Ulcerative ColitisPhase 2NCT03196427
Long-term Safety With Vedolizumab Intravenous (IV) in Pediatric Participants With Ulcerative Colitis (UC) or Crohn's Disease (CD)
A Phase 2 study testing Vedolizumab in people with Ulcerative Colitis and Crohn's Disease, run by Takeda at 23 sites in 7 countries.
Trial summary
The purpose of this study is to determine the safety profile of long-term vedolizumab IV treatment in pediatric participants with UC or CD. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Ulcerative Colitis, Crohn's Disease
- 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
- 59 participants
- Where
- 23 sitesin 7 countries
- Study dates
- 30 Jul 2018to 17 Jul 2025
- Phase
- Phase 2
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
- Is male or female with UC or CD and was between 2 to 17 years, inclusive, at the time of randomization for Study MLN0002-2003.
- (Note: A participant remains eligible to participate in this study after they reach 18 years of age if they continue to meet the inclusion criteria and do not meet any exclusion criteria.)
- Has completed Study MLN0002-2003 and, at Week 22, achieved clinical response as defined by a reduction of partial Mayo score of >=2 points and >=25% from Baseline, or a reduction of the Paediatric Ulcerative Colitis Activity Index (PUCAI) of >=20 points from baseline for participants with UC; or a reduction of the CDAI as defined by a >=70-point decrease from Baseline or a decrease of Pediatric Crohn's Disease Activity Index (PCDAI) of >=15 points for participants with CD.
- May be receiving a therapeutic dose of the following drugs:
- Oral 5-aminosalicylic acid (5-ASA) compounds.
- Oral corticosteroid therapy (prednisone or equivalent steroid at a dose less than or equal to [<=] 50 milligram per day [mg/day]) provided the participant was receiving this medication during prior participation in MLN0002-2003.
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- Topical (rectal) treatment with 5-ASA or corticosteroids.
- Probiotics (example, Saccharomyces boulardii).
- Antidiarrheals (example, loperamide, diphenoxylate with atropine) for control of chronic diarrhea.
- Antibiotics used for the treatment of CD (i.e., ciprofloxacin, metronidazole).
- Azathioprine (AZA) or 6-mercaptopurine (6-MP) or methotrexate (MTX), provided the participant was receiving this medication during prior participation in MLN0002-2003.
- The participant's vaccinations are up to date as per inclusion criteria number 10 in MLN0002-2003.
Who cannot join
- Is female and is lactating or pregnant.
- Has hypersensitivity or allergies to vedolizumab or any of its excipients.
- Has withdrawn from Study MLN0002-2003.
- Has developed any new unstable or uncontrolled cardiovascular, heart failure moderate to severe (New York Class Association III or IV), pulmonary, hepatic, renal, gastrointestinal (GI), genitourinary, hematological, coagulation, immunological, endocrine/metabolic, neurological, or other medical disorder that, in the opinion of the investigator, would confound the study results or compromise participant safety.
- Has a positive progressive multifocal leukoencephalopathy (PML) subjective symptom checklist prior to the administration of the first dose of study drug.
- Currently requires major surgical intervention for UC or CD (example, bowel resection), or is anticipated to require major surgical intervention for UC or CD during the study.
- Has other serious comorbidities that will limit his or her ability to complete the study.
Trial locations and contacts
23 sites in 7 countries.
23 sites in 7 countries
United States9 sites
Children's Center for Digestive Healthcare
Atlanta, Georgia, 30342
Connecticut Children's Medical Center
Hartford, Connecticut, 06106-3322
Texas Children's Hospital
Houston, Texas, 77030
Nemours Childrens Specialty Care - Jacksonville
Jacksonville, Florida, 32207
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Columbia University Medical Center
New York, New York, 10031
Children's Hospital of Orange County
Orange, California, 92868
University of California San Francisco
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Israel5 sites
Carmel Medical Center
Haifa, 3436212
Shaare Zedek Medical Center
Jerusalem, 9103102
Schneider Children's Medical Center of Israel
Petach Tiqwa, 4920235
The Edmond and Lily Safra Children's Hospital - Sheba Medical Center
Ramat Gan, Tel Aviv, 52621
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Hungary3 sites
Debreceni Egyetem Klinikai Kozpont
Debrecen, Hajdú-Bihar, 4032
BAZ Megyei Korhaz es Egyetemi Oktatokorhaz
Miskolc, Borsod-Abauj Zemplen county, 3526
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
Szeged, Csongrád megye, 6720
Poland3 sites
Uniwersytecki Szpital Dzieciecy w Krakowie
Krakow, Lesser Poland Voivodeship, 30-663
Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi Osrodek Pediatryczny im Marii Konopnic
Lodz, Łódź Voivodeship, 91-738
Gabinet Lekarski Dr. Hab. N. Med. Bartosz Korczowski
Rzeszów, Podkarpackie Voivodeship, 35-302
United Kingdom1 site
Barts and The London NHS Trust
London, England, E1 1BB
About this study
Official title: A Phase 2b, Extension Study to Determine the Long-term Safety of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn's Disease
The purpose of this study is to determine the safety profile of long-term vedolizumab IV treatment in pediatric participants with UC or CD.
