Ulcerative Colitis
Conditions
Keywords
Mild, Moderate
Brief summary
To assess clinical response to MMX mesalamine/mesalazine between a low and high dose in children and adolescents aged 5-17 years with mild to moderate Ulcerative Colitis (UC) or who are in remission.
Interventions
DRUGMMX Mesalamine/Mesalazine (Low Dose)
Once daily, tablets - the amount depends on the participants weight; 900 milligram per day (mg/day) for participants weighing 18 kg to less than or equal to (\<=) 23 kilograms (kg); 1200 mg/day for participants weighing greater than (\>) 23 kg to \<= 35 kg; 1800 mg/day for participants weighing \> 35 kg to \<= 50 kg; 2400 mg/day for participants weighing \> 50 kg to \<= 90 kg.
DRUGMMX Mesalamine/Mesalazine (High Dose)
Once daily, tablets - the amount depends on the participants weight;1800 mg/day for participants weighing 18 kg to \<= 23 kg; 2400 mg/day for participants weighing \> 23 kg to \<= 35 kg; 3600 mg/day for participants weighing \> 35 kg to \<= 50 kg; 4800 mg/day for participants weighing \> 50 kg to \<= 90 kg.
Sponsors
Shire
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
5 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative \[LAR\]) informed consent or assent as applicable to participate in the study. 2. Subject's parent/LAR demonstrates an understanding, ability, and willingness to fully comply with study procedures and restrictions. 3. Male and female children and adolescents aged 5-17 years, inclusive. 4. Body weight 18-90kg. 5. Male, or non-pregnant, non-lactating female who agrees to comply with any applicable contraceptive requirements of the protocol or females of non-childbearing potential. 6. Diagnosed with mild to moderate UC, established by sigmoidoscopy or colonoscopy with compatible histology. Screened subjects may also have an unconfirmed diagnosis of mild to moderate UC; however the diagnosis of mild to moderate UC must have been established by sigmoidoscopy or colonoscopy with compatible histology prior to baseline visit. 7. Subject is able to swallow the investigational product whole. Double-blind Acute Phase: 8. Partial UC-DAI score ≥2 (a combined rectal bleeding and stool frequency score ≥1 and PGA=1 or 2) at the Baseline Visit, for which 5-ASA would be used as part of normal treatment. 9. If the subject is on 5-ASA treatment prior to study entry, then the dose must be stable. Stable therapy is defined as no change in dose, or no initiation of 5-ASA, from the onset of the current acute flare through discontinuation of therapy (required at the Baseline Visit). Double-blind Maintenance Phase: 10. Partial UC-DAI ≤1 (rectal bleeding=0, stool frequency ≤1, and PGA=0) at the Baseline Visit.
Exclusion criteria
1. Severe UC (defined by PGA=3). 2. Crohn's disease, bleeding disorders, active peptic ulcer disease, or UC known to be confined to the rectum (isolated rectal proctitis). 3. Asthma, only if known to be 5 ASA sensitive. 4. Positive stool culture for enteric pathogens (including Salmonella, Shigella, Yersinia, Aeromonas, Plesiomonas, or Campylobacter). Clostridium difficile toxin, ova, or parasites present. 5. Systemic or rectal corticosteroid use within 4 weeks prior to the Screening Visit. Topical, intranasal, or inhaled use is not exclusionary. 6. Immunomodulator (6-mercaptopurine, azathioprine) use within 6 weeks prior to the Screening Visit. 7. History of biologic (eg, anti-tumor necrosis factor agents, integrin receptor antagonists) use at any time. 8. Antibiotic use within 7 days prior to the Screening Visit. 9. Any anti-inflammatory drugs, not including 5-ASA treatment but including non-steroidal anti-inflammatory drugs such as aspirin, COX-2 inhibitors or ibuprofen, within 7 days prior to the Screening Visit unless used at over-the-counter levels for \<3 days. However, prophylactic use of a stable dose of aspirin up to 325mg/day for cardiac disease is permitted. 10. Prebiotic/probiotic use within 7 days prior to the Screening Visit. Yogurt products are permitted.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Clinical Response During Double-Blind Acute Phase at Week 8 | Week 8 | Clinical response was defined as partial ulcerative colitis disease activity index (UC-DAI) score \< or =1 with rectal bleeding = 0, stool frequency \< or =1, and physician's global assessment (PGA = 0). Number of participants with clinical response were reported. |
