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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04779307




Registration number
NCT04779307
Ethics application status
Date submitted
1/03/2021
Date registered
3/03/2021

Titles & IDs
Public title
A Study of Vedolizumab in Children and Teenagers with Moderate to Severe Ulcerative Colitis (UC)
Scientific title
A Randomized, Double-Blind, Phase 3 Study to Evaluate the Efficacy and Safety of Vedolizumab Intravenous As Maintenance Therapy in Pediatric Subjects with Moderately to Severely Active Ulcerative Colitis Who Achieved Clinical Response Following Open-Label Vedolizumab Intravenous Therapy
Secondary ID [1] 0 0
2020-004300-34
Secondary ID [2] 0 0
MLN0002-3024
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Colitis, Ulcerative 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 0 0 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vedolizumab

Experimental: Induction Period: Participants =30 kg, Vedolizumab 300 mg - Vedolizumab 300 mg, intravenous (IV) infusion, at Day 1, Weeks 2 and 6 in the Induction Period. Participants with UC having Baseline weight of =30 kg are included in this arm.

Experimental: Induction Period: Participants >15 to <30 kg, Vedolizumab 200 mg - Vedolizumab 200 mg, IV infusion, at Day 1, Weeks 2 and 6 in the Induction Period. Participants with UC having Baseline weight of \>15 to \<30 kg are included in this arm.

Experimental: Induction Period: Participants 10 to 15 kg, Vedolizumab 150 mg - Vedolizumab 150 mg, IV infusion, at Day 1, Weeks 2 and 6 in the Induction Period. Participants with UC having Baseline weight of 10 to 15 kg are included in this arm.

Experimental: Maintenance Period: Participants =30 kg, Vedolizumab 300 mg - Vedolizumab 300 mg, IV infusion, once every 8 weeks (Q8W) from Week 14 up to Week 46 in the Maintenance Period. Participants with Week 14 weight of =30 kg who achieved clinical response at Week 14 randomized to this high dose arm group will receive vedolizumab 300 mg.

Experimental: Maintenance Period: Participants =30 kg, Vedolizumab 150 mg - Vedolizumab 150 mg, IV infusion, Q8W from Week 14 up to Week 46 in the Maintenance Period. Participants with Week 14 weight of =30 kg who achieved clinical response at Week 14 randomized to this low dose arm group will receive vedolizumab 150 mg.

Experimental: Maintenance Period: Participants >15 to <30 kg, Vedolizumab 200 mg - Vedolizumab 200 mg, IV infusion, Q8W from Week 14 up to Week 46 in the Maintenance Period. Participants with Week 14 weight of \>15 to \<30 kg who achieved clinical response at Week 14 randomized to this high dose arm group will receive vedolizumab 200 mg.

Experimental: Maintenance Period: Participants >15 to <30 kg, Vedolizumab 100 mg - Vedolizumab 100 mg, IV infusion, Q8W from Week 14 up to Week 46 in the Maintenance Period. Participants with Week 14 weight of \>15 to \<30 kg who achieved clinical response at Week 14 randomized to this low dose arm group will receive vedolizumab 100 mg.

Experimental: Maintenance Period: Participants 10 to 15 kg, Vedolizumab 150 mg - Vedolizumab 150 mg, IV infusion, Q8W from Week 14 up to Week 46 in the Maintenance Period. Participants with Week 14 weight of 10 to 15 kg who achieved clinical response at Week 14 randomized to this high dose arm group will receive vedolizumab 150 mg.

Experimental: Maintenance Period: Participants 10 to 15 kg, Vedolizumab 100 mg - Vedolizumab 100 mg, IV infusion, Q8W from Week 14 up to Week 46 in the Maintenance Period. Participants with Week 14 weight of 10 to 15 kg who achieved clinical response at Week 14 randomized to this low dose arm group will receive vedolizumab 100 mg.


