A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Ulcerative Colitis (UC)

NCT04779307 | Completed Phase 3 Results DMC FDA Drug

• 4 other identifiers: MLN0002-30242020-004300-34jRCT20712100302023-509018-12-00
• Study Type: interventional
• Target: 121
• Locations: 14 countries
• Sites: 65

Brief Summary

Vedolizumab is a medicine that helps to reduce inflammation and pain in the digestive system. In this study, children and teenagers with moderate to severe ulcerative colitis will be treated with vedolizumab. The main aim of the study is to check if participants achieve remission after treatment with vedolizumab. Remission means symptoms improve or disappear and an endoscopy shows no or limited signs of disease. The study is also evaluating side effects of vedolizumab in the children and teenager with moderately to severely active ulcerative colitis. Participants will receive 3 infusions of vedolizumab over 6 weeks. Then, those who have a clinical response will receive 1 of 3 doses of vedolizumab once every 8 weeks. They will receive the same dose every time.

Conditions

Colitis, Ulcerative

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Clinical Remission at Week 54 Based on Modified Mayo Score

  Clinical remission based on the modified Mayo score was defined as stool frequency sub score 0 to 1 and a decrease of 1 or more from baseline, rectal bleeding sub score of 0, and endoscopy sub score 0 to 1 (modified so that a score of 1 does not include friability) and without presence of any intercurrent event. Mayo score was an instrument designed to measure disease activity of UC. Modified Mayo score was a composite index of 3 disease activity variables (stool frequency, rectal bleeding, and endoscopy). Each subscale was graded from 0 to 3 where higher score indicated more severe disease. These scores were summed to give a total score range of 0 to 9 where, higher score indicated more severe disease.

  At Week 54

Secondary Outcomes (19)

• Percentage of Participants With Clinical Remission at Week 14 Based on Modified Mayo Score

  At Week 14

• Percentage of Participants With Sustained Clinical Remission at Week 54 Based on Modified Mayo Score

  At Week 54

• Percentage of Participants With Sustained Endoscopic Remission at Week 54

  At Week 54

• Percentage of Participants With Endoscopic Response at Week 14

  At Week 14

• Percentage of Participants With Endoscopic Response at Week 54

  At Week 54

Study Arms (9)

Induction Period: Participants ≥30 kg, Vedolizumab 300 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Induction Period: Participants >15 to <30 kg, Vedolizumab 200 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Induction Period: Participants 10 to 15 kg, Vedolizumab 150 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Maintenance Period: Participants ≥30 kg, Vedolizumab 300 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Maintenance Period: Participants ≥30 kg, Vedolizumab 150 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Maintenance Period: Participants >15 to <30 kg, Vedolizumab 200 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Maintenance Period: Participants >15 to <30 kg, Vedolizumab 100 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Maintenance Period: Participants 10 to 15 kg, Vedolizumab 150 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Maintenance Period: Participants 10 to 15 kg, Vedolizumab 100 mg

EXPERIMENTAL

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.

Drug: Vedolizumab

Interventions

Vedolizumab DRUG

Vedolizumab IV infusion.

Also known as: MLN0002, ENTYVIO, KYNTELES

Induction Period: Participants 10 to 15 kg, Vedolizumab 150 mg; Induction Period: Participants >15 to <30 kg, Vedolizumab 200 mg; Induction Period: Participants ≥30 kg, Vedolizumab 300 mg; Maintenance Period: Participants 10 to 15 kg, Vedolizumab 100 mg; Maintenance Period: Participants 10 to 15 kg, Vedolizumab 150 mg; Maintenance Period: Participants >15 to <30 kg, Vedolizumab 100 mg; Maintenance Period: Participants >15 to <30 kg, Vedolizumab 200 mg; Maintenance Period: Participants ≥30 kg, Vedolizumab 150 mg; Maintenance Period: Participants ≥30 kg, Vedolizumab 300 mg

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Has moderately to severely active UC, unresponsive or intolerant to their current standard of care (SOC).
• Weighs ≥10 kg at the time of screening and enrollment into the study.
• 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.
• 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-α) 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.
• Has evidence of UC extending proximal to the rectum (i.e., not limited to proctitis), at a minimum.
• 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.
• Participants with vaccinations that are up-to-date based on the countrywide, accepted schedule of childhood vaccines.