| Number of Participants With Clinical Response During Double-blind Maintenance Phase at Week 26 | Week 26 | Clinical response was defined as partial UC-DAI \<=1 with (rectal bleeding = 0, stool frequency \< or =1, and PGA = 0). Number of participants who had maintained clinical response were reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Baseline to Week 8 | DUCS score was to measure 7 specific signs or symptom and one impact (abdominal pain, nocturnal stool, daytime stool, blood in stool, diarrhea, urgency, tiredness) of UC with each item score ranged from 0 (worst) to 10 (best) with the overall score ranged from 0 (worst) to 70 (best) based on the responses. Change in the DUCS score from baseline to Week 8 during DBA phase were reported. |
| Number of Participants With Improvement in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-blind Acute Phase at Week 8 | Week 8 | PUCAI was a physician-administered measure that focuses on 6 key signs and symptoms of UC and activity limitations producing a total score ranging from 0-85 with higher scores being worse. Recommended cut-off scores to differentiate disease activity are \< 10 (remission); 11-30 (mild); 31-64 (moderate) and \> 65 (severe). Participants with an improvement (change of greater than or equal to \[\> or =\] 20 points) in PUCAI score. Number of participants with improvement in PUCAI score during Double-blind Acute Phase at Week 8 were reported. |
| Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Central Reading | Week 26 | Clinical and endoscopic response was defined as UC-DAI \< or =2 with rectal bleeding=0, stool frequency \< or =1, PGA=0, and with mucosal healing (endoscopy score \< or =1) based on central reading at Week 26. Number of participants with clinical and endoscopic response during double-blind maintenance phase at Week 26 using central reading were reported. |
| Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Central Reading | Week 8 | Clinical and endoscopic response was defined as UC-DAI \<=2 with rectal bleeding = 0 and stool frequency \<=1, and PGA = 0, and with mucosal healing (endoscopy score \<=1) at least a 1 point reduction in endoscopy score from baseline based on central reading. Participants with missing data at week 8 were assumed not to had a clinical response. Participants who completed week 8 but did not have central reading endoscopies at both baseline and week 8 were excluded. Number of participants with clinical and endoscopic response were reported. |
| Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Baseline, Week 13, and Week 26 | DUCS score was to measure 7 specific signs or symptom and one impact (abdominal pain, nocturnal stool, daytime stool, blood in stool, diarrhea, urgency, tiredness) of UC with each score range from 0 (worst) to 10 (best) with the overall score ranging from 0 (worst) to 70 (best) based on the responses. Change from Baseline in DUCS score during double-blind maintenance phase at week 13 and Week 26 wwere reported. |
| Number of Participants With Remission at Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-Blind Maintenance Phase at Week 26 | Week 26 | PUCAI was a physician-administered measure that focuses on 6 key signs and symptoms of UC and activity limitations producing a total score ranging from 0-85 with higher scores being worse. Recommended cut-off scores to differentiate disease activity are \< 10 (remission); 11-30 (mild); 31-64 (moderate) and \> 65 (severe). Number of participants with remission at PUCAI score during double-blind maintenance phase at week 26 were reported. |
| Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Local Reading | Week 26 | Clinical and endoscopic response was defined as UC-DAI \< or = 2 with rectal bleeding=0, stool frequency \< or = 1, PGA=0, and with mucosal healing (endoscopy score \< or = 1) based on local reading. Number of participants who had maintained clinical and endoscopic response during double-blind maintenance phase at week 26 using local reading were reported. |
| Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Local Reading | Week 8 | Clinical and endoscopic response was defined as UC-DAI \< or =2 with rectal bleeding = 0 and stool frequency \< or =1, and PGA = 0, and with mucosal healing (endoscopy score \< or =1) at least a 1 point reduction in endoscopy score from baseline based on local reading. Participants with missing data at week 8 were assumed not to had a clinical response. Participants who completed week 8 but did not have local reading endoscopies at both baseline and week 8 were excluded. Number of participants with clinical and endoscopic response were reported. |
Countries
Canada, Hungary, Israel, Poland, Slovakia, United Kingdom, United States
Participant flow
Recruitment details
The study was conducted at 50 study centers and 33 sites consented at least 1 participant between 12 December 2014 (first participant first visit) and 28 November 2018 (last participant last visit).
