Trial Outcomes & Findings for Clinical Study of TA-650 in Pediatric Patients With Ulcerative Colitis (NCT NCT01585155)

NCT ID: NCT01585155

Last Updated: 2026-01-07

Results Overview

Clinical activity index (CAI) remission was defined as a case where a CAI score was not more than 4 on the evaluation day. CAI score was an activity index to evaluate disease activity, which is calculated as the sum (0 to 29 points) of subscores for 7 clinical conditions, consisting of the number of stools per week, blood in stools (based on weekly average), investigator's global assessment of the symptomatic state, abdominal pain/cramps, temperature elevation due to ulcerative colitis, extraintestinal manifestations, and laboratory findings (hemoglobin or ESR). A higher score indicates greater disease activity.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

21 participants

Primary outcome timeframe

Weeks 2, 6, 8, 10, 14, 18, 22, 26, 30, and the last time point during the period from administration of the study drug to Week 30

Results posted on

2026-01-07

Participant Flow

Participant milestones

Participant milestones
Measure
TA-650
Patients received TA-650 5 mg/kg at Weeks 0, 2, and 6; patients with a decreased CAI score at Week 8 were considered to be responders and received further doses at Week 14 and 22. Non-responders (patients with unchanged/increased CAI score) at Week 8 did not receive further study treatment. Assessments were conducted until Week 30 in responders and until Week 14 in non-responders.
Overall Study
STARTED
21
Overall Study
COMPLETED
16
Overall Study
NOT COMPLETED
5

Reasons for withdrawal

Reasons for withdrawal
Measure
TA-650
Patients received TA-650 5 mg/kg at Weeks 0, 2, and 6; patients with a decreased CAI score at Week 8 were considered to be responders and received further doses at Week 14 and 22. Non-responders (patients with unchanged/increased CAI score) at Week 8 did not receive further study treatment. Assessments were conducted until Week 30 in responders and until Week 14 in non-responders.
Overall Study
Adverse Event
1
Overall Study
Lack of Efficacy
2
Overall Study
Worsening ulcerative colitis
2

Baseline Characteristics

Clinical Study of TA-650 in Pediatric Patients With Ulcerative Colitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
TA-650
n=21 Participants
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
Age, Continuous
13.7 years
STANDARD_DEVIATION 2.1 • n=9 Participants
Sex: Female, Male
Female
10 Participants
n=9 Participants
Sex: Female, Male
Male
11 Participants
n=9 Participants

PRIMARY outcome

Timeframe: Weeks 2, 6, 8, 10, 14, 18, 22, 26, 30, and the last time point during the period from administration of the study drug to Week 30

Population: Five patients were discontinued. The reasons for discontinuation were adverse event in 1 patient, lack of efficacy in 2 patients, and worsening ulcerative colitis in 2 patients. Two non-responders were observed, and both of them completed the efficacy evaluation at Week 8.

Clinical activity index (CAI) remission was defined as a case where a CAI score was not more than 4 on the evaluation day. CAI score was an activity index to evaluate disease activity, which is calculated as the sum (0 to 29 points) of subscores for 7 clinical conditions, consisting of the number of stools per week, blood in stools (based on weekly average), investigator's global assessment of the symptomatic state, abdominal pain/cramps, temperature elevation due to ulcerative colitis, extraintestinal manifestations, and laboratory findings (hemoglobin or ESR). A higher score indicates greater disease activity.

Outcome measures

Outcome measures
Measure
TA-650
n=21 Participants
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
Percent of Patients Who Achieved CAI Remission
Weeks 2
60.0 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 6
80.0 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 8
80.0 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 10
77.8 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 14
87.5 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 18
87.5 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 22
64.3 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 26
85.7 percentage of participants
Percent of Patients Who Achieved CAI Remission
Weeks 30
64.3 percentage of participants
Percent of Patients Who Achieved CAI Remission
The last time point
42.9 percentage of participants

SECONDARY outcome

Timeframe: Baseline,Weeks 2, 6, 8, 10, 14, 18, 22, 26, 30, and the last time point during the period from administration of the study drug to Week 30

Population: Five patients were discontinued. The reasons for discontinuation were adverse event in 1 patient, lack of efficacy in 2 patients, and worsening ulcerative colitis in 2 patients. Two non-responders were observed, and both of them completed the efficacy evaluation at Week 8.

