Colitis, UlcerativePhase 3NCT04779307
A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Ulcerative Colitis (UC)
A Phase 3 study testing Vedolizumab in people with Colitis, Ulcerative, run by Takeda at 65 sites in 14 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Colitis, Ulcerative
- What is being tested?
- Vedolizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 121 participants
- Where
- 65 sitesin 14 countries
- Study dates
- 19 Oct 2021to 1 Jul 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- Has positive stool studies for ova and/or parasites or stool culture at screening visit.
- Has positive Clostridioides difficile (C difficile) stool test at screening visit.
- Other inclusion/exclusion criteria may apply.
Trial locations and contacts
65 sites in 14 countries.
65 sites in 14 countries
United States11 sites
Childrens Center For Digestive Healthcare
Atlanta, Georgia, 30318-4833
Boston Children's Hospital
Boston, Massachusetts, 02115
Texas Childrens Hospital West Campus
Houston, Texas, 77030-2358
MNGI Digestive Health PA-Plymouth
Minneapolis, Minnesota, 55413
Goryeb Children's Hospital
Morristown, New Jersey, 07960-6136
Advocate Children's Hospital Park Ridge
Park Ridge, Illinois, 60068
Phoenix Childrens Hospital -1919 E Thompson Rd
Phoenix, Arizona, 85016-7710
UPMC Children's Hospital of Pittsburgh-120 Lytton Ave
Pittsburgh, Pennsylvania, 15224-1334
Carilion Children's Tanglewood Center
Roanoke, Virginia, 24013-2253
Mayo Clinic - PIN
Rochester, Minnesota, 55905-0001
Rady Childrens Hospital San Diego - PIN
San Diego, California, 92123
Poland8 sites
Gornoslaskie Centrum Zdrowia Dziecka Im. Sw. Jana Pawla II Spsk Nr 6 Sum W Katowicach
Katowice, Silesian Voivodeship, 40-752
Uniwersytecki Szpital Dzieciecy
Krakow, Lesser Poland Voivodeship, 30-663
SPZOZ Centralny Szpital Kliniczny UM w Lodzi - ul. Pomorska 251
Lodz, Łódź Voivodeship, 91-738
Instytut Centrum Zdrowia Matki Polki
Lodz, Łódź Voivodeship, 93-338
Korczowski Bartosz, Gabinet Lekarski
Rzeszów, Podkarpackie Voivodeship, 35-302
Twoja Przychodnia SCM
Szczecin, West Pomeranian Voivodeship, 71-434
WIP Warsaw IBD Point Profesor Kierkus
Warsaw, Masovian Voivodeship, 00-728
Instytut 'Pomnik - Centrum Zdrowia Dziecka'
Warsaw, Masovian Voivodeship, 04-736
Israel6 sites
Rambam Medical Center - PPDS
Haifa, 31096
Carmel Medical Center
Haifa, 3436212
Shaare Zedek Medical Center
Jerusalem, Jerusalem, 91031
Hadassah Medical Center - PPDS
Jerusalem, Jerusalem, 91120
Schneider Childrens Medical Center of Israel Petah Tikvah PIN
Petah Tikva, 49100
Tel Aviv Sourasky Medical Center
Tel Aviv, 64239
Italy6 sites
Azienda USL di Bologna
Bologna, Emilia-Romagna, 40133
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Florence, Tuscany, 50139
Fondazione IRCCS San Gerardo dei Tintori - ASST di Monza A. O. San Gerardo
Monza, Monza E Brianza, 20900
Azienda Ospedaliera Universitaria Federico II
Naples, Campania, 80131
AOU dell'Universita degli Studi della Campania Luigi Vanvitelli - Piazza Luigi Miraglia, 2
Naples, 280138
Azienda Ospedaliera Universitaria Policlinico Umberto I - Universita di Roma La Sapienza
Rome, Lazio, 00161
Japan5 sites
Juntendo University Hospital
Bunkyo-Ku, Tokyo, 113-8431
Japanese Red Cross Kumamoto Hospital
Kumamoto, 861-8520
Kurume University Hospital
Kurume, Hukuoka, 830-0011
Saitama Children's Medical Center
Saitama, 330-8777
National Center for Child Health and Development
Setagaya-ku, Tokyo, 157-8535
Australia4 sites
Monash Health, Monash Medical Centre
Clayton, Victoria, 3168
Royal Children's Hospital Melbourne - PIN
Parkville, Victoria, 3052
Queensland Childrens Hospital
South Brisbane, Queensland, 4101
Children's Hospital at Westmead
Westmead, New South Wales, 2145
China4 sites
Beijing Children's Hospital, Capital Medical University - PIN
Beijing, Beijing Municipality, 100045
The Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310003
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
Henan Children's Hospital Zhengzhou Children's Hospital
Zhengzhou, Henan, 450000
South Korea4 sites
Kyungpook National University Chilgok Hospital
Daegu, Daegu Gwang'yeogsi, 41404
Gachon University Gil Medical Center
Seoul, Incheon Gwang'yeogsi, 3080
Samsung Medical Center
Seoul, Seoul Teugbyeolsi, 06351