Pre-assignment details
Study was conducted in three phases as double blind acute (DBA), open label acute (OLA), and double blind maintenance (DBM) phase. Overall, 107 participants were enrolled and entered into DBA or DBM directly and eligible participants entered into DBM phase after DBA or OLA through DBA. Total, 105 participants received treatment and 65 completed.
Participants by arm
| Arm | Count |
|---|---|
| Overall Study Participants with partial UC-DAI \> or =2 and mucosal appearance = 2 or 3 entered the DBA phase; with UC-DAI between 1 and 2 entered the OLA phase, after completing DBA phase; with UC-DAI \< or = 1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants who had a clinical response (partial UC-DAI \< or =1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBA and DBM phase, participants received 900 to 2400 mg/day (low dose) and 1800 to 4800 mg/day (high dose) of MMX mesalamine/mesalazine tablet orally once daily for 8 and 26 weeks respectively. During OLA phase, participants received the high dose for 8 weeks. | 105 |
| Total | 105 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 |
|---|---|---|---|---|---|---|
| Double Blind Acute Phase (8 Weeks) | Adverse Event | 1 | 0 | 0 | 0 | 0 |
| Double Blind Acute Phase (8 Weeks) | Lack of Efficacy | 5 | 0 | 0 | 0 | 0 |
| Double Blind Acute Phase (8 Weeks) | Subjects who Continued in the Study | 2 | 4 | 0 | 0 | 0 |
| Double Blind Maintenance Phase(26 Weeks) | Adverse Event | 0 | 0 | 0 | 3 | 2 |
| Double Blind Maintenance Phase(26 Weeks) | Lack of Efficacy | 0 | 0 | 0 | 6 | 7 |
| Double Blind Maintenance Phase(26 Weeks) | Missing | 0 | 0 | 0 | 0 | 1 |
| Double Blind Maintenance Phase(26 Weeks) | Other (unspecified) | 0 | 0 | 0 | 1 | 1 |
| Open Label Acute Phase (8 Weeks) | Adverse Event | 0 | 0 | 1 | 0 | 0 |
| Open Label Acute Phase (8 Weeks) | Lack of Efficacy | 0 | 0 | 5 | 0 | 0 |
Baseline characteristics
| Characteristic | Overall Study |
|---|---|
| Age, Continuous | 14.1 years STANDARD_DEVIATION 2.55 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 104 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 3 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 101 Participants |
| Sex: Female, Male Female | 53 Participants |
| Sex: Female, Male Male | 52 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk |
|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 27 | 0 / 26 | 0 / 18 | 0 / 42 | 0 / 45 |
| other Total, other adverse events | 15 / 27 | 15 / 26 | 12 / 18 | 25 / 42 | 25 / 45 |
| serious Total, serious adverse events | 4 / 27 | 0 / 26 | 3 / 18 | 3 / 42 | 2 / 45 |
Outcome results
Primary
Number of Participants With Clinical Response During Double-Blind Acute Phase at Week 8
Clinical response was defined as partial ulcerative colitis disease activity index (UC-DAI) score \< or =1 with rectal bleeding = 0, stool frequency \< or =1, and physician's global assessment (PGA = 0). Number of participants with clinical response were reported.
Time frame: Week 8
Population: Double-blind acute phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind acute phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Clinical Response During Double-Blind Acute Phase at Week 8 | 10 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Clinical Response During Double-Blind Acute Phase at Week 8 | 17 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (last observation carried forward \[LOCF\] and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.03995% CI: [1.04, 9.88]Uncorrected Chi-squared Test
Primary
Number of Participants With Clinical Response During Double-blind Maintenance Phase at Week 26
Clinical response was defined as partial UC-DAI \<=1 with (rectal bleeding = 0, stool frequency \< or =1, and PGA = 0). Number of participants who had maintained clinical response were reported.