Treatment: Drugs: Vedolizumab
Vedolizumab IV infusion.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants with Clinical Remission at Week 54 Based on Modified Mayo Score
Timepoint [1] 0 0
Week 54
Secondary outcome [1] 0 0
Percentage of Participants with Clinical Remission at Week 14 Based on Modified Mayo Score
Timepoint [1] 0 0
Week 14
Secondary outcome [2] 0 0
Percentage of Participants with Sustained Clinical Remission at Week 14 Based on Modified Mayo Score
Timepoint [2] 0 0
Week 14
Secondary outcome [3] 0 0
Percentage of Participants with Sustained Clinical Remission at Week 54 Based on Modified Mayo Score
Timepoint [3] 0 0
Week 54
Secondary outcome [4] 0 0
Percentage of Participants with Sustained Endoscopic Remission at Week 14
Timepoint [4] 0 0
Week 14
Secondary outcome [5] 0 0
Percentage of Participants with Sustained Endoscopic Remission at Week 54
Timepoint [5] 0 0
Week 54
Secondary outcome [6] 0 0
Percentage of Participants with Endoscopic Response at Week 14
Timepoint [6] 0 0
Week 14
Secondary outcome [7] 0 0
Percentage of Participants with Endoscopic Response at Week 54
Timepoint [7] 0 0
Week 54
Secondary outcome [8] 0 0
Percentage of Participants with Corticosteroid-free Clinical Remission at Week 54
Timepoint [8] 0 0
Week 54
Secondary outcome [9] 0 0
Percentage of Participants with Clinical Remission at Week 54 Based on Complete Mayo Score
Timepoint [9] 0 0
Week 54
Secondary outcome [10] 0 0
Serum Trough Concentrations of Vedolizumab over Time
Timepoint [10] 0 0
Predose and postdose at multiple time points (up to 54 weeks)
Secondary outcome [11] 0 0
Percentage of Participants with Positive Anti-vedolizumab Antibodies (AVA)
Timepoint [11] 0 0
Predose (up to 54 weeks)
Secondary outcome [12] 0 0
Percentage of Participants with Positive Neutralizing AVA
Timepoint [12] 0 0
Predose (up to 54 weeks)
Secondary outcome [13] 0 0
Percentage of Participants with Sustained Clinical Response at Week 14 Based on Complete Mayo Score
Timepoint [13] 0 0
Week 14
Secondary outcome [14] 0 0
Percentage of Participants with Sustained Clinical Response at Week 54 Based on Complete Mayo Score
Timepoint [14] 0 0
Week 54
Secondary outcome [15] 0 0
Percentage of Participants with Clinical Response up to Week 54 Based on Partial Mayo Score
Timepoint [15] 0 0
Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Secondary outcome [16] 0 0
Percentage of Participants with Clinical Remission up to Week 54 Based on Partial Mayo Score
Timepoint [16] 0 0
Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Secondary outcome [17] 0 0
Percentage of Participants with at least One Adverse Event (AE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Timepoint [17] 0 0
Up to 158 weeks
Secondary outcome [18] 0 0
Change from Baseline in Weight
Timepoint [18] 0 0
Baseline, up to Week 54
Secondary outcome [19] 0 0
Change from Baseline in Linear Growth Z-score
Timepoint [19] 0 0
Baseline, up to Week 54
Secondary outcome [20] 0 0
Change in Tanner Stage at Week 54 Compared with Baseline
Timepoint [20] 0 0
Week 54

