Trial Outcomes & Findings for VTX002 Versus Placebo for the Treatment of Moderately to Severely Active Ulcerative Colitis (NCT NCT05156125)

NCT ID: NCT05156125

Last Updated: 2024-11-13

Results Overview

The percentage of participants with clinical remission at Week 13. Clinical remission was based on the modified Mayo score (MMS), which is a composite score of participant-reported symptoms and endoscopies which were assessed by a central reader. Clinical remission was defined as stool frequency (SF) subscore = 0 or 1, rectal bleeding (RB) subscore = 0, and endoscopic subscore (ES) ≤ 1 (excluding friability). Each component subscore ranged from 0 to 3 and total score range of the MMS was from 0 to 9, with higher scores indicating more severe disease.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

213 participants

Primary outcome timeframe

Day 1 of Induction treatment period to Week 13

Results posted on

2024-11-13

Participant Flow

Participant milestones

Participant milestones
Measure
VTX002 60 mg Once Daily
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
Placebo tablet administered orally once daily
Overall Study
STARTED
70
73
70
Overall Study
Entered Long-Term Extension Following Induction
29
34
22
Overall Study
Entered Open-Label Extension Following Induction
37
36
42
Overall Study
Did Not Enter Long-Term or Open-Label Extension Following Induction
1
0
2
Overall Study
COMPLETED
67
70
66
Overall Study
NOT COMPLETED
3
3
4

Reasons for withdrawal

Reasons for withdrawal
Measure
VTX002 60 mg Once Daily
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
Placebo tablet administered orally once daily
Overall Study
Lack of Efficacy
1
1
1
Overall Study
Physician Decision
0
0
1
Overall Study
Non-compliance
0
1
1
Overall Study
Withdrawal by Subject
0
0
1
Overall Study
Adverse Event
2
1
0

Baseline Characteristics

VTX002 Versus Placebo for the Treatment of Moderately to Severely Active Ulcerative Colitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
VTX002 60 mg Once Daily
n=70 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=73 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 Participants
Placebo tablet administered orally once daily
Total
n=213 Participants
Total of all reporting groups
Age, Continuous
39.4 years
STANDARD_DEVIATION 13.81 • n=5 Participants
42.3 years
STANDARD_DEVIATION 15.01 • n=7 Participants
40.1 years
STANDARD_DEVIATION 13.78 • n=5 Participants
40.6 years
STANDARD_DEVIATION 14.21 • n=4 Participants
Age, Customized
18 to 44
49 Participants
n=5 Participants
41 Participants
n=7 Participants
46 Participants
n=5 Participants
136 Participants
n=4 Participants
Age, Customized
45 to 65
18 Participants
n=5 Participants
27 Participants
n=7 Participants
21 Participants
n=5 Participants
66 Participants
n=4 Participants
Age, Customized
>65
3 Participants
n=5 Participants
5 Participants
n=7 Participants
3 Participants
n=5 Participants
11 Participants
n=4 Participants
Sex: Female, Male
Female
39 Participants
n=5 Participants
26 Participants
n=7 Participants
32 Participants
n=5 Participants
97 Participants
n=4 Participants
Sex: Female, Male
Male
31 Participants
n=5 Participants
47 Participants
n=7 Participants
38 Participants
n=5 Participants
116 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
3 Participants
n=5 Participants
7 Participants
n=7 Participants
3 Participants
n=5 Participants
13 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
67 Participants
n=5 Participants
66 Participants
n=7 Participants
66 Participants
n=5 Participants
199 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
1 Participants
n=4 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Race (NIH/OMB)
Asian
8 Participants
n=5 Participants
4 Participants
n=7 Participants
7 Participants
n=5 Participants
19 Participants
n=4 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
0 Participants
n=4 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=5 Participants
1 Participants
n=7 Participants
0 Participants
n=5 Participants
2 Participants
n=4 Participants
Race (NIH/OMB)
White
60 Participants
n=5 Participants
67 Participants
n=7 Participants
63 Participants
n=5 Participants
190 Participants
n=4 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
1 Participants
n=7 Participants
0 Participants
n=5 Participants
1 Participants
n=4 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
1 Participants
n=4 Participants

PRIMARY outcome

Timeframe: Day 1 of Induction treatment period to Week 13

Population: Full Analysis Set - Baseline Modified Mayo Score 5-9

The percentage of participants with clinical remission at Week 13. Clinical remission was based on the modified Mayo score (MMS), which is a composite score of participant-reported symptoms and endoscopies which were assessed by a central reader. Clinical remission was defined as stool frequency (SF) subscore = 0 or 1, rectal bleeding (RB) subscore = 0, and endoscopic subscore (ES) ≤ 1 (excluding friability). Each component subscore ranged from 0 to 3 and total score range of the MMS was from 0 to 9, with higher scores indicating more severe disease.

Outcome measures

Outcome measures
Measure
VTX002 60 mg Once Daily
n=68 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=71 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 Participants
Placebo tablet administered orally once daily
Clinical Remission at 13 Weeks
19 Participants
17 Participants
8 Participants

SECONDARY outcome

Timeframe: Day 1 of Induction Treatment Period to Week 13

Population: Full Analysis Set - Baseline MMS 5 to 9

The percentage of participants with endoscopic improvement at Week 13. Endoscopic improvement was assessed from endoscopies assessed by a central reader. Endoscopic improvement was defined as ES ≤ 1 (excluding friability). The ES ranged from 0 to 3 (where 0 = normal/inactive disease and 3 = severe disease).

