Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2013-001744-65 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MMX Mesalamine/Mesalazine (Low Dose) | Experimental | 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. |
|
| MMX Mesalamine/Mesalazine (High Dose) | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MMX Mesalamine/Mesalazine (Low Dose) | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Week 8 |
| 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. | Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Not provided
Inclusion Criteria:
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.
Subject's parent/LAR demonstrates an understanding, ability, and willingness to fully comply with study procedures and restrictions.
Male and female children and adolescents aged 5-17 years, inclusive.
Body weight 18-90kg.
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.
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.
Subject is able to swallow the investigational product whole.
Double-blind Acute Phase:
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.
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:
Partial UC-DAI ≤1 (rectal bleeding=0, stool frequency ≤1, and PGA=0) at the Baseline Visit.
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Shire Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Maryland Children's Hospital | Baltimore | Maryland | 21201 | United States | ||
| John Hopkins |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37855022 | Derived | Croft NM, Korczowski B, Kierkus J, Caballero B, Thakur MK. Safety and efficacy of multimatrix mesalamine in paediatric patients with mild-to-moderate ulcerative colitis: a phase 3, randomised, double-blind study. EClinicalMedicine. 2023 Oct 6;65:102232. doi: 10.1016/j.eclinm.2023.102232. eCollection 2023 Nov. |
Not provided
Not provided
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Not provided
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
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.
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).
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Double-Blind Acute (DBA) Phase: Low Dose | Participants with partial UC-DAI > or =2 and mucosal appearance = 2 or 3 entered the DBA phase. During the DBA low dose phase participants weighing 18 to < or =23 kg, >23 to < or =35 kg, > 35 to < or =50 kg, > 50 to < or =90 kg received 900, 1200, 1800, and 2400 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 8 weeks respectively. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double Blind Acute Phase (8 Weeks) |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 10, 2017 | Oct 15, 2019 |
Not provided
Not provided
Not provided
Not provided
|
|
| MMX Mesalamine/Mesalazine (High Dose) | Drug | 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. |
|
|
| Week 8 |
| 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. | Week 8 |
| 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. | Baseline to Week 8 |
| 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. | Week 8 |
| 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. | Week 26 |
| 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. | Week 26 |
| 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. | Baseline, Week 13, and Week 26 |
| 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. | Week 26 |
| Baltimore |
| Maryland |
| 21287 |
| United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Newton Wellesley Hospital | Newton | Massachusetts | 02462 | United States |
| University of Minnesota Children's Hospital | Minneapolis | Minnesota | 55454 | United States |
| Mayo Clinic Gastroenterology | Rochester | Minnesota | 55905 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Texas Digestive Disease Consultants | Southlake | Texas | 76092 | United States |
| Carilion Medical Center | Roanoke | Virginia | 24013 | United States |
| University of Alberta Pediatric Gastroenterology & Nutrition | Edmonton | Alberta | T6G 1C9 | Canada |
| Szent Janos Korhaz És Észak-budai Egyesitett Korha | Budapest | H-1023 | Hungary |
| Bekes Megyei Pandy Kalman Korhaz | Gyula | H-5700 | Hungary |
| Baz Megyei Korhaz Es Egyetemi Oktatokorhaz | Miskolc | 3526 | Hungary |
| Szabolcs-Szatmar-Bereg Megyei Korhazak es Egyetemi Oktato Korhaz | Nyíregyháza | 4400 | Hungary |
| Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont | Szeged | H-6720 | Hungary |
| Soroka Medical Center | Beersheba | 85025 | Israel |
| Rambam Health Corporation | Haifa | 31096 | Israel |