CAI score was an activity index to evaluate disease activity, which is calculated as the sum (0 to 29 points) of subscores for 7 clinical conditions, consisting of the number of stools per week, blood in stools (based on weekly average), investigator's global assessment of the symptomatic state, abdominal pain/cramps, temperature elevation due to ulcerative colitis, extraintestinal manifestations, and laboratory findings (hemoglobin or ESR). A higher score indicates greater disease activity.

Outcome measures

Outcome measures
Measure
TA-650
n=21 Participants
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
CAI Score
Baseline
9.7 units on a scale
Standard Deviation 2.7
CAI Score
Weeks 2
4.0 units on a scale
Standard Deviation 3.2
CAI Score
Weeks 6
3.5 units on a scale
Standard Deviation 3.5
CAI Score
Weeks 8
3.2 units on a scale
Standard Deviation 3.5
CAI Score
Weeks 10
2.7 units on a scale
Standard Deviation 2.9
CAI Score
Weeks 14
2.7 units on a scale
Standard Deviation 3.0
CAI Score
Weeks 18
2.6 units on a scale
Standard Deviation 2.6
CAI Score
Weeks 22
3.3 units on a scale
Standard Deviation 3.1
CAI Score
Weeks 26
2.5 units on a scale
Standard Deviation 2.3
CAI Score
Weeks 30
3.5 units on a scale
Standard Deviation 2.2
CAI Score
The last time point
5.6 units on a scale
Standard Deviation 3.8

SECONDARY outcome

Timeframe: Baseline,Weeks 2, 6, 8, 10, 14, 18, 22, 26, 30, and the last time point during the period from administration of the study drug to Week 30

Population: Five patients were discontinued. The reasons for discontinuation were adverse event in 1 patient, lack of efficacy in 2 patients, and worsening ulcerative colitis in 2 patients. Two non-responders were observed, and both of them completed the efficacy evaluation at Week 8.

Mayo score consists of four subscores (stool frequency, rectal bleeding, physician's global assessment and findings of endoscopy), each of which was assessed according to a four-level rating scale (0 to 3 points), and was determined from a total of the four subscores (0 to 12 points). In addition, the sum of the subscores (0 to 9 points) for stool frequency, rectal bleeding and physician's global assessment was used as a partial Mayo score. A higher score indicates greater disease activity.

Outcome measures

Outcome measures
Measure
TA-650
n=21 Participants
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
Partial Mayo Score
Baseline
5.6 units on a scale
Standard Deviation 1.6
Partial Mayo Score
Weeks 2
2.6 units on a scale
Standard Deviation 2.3
Partial Mayo Score
Weeks 6
2.2 units on a scale
Standard Deviation 2.0
Partial Mayo Score
Weeks 8
1.7 units on a scale
Standard Deviation 1.7
Partial Mayo Score
Weeks 10
2.2 units on a scale
Standard Deviation 2.1
Partial Mayo Score
Weeks 14
1.9 units on a scale
Standard Deviation 1.9
Partial Mayo Score
Weeks 18
2.1 units on a scale
Standard Deviation 1.8
Partial Mayo Score
Weeks 22
2.4 units on a scale
Standard Deviation 2.1
Partial Mayo Score
Weeks 26
1.8 units on a scale
Standard Deviation 1.7
Partial Mayo Score
Weeks 30
2.8 units on a scale
Standard Deviation 1.9
Partial Mayo Score
The last time point
3.7 units on a scale
Standard Deviation 2.2

SECONDARY outcome

Timeframe: Baseline,Weeks 2, 6, 8, 10, 14, 18, 22, 26, 30, and the last time point during the period from administration of the study drug to Week 30

Population: Five patients were discontinued. The reasons for discontinuation were adverse event in 1 patient, lack of efficacy in 2 patients, and worsening ulcerative colitis in 2 patients. Two non-responders were observed, and both of them completed the efficacy evaluation at Week 8.

