A Long-term Study in Children and Teenagers with Ulcerative Colitis or Crohn’s Disease treated with Vedolizumab IV

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

26 Apr 2023 → ongoing

Decision date (initial)

2024-07-30

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Takeda Development Center Americas, Inc.

External identifiers

EU CT number

2023-509046-36-00

EudraCT number

2021-000630-34

ClinicalTrials.gov

NCT05442567

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

The primary objective of the treatment cohort is to evaluate the safety profile of long-term vedolizumab IV treatment in pediatric subjects with UC or CD.

Secondary objectives 1

1. Secondary Objectives for the Treatment Cohort: · To evaluate the effect of long-term vedolizumab IV treatment on time to major inflammatory bowel disease (IBD)-related events (eg, hospitalizations, surgeries, and procedures) in pediatric subjects with UC or CD. · To evaluate quality of life in subjects aged 9 to 17 years who were treated with vedolizumab IV using the IMPACT-III questionnaire. Objectives for the Observational Cohort: · The objectives for the observational cohort are to assess prespecified safety events of interest and monitor growth and pubertal development for approximately 2 years after the last dose of study drug in Studies MLN0002-3024 or MLN0002-3025.

Conditions and MedDRA coding

Active Ulcerative Colitis (UC) or Crohn's disease (CD)

Study design 1 period

Regulatory references

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 8

1. 1. In the opinion of the investigator, the subject, or subject`s legally authorized representative, subject`s parent, or legal guardian (adult caregiver) is capable of understanding and complying with protocol requirements.
2. 2. The subject, subject’s legally authorized representative or adult caregive signs and dates a written, informed consent and/or pediatric assent form and any required privacy authorization before the initiation of any study procedures, as required per local regulations.
3. The subject is male or female with UC or CD and aged <18 years. (Note: Subjects who reach adult age [18 years of age in most jurisdictions/regions] during the study should be considered to transition to commercial drug if available in their country, especially if transition to an adult care setting is required by local regulation.).
4. The subject has completed Study MLN0002-3024 or Study MLN0002-3025 and has achieved corticosteroid-free clinical response at Week 54 (and has tapered off of steroids, as applicable, at least 12 weeks before Week 54) as defined by a reduction of partial Mayo score of ≥2 points and ≥25% from baseline for subjects with UC; or a reduction of PCDAI of ≥15 points for subjects with CD from baseline of the parent study and with total PCDAI ≤30.
5. 5. A male subject who is sexually active with a female partner of childbearing potential agrees to use a barrier method of contraception (eg, condom with or without spermicide) from signing of subject/ parental informed consent and/or pediatric assent throughout the duration of the study and for 18 weeks after last dose. The female partner of a male subject should also be advised to use a highly effective method of contraception.
6. 6. A female subject of childbearing potential who is sexually active with a nonsterilized male partner agrees to use a highly effective method of contraception from signing of subject/parental informed consent and/or pediatric assent throughout the duration of the study and 18 weeks after the last dose.
7. Main Criteria for Inclusion in the Observational Cohort: The subject, parent, or legal guardian signs and dates a written, informed consent form.
8. The subject has received at least 1 dose of vedolizumab during Study MLN0002-3024 or Study MLN0002-3025 and early terminated OR completed the Week 54 visit of Study MLN0002-3024 or Study MLN0002-3025 but was not eligible to enroll in the treatment cohort of this study.

Exclusion criteria 6

1. Treatment Cohort only: The subject is female and is lactating or pregnant.
2. The subject has hypersensitivity or allergies to vedolizumab or any of its excipients.
3. The subject currently requires major surgical intervention for UC or CD (eg, bowel resection), or is anticipated to require major surgical intervention for UC or CD during the study.
4. The subject has developed any new unstable or uncontrolled cardiovascular, heart failure moderate to severe (New York Class Association III or IV), pulmonary, hepatic, renal, gastrointestinal, 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 subject safety.
5. The subject has other serious comorbidities that will limit their ability to complete the study.
6. The subject is unable to comply with all study assessments.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. For the Treatment Cohort: The primary endpoint for this study is the incidence of adverse events (AEs).
2. For the Observational Cohort: Incidence of prespecified safety events (serious infections, malignancies, progressive multifocal leukoencephalopathy [PML], concerns about growth and pubertal development, and bowel surgery).

Secondary endpoints 1

1. For the Treatment Cohort: • Time to major IBD-related events (eg, hospitalizations, surgeries, or procedures). • Changes from baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III total and subscale scores for subjects aged 9 to 17 years as measured every 24 weeks.

Investigational products

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

Test 1

Auxiliary 1

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

Director, Clinical Operations

Public contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Third parties 8

Locations

6 EU/EEA countries · 23 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 95 files

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

Application history

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