You may not qualify if:

• 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.
• 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.
• 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.
• Has had clinically significant infection (eg, pneumonia, pyelonephritis, coronavirus disease 2019 \[COVID-19\]) within 30 days prior to first dose of study drug.
• Has received any live vaccinations within 30 days prior to first dose of study drug.
• Participants who currently require surgical intervention or are anticipated to require surgical intervention for UC during this study.
• Has had subtotal or total colectomy or have a jejunostomy, ileostomy, colostomy, ileo-anal pouch, or known fixed stenosis of the intestine.
• Participants with a current diagnosis of indeterminate colitis.
• Participants with clinical features suggesting monogenic very early onset inflammatory bowel disease.
• 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.
• 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 \[defined as no evidence of HCV RNA at least 12 weeks before baseline\]).
• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (65)

Phoenix Childrens Hospital -1919 E Thompson Rd

Phoenix, Arizona, 85016-7710, United States

Location

Rady Childrens Hospital San Diego - PIN

San Diego, California, 92123, United States

Location

Childrens Center For Digestive Healthcare

Atlanta, Georgia, 30318-4833, United States

Location

Advocate Children's Hospital Park Ridge

Park Ridge, Illinois, 60068, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MNGI Digestive Health PA-Plymouth

Minneapolis, Minnesota, 55413, United States

Location

Mayo Clinic - PIN

Rochester, Minnesota, 55905-0001, United States

Location

Goryeb Children's Hospital

Morristown, New Jersey, 07960-6136, United States

Location

UPMC Children's Hospital of Pittsburgh-120 Lytton Ave

Pittsburgh, Pennsylvania, 15224-1334, United States

Location

Texas Childrens Hospital West Campus

Houston, Texas, 77030-2358, United States

Location

Carilion Children's Tanglewood Center

Roanoke, Virginia, 24013-2253, United States

Location

Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

Queensland Childrens Hospital

South Brisbane, Queensland, 4101, Australia

Location

Monash Health, Monash Medical Centre

Clayton, Victoria, 3168, Australia

Location

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria, 3052, Australia

Location

UZ Antwerpen

Edegem, Antwerpen, 2650, Belgium

Location

Universitair Ziekenhuis Brussel - PIN

Jette, Brussels Capital, 1090, Belgium

Location

Universitaire Ziekenhuizen(UZ)Leuven-Campus Gasthuisberg

Leuven, Vlaams Brabant, 3000, Belgium

Location

University of Alberta Hospital

Edmonton, Alberta, T6G 2S2, Canada

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

Beijing Children's Hospital, Capital Medical University - PIN

Beijing, Beijing Municipality, 100045, China

Location

Henan Children's Hospital Zhengzhou Children's Hospital

Zhengzhou, Henan, 450000, China

Location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

Location

The Children's Hospital Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310003, China

Location

Klinika Za Djecje Bolesti Zagreb

Zagreb, City of Zagreb, 10000, Croatia

Location

Children's Hospital "Agia Sofia"

Athens, Attica, 115 27, Greece

Location

Attikon University General Hospital

Chaïdári, Attica, 124 62, Greece

Location

Ippokratio General Hospital of Thessaloniki

Thessaloniki, 546 42, Greece

Location

Clinexpert Gyogycentrum

Budapest, 1033, Hungary

Location

Semmelweis Egyetem

Budapest, 1085, Hungary

Location

Borsod-Abauj-Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktatokorhaz

Miskolc, 3526, Hungary

Location

Shaare Zedek Medical Center

Jerusalem, Jerusalem, 91031, Israel

Location

Hadassah Medical Center - PPDS

Jerusalem, Jerusalem, 91120, Israel

Location

Rambam Medical Center - PPDS

Haifa, 31096, Israel

Location

Carmel Medical Center

Haifa, 3436212, Israel

Location

Schneider Childrens Medical Center of Israel Petah Tikvah PIN

Petah Tikva, 49100, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

Azienda Ospedaliera Universitaria Federico II

Naples, Campania, 80131, Italy

Location

Azienda USL di Bologna

Bologna, Emilia-Romagna, 40133, Italy

Location

Azienda Ospedaliera Universitaria Policlinico Umberto I - Universita di Roma La Sapienza