Time frame: Week 26
Population: Double-blind maintenance phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind maintenance phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Clinical Response During Double-blind Maintenance Phase at Week 26 | 23 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Clinical Response During Double-blind Maintenance Phase at Week 26 | 24 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-value s were presented as descriptive statistics.p-value: 0.98195% CI: [0.42, 2.34]Cochran-Mantel-Haenszel
Secondary
Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase
DUCS score was to measure 7 specific signs or symptom and one impact (abdominal pain, nocturnal stool, daytime stool, blood in stool, diarrhea, urgency, tiredness) of UC with each item score ranged from 0 (worst) to 10 (best) with the overall score ranged from 0 (worst) to 70 (best) based on the responses. Change in the DUCS score from baseline to Week 8 during DBA phase were reported.
Time frame: Baseline to Week 8
Population: Double-blind acute phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind acute phase. Here, number of participants analyzed signifies participants who were evaluable for this measure category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Baseline | 32.2 Score on the scale | Standard Error 2.86 |
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Week 2 | -14.1 Score on the scale | Standard Error 3.8 |
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Week 4 | -15.6 Score on the scale | Standard Error 3.96 |
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Week 8 | -18.2 Score on the scale | Standard Error 4.4 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Week 8 | -23.1 Score on the scale | Standard Error 3.59 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Baseline | 31.6 Score on the scale | Standard Error 2.88 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Week 4 | -16.7 Score on the scale | Standard Error 4.01 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Acute Phase | Week 2 | -13.2 Score on the scale | Standard Error 3.5 |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.16895% CI: [-13.1, 2.4]ANCOVA
Secondary
Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase
DUCS score was to measure 7 specific signs or symptom and one impact (abdominal pain, nocturnal stool, daytime stool, blood in stool, diarrhea, urgency, tiredness) of UC with each score range from 0 (worst) to 10 (best) with the overall score ranging from 0 (worst) to 70 (best) based on the responses. Change from Baseline in DUCS score during double-blind maintenance phase at week 13 and Week 26 wwere reported.
Time frame: Baseline, Week 13, and Week 26
Population: Double-blind maintenance phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind maintenance phase. Here, number of participants analyzed signifies participants who were evaluable for this measure category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Baseline | 5.8 Score on the scale | Standard Error 1.37 |
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Week 13 | 1.7 Score on the scale | Standard Error 1.61 |
| Double-Blind Acute (DBA) Phase: Low Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Week 26 | 1.3 Score on the scale | Standard Error 1.19 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Baseline | 4.6 Score on the scale | Standard Error 0.79 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Week 13 | -0.1 Score on the scale | Standard Error 0.89 |
| Double-Blind Acute (DBA) Phase: High Dose | Change From Baseline in Daily Ulcerative Colitis Scale (DUCS) Score During Double-Blind Maintenance Phase | Week 26 | 4.4 Score on the scale | Standard Error 1.79 |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.18295% CI: [-1.4, 7.4]ANCOVA
Secondary
Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Central Reading
Clinical and endoscopic response was defined as UC-DAI \<=2 with rectal bleeding = 0 and stool frequency \<=1, and PGA = 0, and with mucosal healing (endoscopy score \<=1) at least a 1 point reduction in endoscopy score from baseline based on central reading. Participants with missing data at week 8 were assumed not to had a clinical response. Participants who completed week 8 but did not have central reading endoscopies at both baseline and week 8 were excluded. Number of participants with clinical and endoscopic response were reported.