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 or CD.
This study will look at the long-term safety profile in participants who take vedolizumab IV. Participants will continue receiving the same dose assigned from the parent study MLN0002-2003 [NCT03138655], which will remain blinded until week 40.
The dosing regimen selected for the long-term study is intended to maintain clinical response at the lowest possible exposure.
At the discretion of the investigator, participants receiving the low dose (150 or 100 milligram [mg]) of vedolizumab IV may be escalated to the high dose (300 or 200 mg) if the participants demonstrate disease worsening at 2 consecutive visits (scheduled or unscheduled).
Participants who experience continued disease worsening during the study despite being administered vedolizumab 300 or 200 mg every 8 weeks (Q8W) will be discontinued from the study.
Study duration will be until vedolizumab IV is commercially available for pediatric indication(s) in the participant's country or until other drug access programs become available (whichever comes first), the participant turns 18 years of age and can be transitioned to commercial drug, the participant withdraws from the study, or the sponsor decides to close the study (up to approximately 8 years).
- Conditions
- Ulcerative Colitis, Crohn's Disease
- Being tested
- Vedolizumab
- Keywords
- Drug Therapy
- Medical subject headings
- Colitis, Ulcerative, Crohn Disease
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 intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Vedolizumab High Dose Group | Experimental | Participants with UC or CD having baseline weight of greater than or equal to (>=) 30 kilogram (kg) will receive vedolizumab 300 mg and participants with UC or CD having baseline weight of less than (<) 30 kg will receive vedolizumab 200 mg, IV infusion, every 8 weeks until vedolizumab IV is commercially available for pediatric indication(s) in the participant's country or until other drug access programs become available (whichever comes first), the participant turns 18 years of age and can be transitioned to commercial drug (up to approximately 8 years). Receives: Drug: Vedolizumab |
| Vedolizumab Low Dose Group | Experimental | Participants with UC or CD having baseline weight of >= 30 kg will receive vedolizumab 150 mg and participants with UC or CD having baseline weight of < 30 kg will receive vedolizumab 100 mg IV infusion, every 8 weeks until vedolizumab IV is commercially available for pediatric indication(s) in the participant's country or until other drug access programs become available (whichever comes first), the participant turns 18 years of age and can be transitioned to commercial drug (up to approximately 8 years). Receives: Drug: Vedolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
AE defined as any untoward medical occurrence in clinical investigation participants administered drug; it does not necessarily have to have causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug whether or not it was considered related to the drug. TEAE was defined as an AE whose date of onset occurred on or after the first dose of study drug, or an already-present AE that worsened in intensity or frequency following the treatment start, occurring from the first dose of study drug to the day of last dose of study drug.
Measured: From first dose of study drug up to end of follow up (up to 6.8 years)
Secondary outcomes (15)
Percentage of Participants With UC Who Achieved and Maintained Clinical Response Based on Complete Mayo Score
Clinical response was defined as a continued reduction in complete Mayo score of >=3 points and >=30 percent (%) from baseline (at initiation of MLN0002-2003) and continued decrease in rectal bleeding subscore of >=1 point from baseline, or absolute rectal bleeding subscore of less than or equal to (<=1) point. Mayo score was an instrument designed to measure disease activity of UC. It consisted of 4 subscores: stool frequency, rectal bleeding, findings on endoscopy and physician rating of disease activity, each graded from 0 to 3 where 0 indicated normal and 3 indicated more severe disease. These scores were summed to give a total score range of 0 to 12; where higher scores indicated more severe disease.