Eligibility
Key inclusion criteria
1. Has moderately to severely active UC, unresponsive or intolerant to their current standard of care (SOC).
2. Weighs =10 kg at the time of screening and enrollment into the study.
3. Participants with UC diagnosed at least 1 month before screening. Participants with moderately to severely active UC based on a modified Mayo score of 5 to 9 (sum of Mayo endoscopic subscore, stool frequency subscore, and rectal bleeding subscore) with a Mayo endoscopic subscore of =2 (with the presence of mucosal friability excluding an endoscopic subscore of 1 and mandating a score of at least 2) at screening endoscopy.
4. Has failed, lost response to, or been intolerant to treatment with at least 1 of the following agents: corticosteroids (eg, azathioprine [AZA], 6-mercaptopurine [6-MP], methotrexate [MTX]), immunomodulators, and/or tumor necrosis factor alpha (TNF-a) antagonist therapy (eg, infliximab, adalimumab). This includes participants who are dependent on corticosteroids to control symptoms and who are experiencing worsening of disease in the moderate-to-severe range when attempting to wean off corticosteroids.
5. Has evidence of UC extending proximal to the rectum (i.e., not limited to proctitis), at a minimum.
6. Has extensive colitis or pancolitis of >8 years' duration or left-sided colitis of >12 years' duration must have documented evidence of a negative surveillance colonoscopy within 12 months before screening.
7. Participants with vaccinations that are up-to-date based on the countrywide, accepted schedule of childhood vaccines.
Minimum age
2 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Has previous exposure to approved or investigational anti-integrins including, but not limited to natalizumab, efalizumab, etrolizumab, or Abrilumab (AMG 181), or mucosal addressin cell adhesion molecule-1 (MAdCAM-1) antagonists or rituximab.
2. Has received an investigational biologic within 60 days or 5 half-lives before screening (whichever is longer); or an approved biologic or biosimilar agent within 2 weeks before the first dose of study drug or at any time during the screening period.
3. Has active cerebral/meningeal disease, signs/symptoms or history of progressive multifocal leukoencephalopathy (PML) or any other major neurological disorders including stroke, multiple sclerosis, brain tumor or neurodegenerative disease.
4. Has had clinically significant infection (eg, pneumonia, pyelonephritis, coronavirus disease 2019 [COVID-19]) within 30 days prior to first dose of study drug.
5. Has received any live vaccinations within 30 days prior to first dose of study drug.
6. Participants who currently require surgical intervention or are anticipated to require surgical intervention for UC during this study.
7. Has had subtotal or total colectomy or have a jejunostomy, ileostomy, colostomy, ileo-anal pouch, or known fixed stenosis of the intestine.
8. Participants with a current diagnosis of indeterminate colitis.
9. Participants with clinical features suggesting monogenic very early onset inflammatory bowel disease.
10. Participant with active or latent tuberculosis (TB), as evidenced by a diagnostic TB test performed within 30 days of screening or during the screening period that is positive, defined as:

* Positive QuantiFERON test or 2 successive indeterminate QuantiFERON tests, OR
* A TB skin test reaction =5 mm. NOTE: If participants have received Bacillus Calmette-Guérin vaccine then a QuantiFERON TB Gold test should be performed instead of the TB skin test.
11. Participants with evidence of positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). Hepatitis B virus (HBV) immune participants (ie, hepatitis B surface antigen [HBsAg]-negative and hepatitis B antibody-positive) may, however, be included. Note: If a participant tests negative for HBsAg, but positive for HBcAb, the participant would be considered eligible if the absence of HBV DNA is confirmed by HBV DNA polymerase chain reaction reflex testing performed in the central laboratory.

Participants with chronic hepatitis C virus (HCV) (ie, positive HCV antibody [HCVAb] and HCV RNA). Note: Subjects who are HCVAb-positive without evidence of HCV RNA may be considered eligible (spontaneous viral clearance or previously treated and cured [defined as no evidence of HCV RNA at least 12 weeks before baseline]).
12. The participant has evidence of dysplasia or history of malignancy other than a successfully treated nonmetastatic cutaneous squamous cell or basal cell carcinoma or localized carcinoma in situ of the cervix.
13. Has positive stool studies for ova and/or parasites or stool culture at screening visit.
14. Has positive Clostridioides difficile (C difficile) stool test at screening visit.

