Phase 3 Study of Vedolizumab Subcutaneous in Pediatric Subjects With Moderately to Severely Active Ulcerative Colitis or Crohn’s Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Takeda Development Center Americas Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

6 Jan 2025 → ongoing

Decision date (initial)

2024-11-11

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Takeda Development Center Americas, Inc.

External identifiers

EU CT number

2023-503188-40-00

ClinicalTrials.gov

NCT06100289

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Safety

The primary objective of this study is to assess the PK of vedolizumab SC in pediatric subjects with moderately to severely active UC or CD at steady state.

Conditions and MedDRA coding

Active Ulcerative Colitis or Crohn’s Disease

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-000645-PIP01-09

Plan to share IPD

Yes

IPD plan description

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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 01. The participant weighs ≥10 kg at the time of screening and enrollment into the study.
2. 02. Participants with UC or CD diagnosed at least 1 month before screening. Participants with moderately to severely active disease defined as: Participants with UC: 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). (The results of screening endoscopy should be applied.) Participants with CD: a pediatric Crohn's disease activity index (PCDAI) >30 and a simple endoscopic score for Crohn's disease (SES-CD) >6 (or an SES-CD ≥4 if disease is confined to terminal ileum) at screening endoscopy.
3. 03. Participants who have failed, lost response to, or been intolerant to treatment with at least 1 of the following agents: corticosteroids, immunomodulators (eg, azathioprine [AZA], 6-mercaptopurine [6-MP], methotrexate [MTX]), and/or tumor necrosis factor (TNF)-α antagonist therapy (eg, infliximab, adalimumab).
4. 04. Participants with evidence of UC extending proximal to the rectum (i.e., not limited to proctitis), at a minimum.
5. 05. Participants with 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.
6. 06. Participants with vaccinations that are up-to-date based on the countrywide accepted schedule of childhood vaccines.

Exclusion criteria 14

1. 01. Participants who have had 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 (ontamalimab), or rituximab.
2. 02. Participants who have had prior exposure to vedolizumab.
3. 03. Participants with hypersensitivity or allergies to vedolizumab or any of its excipients.
4. 04. Participants with active cerebral/meningeal disease, signs/symptoms or history of progressive multifocal leukoencephalopathy (PML) or any other major neurological disorders.
5. 05. The participant has received any live vaccinations within 30 days before first dose of study drug.
6. 06. Participants who currently require surgical intervention or are anticipated to require surgical intervention for UC or CD during this study.
7. 07. Participants who have had subtotal or total colectomy or have a jejunostomy, ileostomy, colostomy, ileo-anal pouch, known fixed stenosis of the intestine, short bowel syndrome, or >3 small intestine resections.
8. 08. Participants with a current diagnosis of indeterminate colitis.
9. 09. Participants with clinical features suggesting monogenic very early-onset inflammatory bowel disease (IBD).
10. 10. Participants with active or latent tuberculosis (TB).
11. 11. Participants with evidence of positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). Hepatitis B virus (HBV) immune subjects (ie, HBsAg negative and hepatitis B surface antibody [anti-HBs]-positive) may, however, be included.
12. 12. The participant has any identified congenital or acquired immunodeficiency (eg, common variable immunodeficiency, human immunodeficiency virus [HIV] infection, organ transplantation).
13. 13. Participants with positive stool studies for ova and/or parasites or stool culture at screening visit.
14. 14. Participants with positive Clostridioides difficile (C difficile) stool test at screening visit.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. 01. Ctrough,ss: Steady-state Median Observed Plasma Concentration at the End of a Dosing Interval for Vedolizumab at Week 34 [Time Frame: Predose at Week 34]
2. 02. Cavg,ss: Average Serum Concentration at Steady-state for Vedolizumab at Week 34 [Time Frame: Multiple time points prior to Week 34; pre-dose at Week 34]

Secondary endpoints 2

1. 01. Percentage of Participants with Positive Antivedolizumab Antibody (AVA) [Baseline up to 18 weeks after last dose of study drug (up to Week 50)]
2. 02. Percentage of Participants with Positive Neutralizing AVA [Baseline up to 18 weeks after last dose of study drug (up to Week 50)]

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

Sponsor organisation

Takeda Development Center Americas Inc.

Address

500 Kendall Street

City

Cambridge

Postcode

02142-1108

Country

United States

Scientific contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Ninfa Candela

Public contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Third parties 10

Locations

10 EU/EEA countries · 27 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 314 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

22 submissions — initial application plus substantial / non-substantial modifications