| Shaare Zedek Medical Center | Jerusalem | 91031 | Israel |
| Schneider Medical Centre | Petah Tikva | 49202 | Israel |
| Uniwersytecki Dzieciecy Szpital Kliniczny im. Ludwika Zamenhofa | Bialystok | 15-247 | Poland |
| Klinika Pediatrii Gastroenterologii I Zywienia | Krakow | 30-663 | Poland |
| Klinika Gastroenterologii I Pediatrii | Lodz | 93-338 | Poland |
| Wojewodzki Specjalistyczny Szpital Dzieciecy | Olsztyn | 10-561 | Poland |
| Gabinet Lekarski-Bartosz Korczowski | Rzeszów | 35-302 | Poland |
| Oddzial Gastroenterologii I Hepatologii | Warsaw | 04-730 | Poland |
| Uniwersytecki Szpital Kliniczny We Wrocławiu | Wroclaw | 50-369 | Poland |
| Detská fakultná nemocnica s poliklinikou | Banská Bystrica | 974 09 | Slovakia |
| University Children's Hospital | Bratislava | 833 40 | Slovakia |
| Univerzitna Nemocnica Martin | Martin | 03659 | Slovakia |
| Alder Hey Children's Hospital | Liverpool | LI2 2AP | United Kingdom |
| Barts Health NHS Trust, Royal London Hospital | London | E1 1BB | United Kingdom |
| King's College Hospital | London | SE5 9RS | United Kingdom |
| Great Ormond Street Hospital | London | WC1N 3JH | United Kingdom |
| FG001 | Double-Blind Acute (DBA) Phase: High Dose | Participants with partial UC-DAI > or =2 and mucosal appearance = 2 or 3 entered the DBA phase. During the DBA high dose phase participants weighing 18 to < or =23 kg, > 23 to < or =35 kg, > 35 to < or =50 kg, > 50 to < or =90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 8 weeks respectively. |
| FG002 | Open-Label Acute Phase (OLA): High Dose | During the OLA High dose phase participants weighing 18 to <=23 kg, > 23 to < or =35 kg, >35 to < or = 50 kg, > 50 to < or = 90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 8 weeks respectively. |
| FG003 | Double-Blind Maintenance (DBM) Phase: Low Dose | Participants with partial UC-DAI < or =1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants with a clinical response (partial UC-DAI < or =1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBM low dose phase participants weighing 18 to < or =23 kg, > 23 to < or =35 kg, > 35 to < or =50 kg, > 50 to < or =90 kg received 900, 1200, 1800, and 2400 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 26 weeks respectively. |
| FG004 | Double-Blind Maintenance (DBM) Phase: High Dose | Participants with partial UC-DAI < or =1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants with a clinical response (partial UC-DAI < or =1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBM high dose phase participants weighing 18 to <=23 kg, > 23 to < or =35 kg, >35 to < or = 50 kg, > 50 to < or = 90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 26 weeks respectively. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open Label Acute Phase (8 Weeks) |
|
|
| Double Blind Maintenance Phase(26 Weeks) |
|
|
Safety analysis set consisted of randomized participants who had taken at least 1 dose of investigational product. Few participants had multiple movement within the arms of the study at different levels and unique participants baseline was not analysed, only overall participant data was planned, analysed and reported to avoid double-counting.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | 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. | Posted | Count of Participants | Participants | Week 8 |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 26 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 8 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 8 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Mean | Standard Error | Score on the scale | Baseline to Week 8 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 8 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 26 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 26 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Mean | Standard Error | Score on the scale | Baseline, Week 13, and Week 26 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Count of Participants | Participants | Week 26 |
|
From start of study drug administration up to follow-up (up to Week 27)
Overall safety analysis set consisted of randomized participants who had taken at least 1 dose of IP.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Double-Blind Acute (DBA) Phase: Low Dose | Participants with partial UC-DAI >=2 and mucosal appearance = 2 or 3 entered the DBA phase. During the DBA low dose phase participants weighing 18 to <=23 kg, >23 to <=35 kg, >35 to <=50 kg, >50 to <=90 kg received 900, 1200, 1800, and 2400 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 8 weeks respectively. | 0 | 27 | 4 | 27 | 15 | 27 |
