Clinical trial NCT05442567: A Study of Vedolizumab in Children With Ulcerative Colitis (UC) or Crohn's Disease (CD)
Official title: A Phase 3b Extension Study to Evaluate the Long-term Safety of Vedolizumab Intravenous in Pediatric Patients With Ulcerative Colitis or Crohn's Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Ulcerative Colitis
Crohn's Disease
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2022-07-05
Enrollment: 240
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The study is an extension of two parent studies (MLN0002-3024 \[NCT04779307\] and MLN0002-3025 \[NCT04779320\]). Participants must have participated in one of the previous studies. The purpose of this study is to collect the long-term safety of vedolizumab in children with UC or CD.

Detailed description: This multi-center trial is conducted worldwide. Up to 240 patients would be enrolled from Studies MLN0002-3024 \[participants with UC\] and MLN0002-3025 \[participants with CD\], either in the Treatment Cohort or in the Observational Cohort. Approximately 93 participants who have previously participated either in study MLN0002-3024 or MLN0002-3025, referred to as parent study, are expected to roll over to the MLN0002-3029 study in the treatment cohort.

Treatment Cohort:

The drug being tested in this study is called vedolizumab, being studied to treat pediatric patients who have UC or CD.

Participants eligible for the Treatment Cohort can be administered vedolizumab intravenous (IV) at Week 54 visit of parent study or up to 1 week after Week 54 of the parent study based on the availability of test results needed to assess eligibility of the participant. At this study entry, participants will be administered the same blinded dose of vedolizumab IV that was received at Week 46 in the parent study and will then continue to receive vedolizumab IV at a frequency of once every 8 weeks (Q8W) in the following treatment groups:

* Participants 10 to ≤15 kilogram (kg), Vedolizumab 150 milligram (mg) (High dose)
* Participants 10 to ≤15 kg, Vedolizumab 100 mg (Low dose)
* Participants \>15 to \<30 kg, Vedolizumab 200 mg (High dose)
* Participants \>15 to \<30 kg, Vedolizumab 100 mg (Low dose)
* Participants ≥30 kg, Vedolizumab 300 mg (High dose)
* Participants ≥30 kg, Vedolizumab 150 mg (Low dose)

Blinding of dose group assignment of the parent study will continue until the respective parent study is unblinded in order to protect the blinding of the parent study.

The overall time to participate in the Treatment Cohort of this study is up to participant withdrawal, or until vedolizumab IV is commercially available for pediatric indication(s) in the participant's country or until other drug access programs become available, or Sponsor's decision for study closure, or for up to approximately 5 years, whichever comes first. Participants who complete or are discontinued from the study for any reason will complete the final safety/end of study (EOS) visit 18 weeks after their last dose of study drug.

Observational Cohort:

Participants who received at least 1 dose of study drug during parent study and early terminated or are not eligible for the Treatment Cohort of this study after completion of the Week 54 visit of parent study, will be enrolled in the Observational Cohort of this study as part of a long-term follow-up period to assess prespecified safety events of interest and will not receive continued treatment with vedolizumab IV.

The overall time to participate in the Observational Cohort is up to approximately 2 years.

Eligibility criteria: Main Inclusion Criteria:

For Treatment Cohort:

1. The participant should have completed Study MLN0002-3024 or Study MLN0002-3025 and 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 participants with UC, or by a decrease of pediatric Crohn's disease activity index (PCDAI) of ≥15 points for participants with CD and with total PCDAI ≤30.
2. A male participant who is sexually active with a female partner of childbearing potential agrees to use a barrier method of contraception (e.g., condom with or without spermicide) from signing of participant/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 participant should also be advised to use a highly effective method of contraception.
3. A female participant of childbearing potential who is sexually active with a nonsterilized male partner agrees to use a highly effective method of contraception from signing of participant/parental informed consent and/or pediatric assent throughout the duration of the study and 18 weeks after the last dose.

For Observational Cohort:

1. The participant 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.

Main Exclusion Criteria:

For Treatment Cohort only:

1. The participant currently requires major surgical intervention for UC or CD (e.g., bowel resection), or is anticipated to require major surgical intervention for UC or CD during the study.
2. The participant 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.
3. The participant has other serious comorbidities that will limit their ability to complete the study.
4. The participant is unable to comply with all study assessments.
5. The participant has hypersensitivity or allergies to any of the vedolizumab excipients.
6. The participant is lactating or pregnant.