Seoul National University Hospital
Seoul, Seoul Teugbyeolsi, 21565
United Kingdom4 sites
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, West Midlands, B4 6NH
Noahs Ark Childrens Hospital for Wales
Cardiff, CF14 4XW
The Royal London Hospital
London, London, City of, E1 1BB
Great Ormond Street Hospital
London, London, City of, WC1N 3JH
Belgium3 sites
UZ Antwerpen
Edegem, Antwerpen, 2650
Universitair Ziekenhuis Brussel - PIN
Jette, Brussels Capital, 1090
Universitaire Ziekenhuizen(UZ)Leuven-Campus Gasthuisberg
Leuven, Vlaams Brabant, 3000
Canada3 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2S2
London Health Sciences Centre
London, Ontario, N6A 5W9
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
Greece3 sites
Children's Hospital "Agia Sofia"
Athens, Attica, 115 27
Attikon University General Hospital
Chaïdári, Attica, 124 62
Ippokratio General Hospital of Thessaloniki
Thessaloniki, 546 42
Hungary3 sites
Clinexpert Gyogycentrum
Budapest, 1033
Semmelweis Egyetem
Budapest, 1085
Borsod-Abauj-Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktatokorhaz
Miskolc, 3526
About this study
Official 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
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.
Read more
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.
Read the detailed description
The drug being tested in this study is called vedolizumab. Vedolizumab is being tested to treat pediatric participants who have moderately to severely active UC. Participants to be enrolled must have failed response to, lost response to, or been intolerant to at least 1 of the current standard of care (SOC) induction and maintenance therapies for UC including immunomodulators (eg, azathioprine [AZA], 6-mercaptopurine [6-MP], methotrexate [MTX]), and tumor necrosis factor-alpha (TNF-α) antagonists (eg, infliximab, adalimumab).
The study will enroll approximately 120 patients.
During the Induction Period participants will receive 3 doses of vedolizumab IV infusion at Day 1, Week 2, and Week 6 based on their weight at Baseline:
- Participants ≥30 kg, Vedolizumab 300 mg
- Participants >15 to <30 kg, Vedolizumab 200 mg
- Participants 10 to 15 kg, Vedolizumab 150 mg
At Week 14, participants who achieve clinical response will be randomly assigned (by chance, like flipping a coin) in a 1:1 ratio to one of the 2 double-blind dose groups (high dose and low dose), stratified by previous exposure/failure to TNF-α antagonists therapy or naive to TNF-α antagonists therapy, and weight. Participants will receive vedolizumab IV infusions every 8 weeks (Q8W) up to Week 46 during the Maintenance Period as follows:
- Participants ≥30 kg, Vedolizumab 300 mg (High dose) or 150 mg (Low dose)
- Participants >15 to <30 kg, Vedolizumab 200 mg (High dose) or 100 mg (Low dose)
- Participants 10 to 15 kg, Vedolizumab 150 mg (High dose) or 100 mg (Low dose)
The dose will remain blinded to the participant and study doctor and staff during the study (unless there is an urgent medical need). All participants will be administered vedolizumab via IV infusion. In participants who demonstrate lack of maintenance of clinical response during the Maintenance Period the dose will be escalated in a blinded fashion based on the weight at the time of the worsening of disease. In addition one-time rescue therapy with corticosteroids is allowed during Maintenance period.
This multi-center trial will be conducted worldwide. After the Week 54 visit, participants who are younger than 18 years may be eligible to continue receiving vedolizumab in extension study MLN0002-3029 (NCT05442567). Participants who do not maintain corticosteroid-free clinical response at Week 54 or who discontinue study drug at any time during the induction or maintenance periods of this study will undergo an end of study (EOS) or early termination (ET) visit, as well as a safety visit 18 weeks after the last dose of vedolizumab, in addition these participants would enter study MLN0002-3029 for an observational long-term follow-up (LTFU) period of 2 years after the last dose of study drug in the current study. During the LTFU period, data will be collected either by clinic visit OR, if site attendance is not feasible, by phone call every 6 months.