Time frame: Week 8
Population: Double-blind acute phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind acute phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Central Reading | 1 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Central Reading | 3 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.495% CI: [-19.3, 100]Fisher Exact
Secondary
Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Local Reading
Clinical and endoscopic response was defined as UC-DAI \< or =2 with rectal bleeding = 0 and stool frequency \< or =1, and PGA = 0, and with mucosal healing (endoscopy score \< or =1) at least a 1 point reduction in endoscopy score from baseline based on local reading. Participants with missing data at week 8 were assumed not to had a clinical response. Participants who completed week 8 but did not have local reading endoscopies at both baseline and week 8 were excluded. Number of participants with clinical and endoscopic response were reported.
Time frame: Week 8
Population: Double-blind acute phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind acute phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Local Reading | 1 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Clinical and Endoscopic Response During Double Blind Acute Phase at Week 8 Using Local Reading | 4 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate.p-value: 0.33395% CI: [-19.3, 100]Fisher Exact
Secondary
Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Central Reading
Clinical and endoscopic response was defined as UC-DAI \< or =2 with rectal bleeding=0, stool frequency \< or =1, PGA=0, and with mucosal healing (endoscopy score \< or =1) based on central reading at Week 26. Number of participants with clinical and endoscopic response during double-blind maintenance phase at Week 26 using central reading were reported.
Time frame: Week 26
Population: Double-blind maintenance phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind maintenance phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Central Reading | 13 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Central Reading | 11 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.53995% CI: [-25.3, 12.3]Cochran-Mantel-Haenszel
Secondary
Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Local Reading
Clinical and endoscopic response was defined as UC-DAI \< or = 2 with rectal bleeding=0, stool frequency \< or = 1, PGA=0, and with mucosal healing (endoscopy score \< or = 1) based on local reading. Number of participants who had maintained clinical and endoscopic response during double-blind maintenance phase at week 26 using local reading were reported.
Time frame: Week 26
Population: Double-blind maintenance phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind maintenance phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Local Reading | 18 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Clinical and Endoscopic Response During Double-Blind Maintenance Phase at Week 26 Using Local Reading | 12 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.12995% CI: [-36, 3.6]Cochran-Mantel-Haenszel
Secondary
Number of Participants With Improvement in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-blind Acute Phase at Week 8
PUCAI was a physician-administered measure that focuses on 6 key signs and symptoms of UC and activity limitations producing a total score ranging from 0-85 with higher scores being worse. Recommended cut-off scores to differentiate disease activity are \< 10 (remission); 11-30 (mild); 31-64 (moderate) and \> 65 (severe). Participants with an improvement (change of greater than or equal to \[\> or =\] 20 points) in PUCAI score. Number of participants with improvement in PUCAI score during Double-blind Acute Phase at Week 8 were reported.
Time frame: Week 8
Population: Double-blind acute phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind acute phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Improvement in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-blind Acute Phase at Week 8 | 10 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Improvement in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-blind Acute Phase at Week 8 | 16 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.13195% CI: [-1.6, 50.6]Chi-squared, Corrected
Secondary
Number of Participants With Remission at Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-Blind Maintenance Phase at Week 26
PUCAI was a physician-administered measure that focuses on 6 key signs and symptoms of UC and activity limitations producing a total score ranging from 0-85 with higher scores being worse. Recommended cut-off scores to differentiate disease activity are \< 10 (remission); 11-30 (mild); 31-64 (moderate) and \> 65 (severe). Number of participants with remission at PUCAI score during double-blind maintenance phase at week 26 were reported.
Time frame: Week 26
Population: Double-blind maintenance phase safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product during the double-blind maintenance phase.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Double-Blind Acute (DBA) Phase: Low Dose | Number of Participants With Remission at Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-Blind Maintenance Phase at Week 26 | 29 Participants |
| Double-Blind Acute (DBA) Phase: High Dose | Number of Participants With Remission at Pediatric Ulcerative Colitis Activity Index (PUCAI) Score During Double-Blind Maintenance Phase at Week 26 | 27 Participants |
Comparison: This study is not powered to detect differences between treatment groups and other sensitivity analysis (LOCF and complete case) intended to examine the robustness of the treatment estimate. This was an estimation study, and p-values were presented as descriptive statistics.p-value: 0.19495% CI: [-29.1, 11]Cochran-Mantel-Haenszel