PUCAI score was determined as a total of the subscores for each of the six evaluation items (0 to 85), including abdominal pain, rectal bleeding, stool consistency of most stools, number of stools per 24 hours, nocturnal stools and activity level. A higher score indicates greater disease activity.

Outcome measures

Outcome measures
Measure
TA-650
n=21 Participants
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
The last time point
28.8 units on a scale
Standard Deviation 22.5
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Baseline
47.1 units on a scale
Standard Deviation 15.2
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 2
20.3 units on a scale
Standard Deviation 16.3
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 6
17.3 units on a scale
Standard Deviation 17.5
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 8
12.5 units on a scale
Standard Deviation 13.5
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 10
14.7 units on a scale
Standard Deviation 16.9
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 14
12.2 units on a scale
Standard Deviation 12.0
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 18
14.4 units on a scale
Standard Deviation 16.8
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 22
18.2 units on a scale
Standard Deviation 15.6
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 26
13.2 units on a scale
Standard Deviation 14.0
Pediatric Ulcerative Colitis Activity Index (PUCAI) Score
Weeks 30
19.3 units on a scale
Standard Deviation 18.8

Adverse Events

TA-650

Serious events: 3 serious events
Other events: 19 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
TA-650
n=21 participants at risk
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
Gastrointestinal disorders
Colitis ulcerative
9.5%
2/21
Gastrointestinal disorders
Enterocolitis
4.8%
1/21

Other adverse events

Other adverse events
Measure
TA-650
n=21 participants at risk
TA-650 is administered at a dose of 5 mg/kg at Weeks 0, 2 and 6, followed by administration at 8-week intervals at Weeks 14 and 22, based on the evaluation index such as CAI score.
Infections and infestations
Bronchitis
4.8%
1/21
Infections and infestations
Enteritis infectious
9.5%
2/21
Infections and infestations
Gastroenteritis
4.8%
1/21
Infections and infestations
Nasopharyngitis
33.3%
7/21
Infections and infestations
Sinusitis
4.8%
1/21
Infections and infestations
Subcutaneous abscess
4.8%
1/21
Infections and infestations
Upper respiratory tract infection
9.5%
2/21
Blood and lymphatic system disorders
Anaemia
4.8%
1/21
Blood and lymphatic system disorders
Iron deficiency anaemia
9.5%
2/21
Nervous system disorders
Headache
4.8%
1/21
Eye disorders
Scleritis
4.8%
1/21
Vascular disorders
Vasculitis
4.8%
1/21
Respiratory, thoracic and mediastinal disorders
Asthma
4.8%
1/21
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
4.8%
1/21
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
4.8%
1/21
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
4.8%
1/21
Gastrointestinal disorders
Abdominal pain lower
4.8%
1/21
Gastrointestinal disorders
Abdominal pain upper
4.8%
1/21
Gastrointestinal disorders
Autoimmune pancreatitis
4.8%
1/21
Gastrointestinal disorders
Cheilitis
4.8%
1/21
Gastrointestinal disorders
Constipation
4.8%
1/21
Gastrointestinal disorders
Nausea
9.5%
2/21
Gastrointestinal disorders
Pancreatitis
4.8%
1/21
Gastrointestinal disorders
Vomiting
9.5%
2/21
Skin and subcutaneous tissue disorders
Acne
9.5%
2/21
Skin and subcutaneous tissue disorders
Pruritus
4.8%
1/21
Musculoskeletal and connective tissue disorders
Arthralgia
4.8%
1/21
Musculoskeletal and connective tissue disorders
Back pain
4.8%
1/21
Musculoskeletal and connective tissue disorders
Myalgia
4.8%
1/21
General disorders
Chest discomfort
4.8%
1/21
General disorders
Infusion site pain
4.8%
1/21
Investigations
Antinuclear antibody increased
4.8%
1/21
Investigations
Blood pressure decreased
4.8%
1/21
Investigations
Double stranded DNA antibody positive
57.1%
12/21
Investigations
Protein urine present
4.8%
1/21
Injury, poisoning and procedural complications
Thermal burn
4.8%
1/21

Additional Information

Clinical Trials, Information Desk

Tanabe Pharma Corporation

Results disclosure agreements

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

Restriction type: OTHER