary: Time to Development of Macular Edema in At Least One Eye on or After Week 6

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End point title

Time to Development of Macular Edema in At Least One Eye on or After Week 6

End point description

Time in weeks until the development of Macular edema or Week 52 or EOT or ET. Macular edema is determined by OCT and is defined as central retinal thickness ≥ 300 microns if using Cirrus machine, or ≥ 315 microns if using Spectralis machine. Participants in the Evaluable Analysis Set were analyzed. 9999=Not Available as the calculated percentiles of event rate were not reached.

End point type

Secondary

End point timeframe

Week 6 through Week 52

End point values	Filgotinib	Placebo
Number of subjects analysed	32	34
Units: weeks
median (inter-quartile range (Q1-Q3))	7.8 (6.1 to 9999)	12.3 (6.1 to 9999)

Filgotinib vs Placebo

Comparison groups

Filgotinib v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5893 ^[7]

Method

Stratified Log-Rank Test

Parameter type

Stratified Hazard Ratio

Point estimate

1.193

Confidence interval

level

95%

sides

2-sided

lower limit

0.625

upper limit

2.277

Notes
[7] - P-value was derived from the log rank test stratified by the stratification factors.

Secondary: Plasma Concentration of Filgotinib

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End point title

Plasma Concentration of Filgotinib ^[8]

End point description

Participants in the Safety Analysis Set who have at least one non-missing concentration data with available data were analyzed.

End point type

Secondary

End point timeframe

Day 1 post dose, Weeks 4 and 6 predose, Week 12 post dose, Weeks 24, 36, 52 (EOT), Early Termination at any time

Notes
[8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Plasma concentrations were collected and analyzed for filgotinib arm group.

End point values	Filgotinib
Number of subjects analysed	37
Units: nanograms per millilitre (ng/ml)
arithmetic mean (standard deviation)
Day 1 Postdose	748.5 ± 1043.84
Week 4 Predose (N=29)	173.3 ± 364.46
Week 6 Predose (N=28)	133.9 ± 306.18
Week 12 Postdose (N=27)	1088.7 ± 856.57
Week 24 Single Anytime (N=21)	397.5 ± 561.57
Week 36 Single Anytime (N=14)	295.7 ± 420.25
Week 52 (EOT) Single Anytime (N=22)	195.2 ± 344.54
Early Termination Single Anytime (N=3)	434.8 ± 478.72

No statistical analyses for this end point

Secondary: Plasma Concentration of Metabolite, GS-829845

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End point title

Plasma Concentration of Metabolite, GS-829845 ^[9]

End point description

Participants in the Safety Analysis Set who have at least one non-missing concentration data with available data were analyzed.

End point type

Secondary

End point timeframe

Day 1 post dose, Weeks 4 and 6 predose, Week 12 post dose, Weeks 24, 36, 52 (EOT), Early Termination at any time

Notes
[9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Plasma concentrations were collected and analyzed for filgotinib arm group.

End point values	Filgotinib
Number of subjects analysed	37
Units: ng/ml
arithmetic mean (standard deviation)
Day 1 Postdose	230.8 ± 317.98
Week 4 Predose (N=29)	2085.6 ± 924.91
Week 6 Predose (N=28)	2107.7 ± 749.28
Week 12 Postdose (N=27)	3237.0 ± 1180.74
Week 24 Single Anytime (N=21)	3478.1 ± 1137.63
Week 36 Single Anytime (N=14)	2944.3 ± 1130.84
Week 52 (EOT) Single Anytime (N=22)	2510.7 ± 1732.59
Early Termination Single Anytime (N=3)	2171.0 ± 1224.77

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Adverse Events: First dose date up to 30 days after the last dose of study drug (maximum exposure up to 57 weeks); All-Cause Mortality: From randomization up to 57 weeks

Adverse event reporting additional description

Adverse Events: Safety Analysis Set included all participants who received at least 1 dose of study drug. All- Cause Mortality: All Randomized Analysis Set included all participants who were randomized in the study.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

24.0

Reporting group title

Filgotinib

Reporting group description

Participants received filgotinib 200 mg tablet orally, once daily for up to 52 weeks along with a standardized prednisone burst of 60 mg/day at Day 1/Baseline followed by a protocol-defined mandatory taper schedule up to Week 15.