Measured: At Week 32
Percentage of Participants With CD Who Achieved and Maintained Clinical Response Based on Simple Endoscopic Score for Crohn's Disease (SES-CD) Score and Crohn's Disease Activity Index (CDAI) at Week 32
Maintenance of clinical response based on SES-CD and CDAI was defined as a 50% reduction in SES-CD score on endoscopy compared to the baseline endoscopy [at initiation of MLN0002-2003 (NCT03138655)]; and continued reduction in CDAI that is a >= 70 point decrease from the baseline CDAI score at the initiation of MLN0002-2003 (NCT03138655). CDAI was a research tool used to quantify the symptoms of participants with Crohn's disease. SES-CD consisted of 3 variables: ulcer size, ulcerated and affected surfaces and presence of narrowing each graded from 0 to 3 with score of 0 means no colonic lesions or mucosal healing, and SES-CD greater than (>) 1 indicated the presence of mucosal lesions.
Measured: At Week 32
Time to Major Inflammatory Bowel Disease (IBD) - Related Events
Time to major IBD-related events was defined as time from study treatment start to first major IBD-related hospitalization, surgery, or procedure due to UC and CD.
Measured: Up to 6.8 years
Change From Baseline in IMPACT-III - Total Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains: Bowel Symptoms (7 items), Systemic Symptoms (3 items), Social Functioning (12 items), Body Image (3 items), Treatment/Interventions (3 items), and Emotional Functioning (7 items). The IMPACT-III used a 5-point Likert scale ranging from 1 to 5 for all answers. The total score was obtained by summing individual domain scores. The total score ranged from 35 to 175, with higher scores suggesting better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Change From Baseline in IMPACT-III - Bowel Symptoms Domain Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains. The Bowel Symptoms domain consisted of 7 items. Each item was scored using a 5-point Likert scale ranging from 1 to 5. The bowel symptoms domain score ranged from 7 to 35, with higher scores suggesting a better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Change From Baseline in IMPACT-III - Systemic Symptoms Domain Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains. The systemic symptoms domain consisted of 3 items. Each item was scored using a 5-point Likert scale ranging from 1 to 5. The systemic symptoms domain score ranged from 3 to 15, with higher scores suggesting a better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Change From Baseline in IMPACT-III - Social Functioning Domain Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains. The social functioning domain consisted of 12 items. Each item was scored using a 5-point Likert scale ranging from 1 to 5. The social functioning domain score ranged from 12 to 60, with higher scores suggesting a better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Change From Baseline in IMPACT-III - Body Image Domain Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains. The body image domain consisted of 3 items. Each item was scored using a 5-point Likert scale ranging from 1 to 5. The body image domain score ranged from 3 to 15, with higher scores suggesting a better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Change From Baseline in IMPACT-III - Treatment/Interventions Domain Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains. The treatment/interventions domain consisted of 3 items. Each item was scored using a 5-point Likert scale ranging from 1 to 5. The treatment/interventions domain score ranged from 3 to 15, with higher scores suggesting a better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Change From Baseline in IMPACT-III - Emotional Functioning Domain Score
The IMPACT-III questionnaire was a self-reported measure with 35 closed questions encompassing 6 domains. The emotional functioning domain consisted of 7 items. Each item was scored using a 5-point Likert scale ranging from 1 to 5. The emotional functioning domain score ranged from 7 to 35, with higher scores suggesting a better quality of life.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312
Height Velocity at Week 48 and Every 48 Weeks
Height velocity (centimeter per year [cm/year]) was calculated as the change in height divided by the duration [time between the two height measures].
Measured: At Weeks 48, 96, 144, 192, 240, 288, and 336
Change From Baseline in Height
Change from baseline in height was reported.
Measured: Baseline, Weeks 48, 96, 144, 192, 240, 288, 336
Change From Baseline in Weight
Change from baseline in weight was reported.
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, and 336
Change From Baseline in Body Mass Index (BMI)
BMI was calculated as Weight (in kilograms)/height (in meters square).
Measured: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, and 336
Percentage of Participants Who Achieved Tanner Stage V at or Before Age 16 (in Females) or Age 17 (in Males)
Tanner Stage Evaluation was a scale used to evaluate growth parameters standardized for age, sex, and pubertal development. 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). Tanner stage was assessed at or before age 16 years for females or 17 years for males.
Measured: Up to 6.8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2017
- Study start30 Jul 2018
- Primary completion3 Apr 2025
- Study completion17 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 Ulcerative ColitisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03196427
- Sponsor's protocol ID
- Vedolizumab-2005
- Other IDs
- 2017-002182-21, U1111-1176-5741, 17/NE/0258, MOH_2017-09-18_000649, 2023-507766-35-00, 2023, 2017
- Status last verified
- Apr 2026
- First submitted
- 20 Jun 2017
- Last updated
- 13 May 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.