| EG001 | Double-Blind Acute (DBA) Phase: High Dose | Participants with partial UC-DAI >=2 and mucosal appearance = 2 or 3 entered the DBA phase. During the DBA high dose phase participants weighing 18 to <=23 kg, >23 to <=35 kg, >35 to <=50 kg, >50 to <=90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 8 weeks respectively. | 0 | 26 | 0 | 26 | 15 | 26 |
| EG002 | Open-Label Acute (OLA) Phase: High Dose | Participants with partial UC-DAI between 1 and 2 entered the OLA phase, after completing DBA phase. Participants weighing 18 to <=23 kg, >23 to <=35 kg, >35 to <=50 kg, >50 to <=90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 8 weeks respectively during the high dose OLA phase. | 0 | 18 | 3 | 18 | 12 | 18 |
| EG003 | Double-Blind Maintenance (DBM) Phase: Low Dose | Participants with partial UC-DAI <=1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants with a clinical response (partial UC-DAI <=1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBM low dose phase participants weighing 18 to <=23 kg, >23 to <=35 kg, >35 to <=50 kg, >50 to <=90 kg received 900, 1200, 1800, and 2400 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 26 weeks respectively. | 0 | 42 | 3 | 42 | 25 | 42 |
| EG004 | Double-Blind Maintenance (DBM) Phase: High Dose | Participants with partial UC-DAI <=1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants with a clinical response (partial UC-DAI <=1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBM high dose phase participants weighing 18 to <=3 kg, >23 to <=35 kg, >35 to <=50 kg, >50 to <=90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 26 weeks respectively. | 0 | 45 | 2 | 45 | 25 | 45 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Colitis ulcerative | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Injury corneal | Injury, poisoning and procedural complications | MedDRA 16.1 | Non-systematic Assessment |
| |
| Retinal injury | Injury, poisoning and procedural complications | MedDRA 16.1 | Non-systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 16.1 | Non-systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 16.1 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 16.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Torticollis | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Colitis ulcerative | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 16.1 | Non-systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA 16.1 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Non-systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Non-systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 31, 2018 | Oct 15, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003093 | Colitis, Ulcerative |
| D008224 | Lymphoma, Follicular |
| ID | Term |
|---|---|
| D003092 | Colitis |
| D005759 | Gastroenteritis |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D015212 | Inflammatory Bowel Diseases |
| D003108 | Colonic Diseases |
| D007410 | Intestinal Diseases |
| D008228 | Lymphoma, Non-Hodgkin |
| D008223 | Lymphoma |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D019804 | Mesalamine |
| ID | Term |
|---|---|
| D062368 | meta-Aminobenzoates |
| D062365 | Aminobenzoates |
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D000636 | Aminosalicylic Acids |
| D012459 | Salicylates |
| D062385 | Hydroxybenzoates |
| D006880 | Hydroxy Acids |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D010636 | Phenols |
Not provided
Not provided
| Lack of Efficacy |
|
| Lack of Efficacy |
|
| Other (unspecified) |
|
| Missing |
|
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Participants with partial UC-DAI < or =1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants with a clinical response (partial UC-DAI < or =1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBM high dose phase participants weighing 18 to < or =23 kg, > 23 to < or =35 kg, > 35 to < or =50 kg, > 50 to < or =90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 26 weeks respectively. |
|
|
|
Participants with partial UC-DAI < or =1 and mucosal appearance = 0 or 1 entered the DBM phase directly. Also, participants with a clinical response (partial UC-DAI < or =1) after completion of DBA phase or OLA phase entered into the DBM phase. During the DBM high dose phase participants weighing 18 to <=23 kg, >23 to <=35 kg, >35 to < or =50 kg, > 50 to < or =90 kg received 1800, 2400, 3600, and 4800 mg/day of MMX mesalamine/mesalazine tablet orally once daily for 26 weeks respectively.
|
|
|