Outcome measures

Outcome measures
Measure
VTX002 60 mg Once Daily
n=68 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=71 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 Participants
Placebo tablet administered orally once daily
Endoscopic Improvement at Week 13
25 Participants
23 Participants
11 Participants

SECONDARY outcome

Timeframe: Day 1 of Induction Treatment Period to Week 13

Population: Full analysis set - Baseline MMS 5 to 9

The percentage of participants with symptomatic remission at Week 13. Symptomatic remission was measured using participant-reported symptoms and was defined as SF subscore = 0 or 1 and RB subscore = 0. The SF subscore ranged from 0 to 3 (where 0 = normal number of stools and 3 = at least 5 stools more than normal) and RB subscore ranged from 0 to 3 (where 0 = no blood and 3 = blood alone passes). Higher scores indicate more severe disease.

Outcome measures

Outcome measures
Measure
VTX002 60 mg Once Daily
n=68 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=71 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 Participants
Placebo tablet administered orally once daily
Symptomatic Remission at Week 13
29 Participants
29 Participants
18 Participants

SECONDARY outcome

Timeframe: Day 1 of Induction Treatment Period to Week 13

Population: Full analysis set - Baseline MMS 5 to 9

The percentage of participants with histologic remission at Week 13. Histologic remission was assessed using the Geboes Index score and defined for this outcome measure as a Geboes score \< 2.0. The Geboes score grading system is a validated score for evaluating histologic disease activity in ulcerative colitis and is graded on a scale of 0 to 5. A higher Geboes score indicates more severe disease.

Outcome measures

Outcome measures
Measure
VTX002 60 mg Once Daily
n=68 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=71 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 Participants
Placebo tablet administered orally once daily
Histologic Remission at Week 13
13 Participants
20 Participants
5 Participants

SECONDARY outcome

Timeframe: Day 1 of Induction Treatment Period to Week 13

Population: Full Analysis Set - Baseline MMS 5 to 9

The percentage of participants with endoscopic improvement-histologic remission at Week 13. This outcome measure was assessed by endoscopic histologic scores and defined as ES ≤ 1 (excluding friability) and a Geboes Index score \< 2.0. The ES ranged from 0 to 3 (where 0 = normal/inactive disease and 3 = severe disease). The Geboes score grading system is a validated score for evaluating histologic disease activity in ulcerative colitis and is graded on a scale of 0 to 5. A higher Geboes score indicates more severe disease.

Outcome measures

Outcome measures
Measure
VTX002 60 mg Once Daily
n=68 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=71 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 Participants
Placebo tablet administered orally once daily
Endoscopic Improvement-Histologic Remission at Week 13
7 Participants
14 Participants
0 Participants

SECONDARY outcome

Timeframe: Weeks 1, 4, 8, and 13 of the Induction Treatment Period

Population: The Pharmacokinetics Set consisted of all participants who received VTX002 and had at least 1 measured concentration at a scheduled PK timepoint after start of dosing for VTX002.

Plasma concentrations of VTX002 in samples obtained predose at Weeks 1, 4, 8, and 13 in the Induction Treatment Period

Outcome measures

Outcome measures
Measure
VTX002 60 mg Once Daily
n=70 Participants
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=73 Participants
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
Placebo tablet administered orally once daily
PK of VTX002
Week 1
640.8 ng/mL
Standard Deviation 378.77
363.2 ng/mL
Standard Deviation 237.79
PK of VTX002
Week 4
911.5 ng/mL
Standard Deviation 777.40
374.7 ng/mL
Standard Deviation 242.93
PK of VTX002
Week 8
935.1 ng/mL
Standard Deviation 718.16
393.2 ng/mL
Standard Deviation 251.98
PK of VTX002
Week 13
1050 ng/mL
Standard Deviation 930.15
444.2 ng/mL
Standard Deviation 341.13

Adverse Events

VTX002 60 mg Once Daily

Serious events: 3 serious events
Other events: 10 other events
Deaths: 0 deaths

VTX002 30 mg Once Daily

Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths

Placebo Once Daily

Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
VTX002 60 mg Once Daily
n=70 participants at risk
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=73 participants at risk
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 participants at risk
Placebo tablet for VTX002 administered orally once daily
Blood and lymphatic system disorders
Anaemia
1.4%
1/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
Hepatobiliary disorders
Cholecystitis acute
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
1.4%
1/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
Gastrointestinal disorders
Colitis ulcerative
1.4%
1/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
1.4%
1/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
Gastrointestinal disorders
Rectal haemorrhage
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
1.4%
1/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
0.00%
0/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.

Other adverse events

Other adverse events
Measure
VTX002 60 mg Once Daily
n=70 participants at risk
VTX002 60 mg tablet administered orally once daily
VTX002 30 mg Once Daily
n=73 participants at risk
VTX002 30 mg tablet administered orally once daily
Placebo Once Daily
n=70 participants at risk
Placebo tablet for VTX002 administered orally once daily
Infections and infestations
Upper respiratory tract infection
8.6%
6/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
1.4%
1/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
1.4%
1/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
Nervous system disorders
Headache
5.7%
4/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
6.8%
5/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
2.9%
2/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
Blood and lymphatic system disorders
Anaemia
2.9%
2/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
5.5%
4/73 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.
8.6%
6/70 • From the signing of the Informed Consent Form through 30 days after the last study treatment, up to 13 weeks in the Induction Treatment Period.

Additional Information

Clinical Trial Contact

Oppilan Pharma Ltd

Phone: +1(888)-411-5176

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: GT60