Rome, Lazio, 00161, Italy

Location

Fondazione IRCCS San Gerardo dei Tintori - ASST di Monza A. O. San Gerardo

Monza, Monza E Brianza, 20900, Italy

Location

Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN

Florence, Tuscany, 50139, Italy

Location

AOU dell'Universita degli Studi della Campania Luigi Vanvitelli - Piazza Luigi Miraglia, 2

Naples, 280138, Italy

Location

Kurume University Hospital

Kurume, Hukuoka, 830-0011, Japan

Location

Juntendo University Hospital

Bunkyo-Ku, Tokyo, 113-8431, Japan

Location

National Center for Child Health and Development

Setagaya-ku, Tokyo, 157-8535, Japan

Location

Japanese Red Cross Kumamoto Hospital

Kumamoto, 861-8520, Japan

Location

Saitama Children's Medical Center

Saitama, 330-8777, Japan

Location

Uniwersytecki Szpital Dzieciecy

Krakow, Lesser Poland Voivodeship, 30-663, Poland

Location

WIP Warsaw IBD Point Profesor Kierkus

Warsaw, Masovian Voivodeship, 00-728, Poland

Location

Instytut 'Pomnik - Centrum Zdrowia Dziecka'

Warsaw, Masovian Voivodeship, 04-736, Poland

Location

Korczowski Bartosz, Gabinet Lekarski

Rzeszów, Podkarpackie Voivodeship, 35-302, Poland

Location

Gornoslaskie Centrum Zdrowia Dziecka Im. Sw. Jana Pawla II Spsk Nr 6 Sum W Katowicach

Katowice, Silesian Voivodeship, 40-752, Poland

Location

Twoja Przychodnia SCM

Szczecin, West Pomeranian Voivodeship, 71-434, Poland

Location

SPZOZ Centralny Szpital Kliniczny UM w Lodzi - ul. Pomorska 251

Lodz, Łódź Voivodeship, 91-738, Poland

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, Łódź Voivodeship, 93-338, Poland

Location

Kyungpook National University Chilgok Hospital

Daegu, Daegu Gwang'yeogsi, 41404, South Korea

Location

Gachon University Gil Medical Center

Seoul, Incheon Gwang'yeogsi, 3080, South Korea

Location

Samsung Medical Center

Seoul, Seoul Teugbyeolsi, 06351, South Korea

Location

Seoul National University Hospital

Seoul, Seoul Teugbyeolsi, 21565, South Korea

Location

The Royal London Hospital

London, London, City of, E1 1BB, United Kingdom

Location

Great Ormond Street Hospital

London, London, City of, WC1N 3JH, United Kingdom

Location

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, B4 6NH, United Kingdom

Location

Noahs Ark Childrens Hospital for Wales

Cardiff, CF14 4XW, United Kingdom

Location

Related Links

• Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.

MeSH Terms

Conditions

Colitis, Ulcerative

Interventions

vedolizumab

Condition Hierarchy (Ancestors)

Colitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Inflammatory Bowel Diseases; Colonic Diseases; Intestinal Diseases

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2021
• First Posted: March 3, 2021
• Study Start: October 19, 2021
• Primary Completion: July 1, 2025
• Study Completion: July 1, 2025
• Last Updated: April 30, 2026
• Results First Posted: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: 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.

Locations

GB (4); HU (3); US (11); IT (6); BE (3); CN (4); CR (1); JP (5); PL (8); AU (4); CA (3); KR (4); IL (6); GR (3)