Trial Outcomes & Findings for A Study Evaluating the Effect of Filgotinib Dose De-escalation in Participants With Ulcerative Colitis (UC) in Remission (NCT NCT05479058)

Last Updated: 2024-10-04

Results Overview

The mMCS is a tool designed to measure disease activity for ulcerative colitis. The mMCS was calculated as the sum of the 3 subscores: stool frequency, rectal bleeding, and endoscopy. Each subscore was graded from 0 to 3 with higher scores indicating more severe disease activity. The total mMCS score ranged from 0 to 9 with higher scores indicating more severe disease activity. The mMCS remission was defined as a total score of score ≤2, with endoscopic subscore of ≤1, stool frequency subscore of ≤1, and a rectal bleeding subscore of 0. Corticosteroid-free mMCS remission was defined as being free of corticosteroids for at least 12 weeks.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

22 participants

Primary outcome timeframe

Week 48

Results posted on

2024-10-04

Participant Flow

Participants who were in clinical remission with filgotinib 200 milligrams (mg) once daily for at least 2 consecutive quarterly visits in the ongoing long-term extension (LTE) SELECTION-LTE study (GS-US-418-3899; NCT02914535) and who met the eligibility criteria, were rolled over and randomized to this study. Participants were enrolled at study sites in Poland, France, Germany, the United States, Belgium, Republic of Korea, Spain, and the United Kingdom.

The clinical trial was originally designed with the primary endpoint to be assessed at Week 48 after which participants would have received unblinded treatment. Due to early termination of the study, none of the participants completed 48 weeks of treatment. All participants participated in blinded treatment period only and the study was unblinded globally after study completion.

Participant milestones

Participant milestones
Measure	Filgotinib 200 mg Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Overall Study STARTED	11	11
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	11	11

Reasons for withdrawal

Reasons for withdrawal
Measure	Filgotinib 200 mg Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Overall Study Withdrawal by Subject	1	1
Overall Study Study terminated by sponsor	10	10

Baseline Characteristics

A Study Evaluating the Effect of Filgotinib Dose De-escalation in Participants With Ulcerative Colitis (UC) in Remission

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Filgotinib 200 mg n=11 Participants Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=11 Participants Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.	Total n=22 Participants Total of all reporting groups
Age, Continuous	56.5 years STANDARD_DEVIATION 8.5 • n=5 Participants	58.3 years STANDARD_DEVIATION 10.7 • n=7 Participants	57.4 years STANDARD_DEVIATION 9.5 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	6 Participants n=7 Participants	14 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants	11 Participants n=7 Participants	21 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	1 Participants n=5 Participants	0 Participants n=7 Participants	1 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	10 Participants n=5 Participants	11 Participants n=7 Participants	21 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

PRIMARY outcome

Timeframe: Week 48

Population: As the study was terminated prior to any participant reaching the primary analysis time point at Week 48, the data for this endpoint was not collected and analyzed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline up to Week 48

Population: Participants in the Full Analysis Set (FAS) (all randomized participants who were administered study drug at least once) were analyzed.

PRO2 flare was defined as a PRO2 score worsening of at least 2 points and an absolute PRO2 score of at least 3, with stool frequency subscore ≥2, and rectal bleeding subscore ≥1. PRO2 included items of stool frequency and rectal bleeding. The range of each item score was 0 to 3 with higher scores indicating more severe disease.

Outcome measures

Outcome measures
Measure	Filgotinib 200 mg n=11 Participants Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=11 Participants Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Time to Patient-Reported Outcome Based on 2 Items (PRO2) Flare	NA days Due to insufficient number of participants with PRO2 flare, the median and 95% CI data could not be calculated.	NA days Due to insufficient number of participants with PRO2 flare, the median and 95% CI data could not be calculated.

SECONDARY outcome

Timeframe: Baseline up to Week 48

Population: Participants in the FAS were analyzed.

An ES-confirmed UC flare was defined as an increase in rectal bleeding subscore by at least 1 point and an increase in stool frequency subscore by at least 2 points and an increase in endoscopic subscore by at least 1 point. Each subscore graded from 0 to 3 with higher scores indicating more severe disease.

Outcome measures

Outcome measures
Measure	Filgotinib 200 mg n=11 Participants Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=11 Participants Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Time to ES-Confirmed UC Flare	NA days Due to insufficient number of participants with ES-confirmed UC flare, the median and 95% CI data could not be calculated.	NA days Due to insufficient number of participants with ES-confirmed UC flare, the median and 95% CI data could not be calculated.

SECONDARY outcome

Timeframe: Baseline, Week 4, Week 12, Week 24, Week 36, and Week 48

Population: Participants in the FAS with available data were analyzed. As the study was terminated prior to any participant reaching the Week 48 time point, the data for Week 48 time point was not collected and analyzed.

CRP is an acute-phase protein which provides an objective criterion of inflammatory activity.

Outcome measures

Outcome measures
Measure	Filgotinib 200 mg n=9 Participants Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=10 Participants Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Change From Baseline in C-Reactive Protein (CRP) Change at Week 4	0.166 milligrams per liter (mg/L) Standard Deviation 0.533	1.713 milligrams per liter (mg/L) Standard Deviation 4.423
Change From Baseline in C-Reactive Protein (CRP) Change at Week 12	0.532 milligrams per liter (mg/L) Standard Deviation 1.128	10.406 milligrams per liter (mg/L) Standard Deviation 26.944
Change From Baseline in C-Reactive Protein (CRP) Change at Week 24	0.156 milligrams per liter (mg/L) Standard Deviation 0.128	16.002 milligrams per liter (mg/L) Standard Deviation 34.093
Change From Baseline in C-Reactive Protein (CRP) Change at Week 36	0.367 milligrams per liter (mg/L) Standard Deviation 0.192	1.443 milligrams per liter (mg/L) Standard Deviation 0.525

SECONDARY outcome

Timeframe: Baseline, Week 4, Week 12, Week 24, Week 36, and Week 48

Fecal calprotectin, a very stable biomarker, was a 36 kilodalton calcium and zinc binding protein of S-100 protein family which was neutrophil derived. It represents 60% of cytosolic proteins in neutrophils and was a measurement of neutrophil migration to the gastrointestinal tract.