- Conditions
- Colitis, Ulcerative
- Being tested
- Vedolizumab
- Keywords
- Drug Therapy
- Medical subject headings
- Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VedolizumabDrug
Also called MLN0002, ENTYVIO, KYNTELES
Vedolizumab IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: 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. Receives: Drug: Vedolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At Week 54
Secondary outcomes (19)
Percentage of Participants With Clinical Remission at Week 14 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.
Measured: At Week 14
Percentage of Participants With Sustained Clinical Remission at Week 54 Based on Modified Mayo Score
Participants who had clinical remission at Week 14 were analyzed for sustained clinical remission at Week 54. 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.
Measured: At Week 54
Percentage of Participants With Sustained Endoscopic Remission at Week 54
Participants who had endoscopic remission at Week 14 were analyzed for sustained endoscopic remission at Week 54. Mayo endoscopic sub score (MES) was a subscale of the Mayo score, an instrument designed to measure disease activity of UC. The subscale was graded from 0 to 3 based on the findings on endoscopy where 0=normal or inactive disease, 1=mild disease (erythema, decreased vascular pattern), 2=moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3=severe disease (spontaneous bleeding, ulceration). Higher score indicated more severe disease.
Measured: At Week 54
Percentage of Participants With Endoscopic Response at Week 14
Endoscopic response was defined as a decrease from baseline in the MES >=1 point. MES was a subscale of the Mayo score, an instrument designed to measure disease activity of UC. The subscale was graded from 0 to 3 based on the findings on endoscopy where 0=normal or inactive disease, 1=mild disease (erythema, decreased vascular pattern), 2=moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3=severe disease (spontaneous bleeding, ulceration). Higher score indicated more severe disease.
Measured: At Week 14
Percentage of Participants With Endoscopic Response at Week 54
Endoscopic response was defined as a decrease from baseline in the MES >=1 point. MES was a subscale of the Mayo score, an instrument designed to measure disease activity of UC. The subscale was graded from 0 to 3 based on the findings on endoscopy where 0=normal or inactive disease, 1=mild disease (erythema, decreased vascular pattern), 2=moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3=severe disease (spontaneous bleeding, ulceration). Higher score indicated more severe disease.
Measured: At Week 54
Percentage of Participants With Corticosteroid-free Clinical Remission at Week 54
Corticosteroid-free clinical remission based on the modified Mayo score was defined as when a participant meets the definition described in the primary endpoint and was off corticosteroids at least 12 weeks prior to and at Week 54 and without presence of any intercurrent event. 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.
Measured: At Week 54
Percentage of Participants With Clinical Remission at Week 54 Based on Complete Mayo Score
Clinical remission based on complete Mayo score was a score (inclusive of physician global assessment [PGA]) of <=2 points with no individual sub score >1 and without presence of any intercurrent event. Mayo score was an instrument designed to measure disease activity of UC. Complete Mayo score was a composite index of 4 disease activity variables (stool frequency, rectal bleeding, endoscopy [modified so that a score of 1 does not include friability], and PGA sub scores) ranging from 0-12. Higher score indicated more severe disease.
Measured: At Week 54
Serum Trough Concentrations of Vedolizumab Over Time
Serum trough concentration of vedolizumab was reported.
Measured: Predose at Week 14 and post dose at Week 54
Percentage of Participants With Positive Anti-vedolizumab Antibodies (AVA)
AVA positive was defined as a confirmed AVA positive result.
Measured: Up to Week 54
Percentage of Participants With Positive Neutralizing AVA
Positive Neutralizing AVA was defined as a positive result in the neutralizing AVA assay at any visit.
Measured: Up to Week 54
Percentage of Participants With Sustained Clinical Response at Week 54 Based on Complete Mayo Score
Participants who had clinical response at Week 14 were analyzed for sustained clinical response at Week 54. Sustained refers to meeting the specific endpoint criteria at both Week 14 and Week 54. Sustained clinical response is defined as meeting the following criteria at both Week 14 and Week 54: reduction in complete Mayo score of >=3 points and >=30% from baseline with an accompanying decrease in rectal bleeding sub score of >=1 point or absolute rectal bleeding sub score of <=1 point. Mayo score was an instrument designed to measure disease activity of UC. Complete Mayo score was a composite index of 4 disease activity variables (stool frequency, rectal bleeding, endoscopy [modified so that a score of 1 does not include friability], and PGA sub scores) ranging from 0-12. Higher score indicated more severe disease.