Reporting group title

Placebo

Reporting group description

Serious adverse events	Filgotinib	Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	5 / 37 (13.51%)	2 / 35 (5.71%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Nervous system disorders
Epilepsy
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Eye disorders
Retinal vasculitis
subjects affected / exposed	0 / 37 (0.00%)	1 / 35 (2.86%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Uveitis
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Gastrointestinal disorders
Inflammatory bowel disease
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Psychiatric disorders
Suicidal ideation
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Renal and urinary disorders
Bladder prolapse
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Spinal stenosis
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Infections and infestations
Covid-19
subjects affected / exposed	1 / 37 (2.70%)	0 / 35 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Urinary tract infection
subjects affected / exposed	0 / 37 (0.00%)	1 / 35 (2.86%)
occurrences causally related to treatment / all	0 / 0	1 / 1
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Filgotinib	Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	28 / 37 (75.68%)	19 / 35 (54.29%)
Investigations
Intraocular pressure increased
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Weight increased
subjects affected / exposed	3 / 37 (8.11%)	1 / 35 (2.86%)
occurrences all number	3	1
Injury, poisoning and procedural complications
Fall
subjects affected / exposed	3 / 37 (8.11%)	0 / 35 (0.00%)
occurrences all number	3	0
Vascular disorders
Hypertension
subjects affected / exposed	1 / 37 (2.70%)	3 / 35 (8.57%)
occurrences all number	2	3
Nervous system disorders
Headache
subjects affected / exposed	3 / 37 (8.11%)	7 / 35 (20.00%)
occurrences all number	4	7
Dizziness
subjects affected / exposed	3 / 37 (8.11%)	0 / 35 (0.00%)
occurrences all number	3	0
General disorders and administration site conditions
Fatigue
subjects affected / exposed	3 / 37 (8.11%)	3 / 35 (8.57%)
occurrences all number	3	3
Eye disorders
Dry eye
subjects affected / exposed	2 / 37 (5.41%)	4 / 35 (11.43%)
occurrences all number	2	4
Visual impairment
subjects affected / exposed	5 / 37 (13.51%)	1 / 35 (2.86%)
occurrences all number	5	1
Chorioretinal disorder
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Eye irritation
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Uveitis
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Vitreous floaters
subjects affected / exposed	3 / 37 (8.11%)	1 / 35 (2.86%)
occurrences all number	3	1
Anterior chamber inflammation
subjects affected / exposed	1 / 37 (2.70%)	2 / 35 (5.71%)
occurrences all number	1	2
Cataract
subjects affected / exposed	2 / 37 (5.41%)	1 / 35 (2.86%)
occurrences all number	2	1
Eye pain
subjects affected / exposed	2 / 37 (5.41%)	1 / 35 (2.86%)
occurrences all number	2	1
Macular oedema
subjects affected / exposed	2 / 37 (5.41%)	1 / 35 (2.86%)
occurrences all number	2	1
Photopsia
subjects affected / exposed	3 / 37 (8.11%)	0 / 35 (0.00%)
occurrences all number	3	0
Chalazion
subjects affected / exposed	0 / 37 (0.00%)	2 / 35 (5.71%)
occurrences all number	0	2
Ocular hyperaemia
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Photophobia
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Vitreous haze
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Gastrointestinal disorders
Nausea
subjects affected / exposed	4 / 37 (10.81%)	1 / 35 (2.86%)
occurrences all number	4	1
Abdominal pain
subjects affected / exposed	4 / 37 (10.81%)	0 / 35 (0.00%)
occurrences all number	4	0
Diarrhoea
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Dyspepsia
subjects affected / exposed	3 / 37 (8.11%)	1 / 35 (2.86%)
occurrences all number	3	1
Abdominal distension
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Constipation
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Dry mouth
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Sleep apnoea syndrome
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Psychiatric disorders
Insomnia
subjects affected / exposed	4 / 37 (10.81%)	4 / 35 (11.43%)
occurrences all number	4	4
Anxiety
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Depression
subjects affected / exposed	2 / 37 (5.41%)	1 / 35 (2.86%)
occurrences all number	2	1
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	3 / 37 (8.11%)	2 / 35 (5.71%)
occurrences all number	4	2
Back pain
subjects affected / exposed	3 / 37 (8.11%)	1 / 35 (2.86%)
occurrences all number	3	1
Pain in extremity
subjects affected / exposed	2 / 37 (5.41%)	2 / 35 (5.71%)
occurrences all number	2	2
Myalgia
subjects affected / exposed	1 / 37 (2.70%)	2 / 35 (5.71%)
occurrences all number	1	2
Muscle spasms
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0
Musculoskeletal stiffness
subjects affected / exposed	0 / 37 (0.00%)	2 / 35 (5.71%)
occurrences all number	0	2
Infections and infestations
Urinary tract infection
subjects affected / exposed	4 / 37 (10.81%)	3 / 35 (8.57%)
occurrences all number	6	3
Upper respiratory tract infection
subjects affected / exposed	3 / 37 (8.11%)	2 / 35 (5.71%)
occurrences all number	3	3
Bronchitis
subjects affected / exposed	1 / 37 (2.70%)	2 / 35 (5.71%)
occurrences all number	1	2
Covid-19
subjects affected / exposed	2 / 37 (5.41%)	0 / 35 (0.00%)
occurrences all number	2	0

