Trial Outcomes & Findings for Evaluation of Safety, Efficacy, Pharmacokinetic and Pharmacodynamic of Bertilimumab in Patients With Active Moderate to Severe Ulcerative Colitis (NCT NCT01671956)

NCT ID: NCT01671956

Last Updated: 2025-11-25

Results Overview

The Total Mayo score is an instrument designed to measure disease activity of ulcerative colitis and ranged from 0 (normal or inactive disease) to 12 (severe disease). It is a composite of 4 sub-scores: Stool frequency sub-score, rectal bleeding sub-score, endoscopic finding sub-score, and physician's global assessment sub-score, each of which ranges from 0 (normal) to 3 (severe disease). The sub-scores were summed to give a total score that ranged from 0-12. Change in mayo score was calculated as the sum of scores at Day 56 minus the sum of scores at Baseline divided by 14 for bertilimumab arm and sum of scores at Day 56 minus the sum of scores at Baseline divided by 6 for placebo arm.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

Baseline, Day 56

Results posted on

2025-11-25

Participant Flow

The study was initiated in Jul 2015 and was early terminated due to sponsor decision.

Participant milestones

Participant milestones
Measure	Bertilimumab Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg intravenous (IV) infusion biweekly for 12 weeks.	Placebo Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.
Overall Study STARTED	20	12
Overall Study Received at Least 1 Dose of Study Drug	20	12
Overall Study COMPLETED	18	10
Overall Study NOT COMPLETED	2	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Bertilimumab Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg intravenous (IV) infusion biweekly for 12 weeks.	Placebo Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.
Overall Study Disease progression	0	1
Overall Study Withdrawal by Subject	1	1
Overall Study Adverse Event	1	0

Baseline Characteristics

Evaluation of Safety, Efficacy, Pharmacokinetic and Pharmacodynamic of Bertilimumab in Patients With Active Moderate to Severe Ulcerative Colitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bertilimumab n=20 Participants Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg IV infusion biweekly for 12 weeks.	Placebo n=12 Participants Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.	Total n=32 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=45 Participants	12 Participants n=12929 Participants	31 Participants n=6349 Participants
Age, Categorical >=65 years	1 Participants n=45 Participants	0 Participants n=12929 Participants	1 Participants n=6349 Participants
Sex: Female, Male Female	9 Participants n=45 Participants	7 Participants n=12929 Participants	16 Participants n=6349 Participants
Sex: Female, Male Male	11 Participants n=45 Participants	5 Participants n=12929 Participants	16 Participants n=6349 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants
Race (NIH/OMB) Asian	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants
Race (NIH/OMB) Black or African American	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants
Race (NIH/OMB) White	20 Participants n=45 Participants	12 Participants n=12929 Participants	32 Participants n=6349 Participants
Race (NIH/OMB) More than one race	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=45 Participants	0 Participants n=12929 Participants	0 Participants n=6349 Participants

PRIMARY outcome

Timeframe: Baseline, Day 56

Population: The modified intent to treat (mITT) analysis set included all randomized participants who received at least one dose of study treatment (placebo or bertilimumab) and had at least a full Mayo score at screening and at least one post-baseline partial Mayo score from visit 3 onwards. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Bertilimumab n=14 Participants Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg IV infusion biweekly for 12 weeks.	Placebo n=6 Participants Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.
Change From Baseline in Total Mayo Score at Day 56	2.7 scores on a scale Interval 0.0 to 8.0	2.5 scores on a scale Interval 1.0 to 6.0

SECONDARY outcome

Timeframe: Baseline, Day 56

Population: The mITT analysis set included all randomized participants who received at least one dose of study treatment (placebo or bertilimumab) and had at least a full Mayo score at screening and at least one post-baseline partial Mayo score from visit 3 onwards. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.

The UCEIS is the validated index for the assessment of overall endoscopic activity. The model incorporated the vascular pattern score (0-2), the presence of bleeding score (0-3) and the presence of erosions and ulcers score (0-3). The total score ranged from 0-8. Higher scores indicated more severe disease. Change in mayo score was calculated as the sum of scores at Day 56 minus the sum of scores at Baseline divided by 15 for bertilimumab arm and sum of scores at Day 56 minus the sum of scores at Baseline divided by 6 for placebo arm.

Outcome measures

Outcome measures
Measure	Bertilimumab n=15 Participants Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg IV infusion biweekly for 12 weeks.	Placebo n=6 Participants Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.
Change From Baseline in Ulcerative Colitis Endoscopic Index of Severity (UCEIS) Score at Day 56	1.2 score on a scale Interval -3.0 to 5.0	0.4 score on a scale Interval -1.0 to 2.0

Adverse Events

Bertilimumab

Serious events: 2 serious events

Other events: 14 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bertilimumab n=20 participants at risk Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg IV infusion biweekly for 12 weeks.	Placebo n=12 participants at risk Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.
Immune system disorders Hypersenstivity	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Anal squamous cell carcinoma	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Gastrointestinal disorders Diarrhea	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.

Other adverse events

Additional Information

Alexion Pharmaceuticals, Inc.

Phone: 855-752-2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Bertilimumab n=20 participants at risk Participants with active moderate to severe ulcerative colitis received bertilimumab 10 mg/kg IV infusion biweekly for 12 weeks.	Placebo n=12 participants at risk Participants with active moderate to severe ulcerative colitis received placebo IV infusion matched to bertilimumab biweekly for 12 weeks.
Musculoskeletal and connective tissue disorders Back pain	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Gastrointestinal disorders Tongue disorder	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Skin and subcutaneous tissue disorders Dermatitis allergic	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Immune system disorders Drug hypersensitivity	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Investigations Cardiac murmur	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Investigations Blood alkaline phosphatase increased	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
General disorders Oedema peripheral	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Gastrointestinal disorders Abdominal distension	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Nervous system disorders Dysgeusia	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Injury, poisoning and procedural complications Infusion related reaction	15.0% 3/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Reproductive system and breast disorders Ovulation pain	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
General disorders Pyrexia	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	16.7% 2/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
General disorders Chest pain	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Musculoskeletal and connective tissue disorders Arthralgia	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Gastrointestinal disorders Abdominal pain	10.0% 2/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Psychiatric disorders Anxiety	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Skin and subcutaneous tissue disorders Acute febrile neutrophilic dermatosis	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Musculoskeletal and connective tissue disorders Osteopenia	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Infections and infestations Nasopharyngitis	10.0% 2/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Gastrointestinal disorders Vomiting	0.00% 0/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
General disorders Chest discomfort	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Nervous system disorders Dizziness	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	8.3% 1/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Infections and infestations Gastroenteritis	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Infections and infestations Sinusitis	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
General disorders Asthenia	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Nervous system disorders Headache	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	16.7% 2/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.
Gastrointestinal disorders Dyspepsia	5.0% 1/20 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.	0.00% 0/12 • Baseline up to Day 90 The safety analysis set included all participants who were exposed to any of the study treatments.