Other inclusion/exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/04/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/08/2025
Actual
Sample size
Target
Accrual to date
Final
121
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Queensland Childrens Hospital - South Brisbane
Recruitment hospital [3] 0 0
Monash Health, Monash Medical Centre - Clayton
Recruitment hospital [4] 0 0
Royal Children's Hospital Melbourne - PIN - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
New Jersey
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Virginia
Country [14] 0 0
Belgium
State/province [14] 0 0
Antwerpen
Country [15] 0 0
Belgium
State/province [15] 0 0
Brussels
Country [16] 0 0
Belgium
State/province [16] 0 0
Vlaams Brabant
Country [17] 0 0
Canada
State/province [17] 0 0
Alberta
Country [18] 0 0
Canada
State/province [18] 0 0
British Columbia
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
China
State/province [20] 0 0
Beijing
Country [21] 0 0
China
State/province [21] 0 0
Henan
Country [22] 0 0
China
State/province [22] 0 0
Shanghai
Country [23] 0 0
China
State/province [23] 0 0
Zhejiang
Country [24] 0 0
Croatia
State/province [24] 0 0
Grad Zagreb
Country [25] 0 0
Croatia
State/province [25] 0 0
Split
Country [26] 0 0
Czechia
State/province [26] 0 0
Praha
Country [27] 0 0
Greece
State/province [27] 0 0
Attiki
Country [28] 0 0
Greece
State/province [28] 0 0
Thessaloniki
Country [29] 0 0
Hungary
State/province [29] 0 0
Budapest
Country [30] 0 0
Hungary
State/province [30] 0 0
Miskolc
Country [31] 0 0
Israel
State/province [31] 0 0
HaDarom
Country [32] 0 0
Israel
State/province [32] 0 0
Yerushalayim
Country [33] 0 0
Israel
State/province [33] 0 0
Haifa
Country [34] 0 0
Israel
State/province [34] 0 0
Petah Tikva
Country [35] 0 0
Israel
State/province [35] 0 0
Tel Aviv
Country [36] 0 0
Italy
State/province [36] 0 0
Campania
Country [37] 0 0
Italy
State/province [37] 0 0
Emilia-Romagna
Country [38] 0 0
Italy
State/province [38] 0 0
Lazio
Country [39] 0 0
Italy
State/province [39] 0 0
Monza E Brianza
Country [40] 0 0
Italy
State/province [40] 0 0
Toscana
Country [41] 0 0
Italy
State/province [41] 0 0
Veneto
Country [42] 0 0
Italy
State/province [42] 0 0
Napoli
Country [43] 0 0
Japan
State/province [43] 0 0
Hukuoka
Country [44] 0 0
Japan
State/province [44] 0 0
Tokyo
Country [45] 0 0
Japan
State/province [45] 0 0
Kumamoto
Country [46] 0 0
Japan
State/province [46] 0 0
Saitama
Country [47] 0 0
Korea, Republic of
State/province [47] 0 0
Daegu Gwang'yeogsi
Country [48] 0 0
Korea, Republic of
State/province [48] 0 0
Incheon Gwang'yeogsi
Country [49] 0 0
Korea, Republic of
State/province [49] 0 0
Seoul Teugbyeolsi
Country [50] 0 0
Poland
State/province [50] 0 0
Lodzkie
Country [51] 0 0
Poland
State/province [51] 0 0
Malopolskie
Country [52] 0 0
Poland
State/province [52] 0 0
Mazowieckie
Country [53] 0 0
Poland
State/province [53] 0 0
Podkarpackie
Country [54] 0 0
Poland
State/province [54] 0 0
Slaskie
Country [55] 0 0
Poland
State/province [55] 0 0
Zachodniopomorskie
Country [56] 0 0
United Kingdom
State/province [56] 0 0
London, City Of
Country [57] 0 0
United Kingdom
State/province [57] 0 0
West Midlands
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Cardiff

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Takeda
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Takeda Contact
Address 0 0
Country 0 0
Phone 0 0
+1-877-825-3327
Fax 0 0
Email 0 0
medinfoUS@takeda.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04779307