Outcome measures

Outcome measures
Measure	Filgotinib 200 mg n=7 Participants Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=8 Participants Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Change From Baseline in Fecal Calprotectin (FCP) Change at Week 4	-27.6 milligrams per kilogram (mg/kg) Standard Deviation 37.2	142.5 milligrams per kilogram (mg/kg) Standard Deviation 307.7
Change From Baseline in Fecal Calprotectin (FCP) Change at Week 12	-16.7 milligrams per kilogram (mg/kg) Standard Deviation 48.1	920.6 milligrams per kilogram (mg/kg) Standard Deviation 1996.2
Change From Baseline in Fecal Calprotectin (FCP) Change at Week 24	-52.0 milligrams per kilogram (mg/kg) Standard Deviation 54.6	374.3 milligrams per kilogram (mg/kg) Standard Deviation 674.8
Change From Baseline in Fecal Calprotectin (FCP) Change at Week 36	-46.5 milligrams per kilogram (mg/kg) Standard Deviation 65.8	-8.0 milligrams per kilogram (mg/kg) Standard Deviation 70.4

SECONDARY outcome

Timeframe: Baseline, Week 48

Population: As the study was terminated prior to any participant reaching the Week 48 time point, the data for this endpoint was not collected and analyzed.

The IBDQ is disease-specific questionnaire used for an assessment of Health Related Quality of Life (HRQoL) in participants with the Inflammatory Bowel Disease (IBD). It comprised of 32 questions divided into four health subscales: bowel symptoms (10 questions); systemic symptoms, including sleep disorders and fatigue (5 questions); emotional function such as depression, aggression, and irritation (12 questions); and social function, meaning the ability to participate in social activities and to work (5 questions). The IBDQ total score was calculated as the sum of the responses (each ranging from 1 \[severe problem\] to 7 \[normal health\]) to all 32 questions. Total IBDQ score ranged from 32 to 224 with a higher score indicating a better HRQoL.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline up to Week 48

Population: Participants in the Safety analysis set were analyzed.

An adverse event (AE) was any untoward medical occurrence, new or worsening of any preexisting condition, in a clinical study participant administered a medicinal product and which did not necessarily had to have a causal relationship with this treatment. A TEAE was defined as * An AE which had a start date equal to or after the date of the first administration of study drug in this study and no later than 30 days after last administration of study drug. * And was either a newly reported event, or a worsening of an existing event. Serious TEAE was defined as a TEAE that * Resulted in death and was life-threatening; * Required in-patient hospitalization or prolongation of existing hospitalization; * Resulted in persistent or significant disability/incapacity; * Was a congenital anomaly / birth defect; * Was medically significant.

Outcome measures

Outcome measures
Measure	Filgotinib 200 mg n=11 Participants Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=11 Participants Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (SAEs), and TEAEs Leading to Treatment Discontinuation TEAEs	6 Participants	6 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (SAEs), and TEAEs Leading to Treatment Discontinuation Serious TEAEs	0 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (SAEs), and TEAEs Leading to Treatment Discontinuation TEAEs Leading to Treatment Discontinuation	0 Participants	0 Participants

Adverse Events

Filgotinib 200 mg

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Filgotinib 100 mg

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Galapagos Medical Information

Galapagos N.V.

Phone: +32 15 342 900

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The sponsor must review and approve any results of the study or abstracts for professional meetings prepared by the investigator(s). Published data must not compromise the objectives of the study. Data from individual study centers in multicenter studies must not be published separately.
Publication restrictions are in place

Restriction type: OTHER

Measure	Filgotinib 200 mg n=11 participants at risk Participants received filgotinib 200 mg and placebo to match filgotinib 100 mg once daily orally.	Filgotinib 100 mg n=11 participants at risk Participants received filgotinib 100 mg and placebo to match filgotinib 200 mg once daily orally.
Infections and infestations Gastroenteritis	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Infections and infestations Campylobacter infection	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Infections and infestations Conjunctivitis	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Infections and infestations Laryngitis	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Infections and infestations Nasopharyngitis	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Infections and infestations Sinusitis	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Gastrointestinal disorders Colitis ulcerative	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	18.2% 2/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Gastrointestinal disorders Diarrhoea	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	18.2% 2/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Gastrointestinal disorders Vomiting	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	18.2% 2/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Gastrointestinal disorders Abdominal pain upper	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Gastrointestinal disorders Nausea	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Musculoskeletal and connective tissue disorders Back pain	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Musculoskeletal and connective tissue disorders Bursitis	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Musculoskeletal and connective tissue disorders Tendonitis	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
General disorders Fatigue	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
General disorders Influenza like illness	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Metabolism and nutrition disorders Hypercholesterolaemia	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Metabolism and nutrition disorders Vitamin D deficiency	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Injury, poisoning and procedural complications Tendon rupture	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Investigations Liver function test abnormal	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Renal and urinary disorders Renal colic	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Skin and subcutaneous tissue disorders Actinic keratosis	0.00% 0/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	9.1% 1/11 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.
Social circumstances Postmenopause	0.00% 0/8 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.	16.7% 1/6 • Baseline up to Week 48 The Safety Analysis Set included all randomized participants who were administered study drug at least once.