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Were there any global substantial amendments to the protocol? Yes

14 Nov 2017

• ECG collection at the Screening visit only. • Allowed for the safety laboratory assessments to be performed locally or centrally at Screening visit only. • Removed lens photography and reduced fundus photography assessments. • Removed sarcoidosis assessments and sarcoidosis imaging requirements. • Clarified requirements for Unscheduled visits related to uveitis symptoms. • Updated Inclusion criteria to clarify oral corticosteroid requirements prior to Day 1 dosing. • Updated Exclusion criteria to clarify severe glaucoma. • Updated Inclusion criteria to clarify QuantiFERON® TB Gold and TB testing requirements. • Updated Exclusion criteria to clarify immunosuppressive therapy. • Updated Exclusion criteria to clarify concomitant immunosuppressive therapy. • Updated Exclusion criteria to exclude eyelid surgery from prior ocular surgery. • Updated Exclusion criteria to exclude eyelid surgery from planned (elective) eye surgery. • Updated Exclusion criteria to clarify HIV and Syphilis testing requirements. • Updated Exclusion criteria to define marijuana and tobacco use. • Updated storage and handling conditions for filgotinib. • Updated tonometry requirements. • Updated Fluorescein Angiogram requirements. • Updated risk/benefit assessment language. • Updated study drug return or disposal language. • Updated study schema. • Updated prohibited medications. • Updated study title. • Updated pregnancy precautions, definition for female of childbearing potential, and contraceptive requirements. • Updated clinical laboratory assessment table. • Updated references. • Added Ct.gov NCT number.

18 Dec 2017

• Updated synopsis, study design to correct inconsistencies made when incorporating changes into protocol amendment 1. • Updated synopsis and main eligibility criteria to correct inconsistencies between changes outlined in the summary of changes document and protocol amendment 1. • Updated synopsis, study procedures/frequency to delete lipid profile from screening. • Updated Inclusion criteria to correct inconsistencies made when incorporating changes into protocol amendment 1. • Updated subscripts to provide clarity around HBV Viral Monitoring and also, childbearing potential. • Updated to clarify the definition of childbearing potential.

12 Dec 2019

• Relaxation of entry criteria to allow participants that have previously failed anti-TNF treatment. • Relaxation of entry criteria to allow participants with previously treated active or latent TB. • DSPH (Drug Safety and Public Health) was replaced by PVE (Pharmacovigilance & Epidemiology) within the whole document.

17 Mar 2020

• Expand the sample size to N = 248 participants. • Expand the number of sites globally to approximately 75 centers. • Incorporate an independent adjudication committee to review major adverse cardiovascular events and thromboembolic events. • Add a new study drug discontinuation criterion: any thromboembolic events meeting serious adverse event (SAE) reporting criteria. • Language regarding independent adjudication committee reviews for major adverse cardiovascular events and thromboembolic events has been updated throughout the protocol.

