An extension study to investigate how safe Vedolizumab is in child patients with 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

9 Jan 2018 → 17 Jul 2025

Decision date (initial)

2024-03-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Takeda Development Centre Americas, Ltd

External identifiers

EU CT number

2023-507766-35-00

EudraCT number

2017-002182-21

WHO UTN

U1111-1176-5741

ClinicalTrials.gov

NCT03196427

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To determine the safety profile of long-term vedolizumab IV treatment in pediatric subjects with UC or CD.

Secondary objectives 4

1. To evaluate the efficacy of long-term vedolizumab IV in pediatric subjects with UC or CD
2. To determine the effect of long-term vedolizumab IV treatment on time to major inflammatory bowel disease (IBD)-related events (hospitalizations, surgeries, and procedures) in pediatric subjects with UC or CD
3. To examine the effect of long-term vedolizumab IV treatment on healthrelated quality-of- life measurements in pediatric subjects with UC or CD
4. To determine the effect of long-term vedolizumab IV treatment onpatterns of growth and development in pediatric subjects with UC or CD

Conditions and MedDRA coding

Ulcerative Colitis or Crohn's Disease

Study design 1 period

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 3

1. The subject is male or female with UC or CD and was between 2 to 17 years, inclusive, at the time of their randomization in Study MLN0002-2003. (Note: A subject remains eligible to participate in this study after they reach 18 years of age if they continue to meet the inclusion criteriaand do not meet any exclusion criteria)
2. The subject 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 PUCAI of ≥20 points from baseline for subjects with UC; or a reduction of the CDAI as defined by a ≥70-point decrease from Baseline or a decrease of PCDAI of ≥15 points for subjects with CD
3. The subject may be receiving a therapeutic dose of the following drugs:Oral 5-aminosalicylic (5-ASA) compounds; Oral corticosteroid therapy (prednisone or equivalent steroid at a dose ≤50 mg/day);Topical (rectal) treatment with 5-ASA or corticosteroids; Probiotics (eg, Saccharomyces boulardii);Antidiarrheals (eg, loperamide, diphenoxylate with atropine) for control of chronic diarrhea; Antibiotics used for treatment of CD (eg, ciprofloxacin, metronidazole);Azathioprine, 6-mercaptopurine, or methotrexate provided the subject was receiving this medication during prior participation in Study MLN0002-2003.

Exclusion criteria 7

1. 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 has withdrawn from Study MLN0002-2003.
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 a positive progressive multifocal leukoencephalopathy (PML) subjective symptom checklist prior to the administration of the first dose of study drug.
6. 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.
7. The subject has other serious comorbidities that will limit their ability to complete the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint for this study is percentage of subjects with treatment-emergent adverse events (TEAEs).

Secondary endpoints 7

1. Percentage of UC subjects who, at Week 32, achieve and maintain clinical response based on complete Mayo score, as defined by a continued reduction in complete Mayo score of ≥3 points from the baseline (at initiation of MLN0002-2003) and continued decrease in rectal bleeding subscore of ≥1 point from baseline, or absolute rectal bleeding subscore of ≤1 point at Week 32.
2. Percentage of CD subjects who, at Week 32, achieve and maintain clinical response as defined by a 50% reduction in SES-CD score on endoscopy compared to the baselineendoscopy (at initiation of MLN0002-2003); and continued reduction in CDAI that is a ≥70 point decrease from the baseline CDAI score at the initiation of MLN0002-2003.
3. Time to major IBD-related events (hospitalizations, surgeries, or procedures).
4. Changes from Baseline in IMPACT-III (where translations are available) total and subscale scores at Week 24 and every 24 weeks, thereafter.
5. Height velocity at Week 48 and every 48 weeks, thereafter.
6. Change from Baseline in height, weight, and body mass index (BMI) at Week 24 and every 24 weeks, thereafter.
7. Percentage of subjects achieving Tanner stage V at or before age 16 years (females) or 17 years (males).

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

Sponsor organisation

Takeda Development Center Americas Inc.

Address

95 Hayden Avenue

City

Lexington

Postcode

02421-7942

Country

United States

Scientific contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Public contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Third parties 9

Locations

2 EU/EEA countries · 6 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 27 files

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

Application history

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