Measured: At Week 54
Percentage of Participants With Clinical Response up to Week 54 Based on Partial Mayo Score
Clinical response was where a participant achieved clinical response if they had a reduction of >=2 points and >=25% from the baseline partial Mayo score, including a >=1 point decrease in the Mayo stool frequency sub score and a >=1 point reduction in the rectal bleeding sub score or absolute rectal bleeding sub score of <=1 point. Mayo score was an instrument designed to measure disease activity of UC. A partial Mayo score was defined as composite index of 3 disease activity variables (stool frequency, rectal bleeding, and PGA sub scores) that ranged from 0-9 and excluded the endoscopy sub score. Higher score indicates more severe disease.
Measured: At Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Percentage of Participants With Clinical Remission up to Week 54 Based on Partial Mayo Score
Clinical remission based on partial Mayo score was defined as a partial Mayo score of <=2 points and no individual sub score >1 point. Mayo score was an instrument designed to measure disease activity of UC. A partial Mayo score was defined as composite index of 3 disease activity variables (stool frequency, rectal bleeding, and PGA sub scores) that ranged from 0-9 and excluded the endoscopy sub score. Higher score indicates more severe disease.
Measured: At Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Percentage of Participants With at Least One Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (TESAE), and Adverse Event of Special Interest (AESI)
A TEAE was defined as an AE whose date of onset occurred on or after the first dose of study drug through Week 54 for participants entering the extension study or the final safety visit 18 weeks after their last dose of study drug for those who do not enter the extension study or those who early terminate. A TESAE was defined as an undesirable event that was not present prior to medical treatment or an already present event that worsened either in intensity or frequency following the first dose of study drug, that occurred from the first dose of study drug to the day of last dose of study drug + 126 days. AESI was defined as an AE (serious or nonserious) of medical concern specific to the compound or program, for which ongoing monitoring and rapid communication by the investigator to the sponsor was appropriate. AESIs include infusion-related reactions and hypersensitivity, serious infection, malignancy, or other (liver injury and progressive multifocal leukoencephalopathy [PML]).
Measured: From first dose of study drug up to end of follow up (up to 3.7 years)
Change From Baseline in Weight
Change from baseline in weight was reported.
Measured: At Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Change From Baseline in Weight Z-score
Change from baseline in weight Z-score was reported. Weight z-score expresses how an individual's measured weight compares to the expected weight of a reference population of the same age and sex, standardized using population growth charts. It represents the number of standard deviations (SDs) an individual's weight is above or below the mean of the reference population. Z-score was calculated as: Z-score = (observed value - median value of the reference population) / standard deviation value of reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score indicates that the observed value is below (lower than) the reference mean, while a positive Z-score indicates that the observed value is above (higher than) the reference mean.
Measured: At Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Change From Baseline in Height
Change from baseline in height was reported.
Measured: At Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Change From Baseline in Linear Growth Z-score
Change from baseline in linear growth Z-score were reported. Linear growth Z-score is a standardized measure that describes how far a measured height deviates from the median height of a reference population of the same age and sex based on established growth charts. It is expressed in units of standard deviations (SD). Z-score was calculated as: Z-score = (observed value - median value of the reference population) / standard deviation value of reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score indicates that the observed value is below (lower than) the reference mean, while a positive Z-score indicates that the observed value is above (higher than) the reference mean.
Measured: At Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Number of Participants With Change From Baseline in Tanner Stage at Week 54
Tanner stages are used to evaluate growth parameters. They are standardized for age, sex, and pubertal development, with Stage 1 representing the prepubertal stage and Stage 5 representing the fully mature adult stage. It measures Female pubertal development staged by pubic hair development and breast size; male pubertal development staged by size of the genitalia and development of pubic hair. Rated in 5 stages: stage 1 (no development) to 5 (adult-like development in quantity and size). The data reported shows shifts in participants' Tanner stages from baseline to Week 54.
Measured: At Week 54
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2021
- Study start19 Oct 2021
- Primary completion1 Jul 2025
- Study completion1 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Colitis, UlcerativeStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 121 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04779307
- Sponsor's protocol ID
- MLN0002-3024
- Other IDs
- 2020-004300-34, jRCT2071210030, 2023-509018-12-00, 2023
- Status last verified
- Apr 2026
- First submitted
- 1 Mar 2021
- Last updated
- 30 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.