Were there any global interruptions to the trial? Yes

Date	Interruption	Restart date
20 Mar 2020	There were global interruptions to enrollment due to corona virus disease 2019 (COVID-19) outbreak, measures were taken to introduce a global screening halt. Decision to restart enrolment was made on data monitoring committee (DMC) recommendation.	21 May 2020

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

Study was terminated early due to termination of the development program. Due to early termination and small sample size, PK analysis was not performed for this study.

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Clinical trials

Clinical Trial Results:
A Phase 2, Randomized, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Filgotinib in Subjects with Active Noninfectious Uveitis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Percentage of Participants Failing Treatment for Active NonInfectious Uveitis by Week 24

Primary: Percentage of Participants Failing Treatment for Active NonInfectious Uveitis by Week 24

Secondary: Time to Treatment Failure on or After Week 6

Secondary: Time to Treatment Failure on or After Week 6

Secondary: Change in Vitreous Haze (VH) Grade in Each Eye (NEI/SUN Criteria), From Best State Achieved Prior to Week 6 to Week 52 or End of Treatment (EOT) Visit or Early Termination (ET)

Secondary: Change in Vitreous Haze (VH) Grade in Each Eye (NEI/SUN Criteria), From Best State Achieved Prior to Week 6 to Week 52 or End of Treatment (EOT) Visit or Early Termination (ET)

Secondary: Change in Anterior Chamber (AC) Cell Grade in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Change in Anterior Chamber (AC) Cell Grade in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Change in Logarithm of the Minimal Angle of Resolution (logMAR) Best Corrected Visual Acuity (BCVA) in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Change in Logarithm of the Minimal Angle of Resolution (logMAR) Best Corrected Visual Acuity (BCVA) in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Log Change in Central Retinal Thickness in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Log Change in Central Retinal Thickness in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Time to Development of Macular Edema in At Least One Eye on or After Week 6

Secondary: Time to Development of Macular Edema in At Least One Eye on or After Week 6

Secondary: Plasma Concentration of Filgotinib

Secondary: Plasma Concentration of Filgotinib

Secondary: Plasma Concentration of Metabolite, GS-829845

Secondary: Plasma Concentration of Metabolite, GS-829845

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Greece
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Italy
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United Kingdom
Outside EU/EEA

Clinical trials

Clinical Trial Results: A Phase 2, Randomized, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Filgotinib in Subjects with Active Noninfectious Uveitis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Percentage of Participants Failing Treatment for Active NonInfectious Uveitis by Week 24

Primary: Percentage of Participants Failing Treatment for Active NonInfectious Uveitis by Week 24

Secondary: Time to Treatment Failure on or After Week 6

Secondary: Time to Treatment Failure on or After Week 6

Secondary: Change in Vitreous Haze (VH) Grade in Each Eye (NEI/SUN Criteria), From Best State Achieved Prior to Week 6 to Week 52 or End of Treatment (EOT) Visit or Early Termination (ET)

Secondary: Change in Vitreous Haze (VH) Grade in Each Eye (NEI/SUN Criteria), From Best State Achieved Prior to Week 6 to Week 52 or End of Treatment (EOT) Visit or Early Termination (ET)

Secondary: Change in Anterior Chamber (AC) Cell Grade in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Change in Anterior Chamber (AC) Cell Grade in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Change in Logarithm of the Minimal Angle of Resolution (logMAR) Best Corrected Visual Acuity (BCVA) in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Change in Logarithm of the Minimal Angle of Resolution (logMAR) Best Corrected Visual Acuity (BCVA) in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Log Change in Central Retinal Thickness in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Log Change in Central Retinal Thickness in Each Eye, From Best State Achieved Prior to Week 6 to Week 52 or EOT Visit or ET

Secondary: Time to Development of Macular Edema in At Least One Eye on or After Week 6

Secondary: Time to Development of Macular Edema in At Least One Eye on or After Week 6

Secondary: Plasma Concentration of Filgotinib

Secondary: Plasma Concentration of Filgotinib

Secondary: Plasma Concentration of Metabolite, GS-829845

Secondary: Plasma Concentration of Metabolite, GS-829845

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 2, Randomized, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Filgotinib in Subjects with Active Noninfectious Uveitis