Primary outcomes:
measure: Treatment Cohort: Number of Participants With at Least One Adverse Event (AE)
timeFrame: From first dose of study drug up to approximately 5 years
measure: Observational Cohort: Number of Participants With Prespecified Safety Events
timeFrame: Up to approximately 2 years
Secondary outcomes:
measure: Treatment Cohort: Time to Major Inflammatory Bowel Disease (IBD)-related Events
timeFrame: Up to approximately 5 years
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Total Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Bowel Symptom Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Systemic Symptom Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Social Functioning Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Body Image Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Treatment/Intervention Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
measure: Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Emotional Functioning Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
timeFrame: Baseline, every 24 weeks in this study (up to approximately 5 years)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Childrens Hospital -1919 E Thompson Rd
state: Arizona
city: San Diego
country: United States
facility: Rady Childrens Hospital San Diego - PIN
state: California
city: Atlanta
country: United States
facility: Childrens Center For Digestive Healthcare
state: Georgia
city: Park Ridge
country: United States
facility: Advocate Children's Hospital Park Ridge
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: MNGI Digestive Health PA-Plymouth
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic - PIN
state: Minnesota
city: Morristown
country: United States
facility: Goryeb Children's Hospital
state: New Jersey
city: New Hyde Park
country: United States
facility: The Steven and Alexandra Cohen Childrens Medical Center of New York - BRANY - PPDS
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Roanoke
country: United States
facility: Carilion Children's Tanglewood Center
state: Virginia
city: Westmead
country: Australia
facility: Children's Hospital at Westmead
state: New South Wales
city: South Brisbane
country: Australia
facility: Queensland Childrens Hospital
state: Queensland
city: Clayton
country: Australia
facility: Monash Health, Monash Medical Centre
state: Victoria
city: Parkville
country: Australia
facility: Royal Children's Hospital Melbourne - PIN
state: Victoria
city: Edegem
country: Belgium
facility: UZ Antwerpen
state: Antwerpen
city: Jette
country: Belgium
facility: Universitair Ziekenhuis Brussel - PIN
state: Brussels
city: Leuven
country: Belgium
facility: UZ Leuven
state: Vlaams Brabant
city: Edmonton
country: Canada
facility: University of Alberta Hospital
state: Alberta
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
city: Beijing
country: China
facility: Beijing Children Hospital,Capital Medical University
state: Beijing
city: Zhengzhou
country: China
facility: Henan Children's Hospital (Zhengzhou Children's Hospital)
state: Henan
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
city: Hangzhou
country: China
facility: The Children's Hospital Zhejiang UniversitySchool of Medicine
state: Zhejiang
city: Zagreb
country: Croatia
facility: Klinika Za Djecje Bolesti Zagreb
state: Grad Zagreb
city: Athens
country: Greece
facility: Attikon University General Hospital
state: Attiki
city: Athens
country: Greece
facility: Children's Hospital "Agia Sofia"
city: Thessaloniki
country: Greece
facility: Ippokratio General Hospital of Thessaloniki
city: Miskolc
country: Hungary
facility: Borsod-Abauj-Zemplen Megyei Kozponti Korhaz es Egyetemi Oktato Korhaz
state: Borsod-Abauj-Zemplen
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Petah Tikva
country: Israel
facility: Schneider Childrens Medical Center of Israel Petah Tikvah PIN
state: HaMerkaz
city: Jerusalem
country: Israel
facility: Hadassah Medical Center - PPDS
state: Yerushalayim
city: Haifa
country: Israel
facility: Rambam Medical Center - PPDS
city: Haifa
country: Israel
facility: Carmel Medical Center
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Tel-Aviv
country: Israel
facility: Tel Aviv Sourasky Medical Center PPDS
city: Napoli
country: Italy
facility: AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
state: Campania
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
state: Campania
city: Bologna
country: Italy
facility: Azienda USL di Bologna
state: Emilia-Romagna
city: Roma
country: Italy
facility: Sapienza University of Rome
state: Lazio
city: Monza
country: Italy
facility: Fondazione IRCCS San Gerardo dei Tintori - ASST di Monza A. O. San Gerardo
state: Lombardia
city: Padova
country: Italy
facility: Universita degli Studi di Padova
state: Veneto
city: Kurume-Shi
country: Japan
facility: Kurume University Hospital
state: Hukuoka
city: Kumamoto-shi
country: Japan
facility: Japanese Red Cross Kumamoto Hospital
state: Kumamoto
city: Bunkyo-Ku
country: Japan
facility: Juntendo University Hospital
state: Tokyo
city: Setagaya-Ku
country: Japan
facility: National Center for Child Health and Development
state: Tokyo
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Chilgok hospital
state: Daegu Gwang'yeogsi
city: Incheon
country: Korea, Republic of
facility: Gachon University Gil Medical Center
state: Incheon Gwang'yeogsi
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center - PPDS
city: Lodz
country: Poland
facility: Instytut Centrum Zdrowia Matki Polki
state: Lodzkie
city: Krakow
country: Poland
facility: Uniwersytecki Szpital Dzieciecy
state: Malopolskie
city: Warszawa
country: Poland
facility: WIP Warsaw IBD Point Profesor Kierkus
state: Mazowieckie
city: Warszawa
country: Poland
facility: Instytut Pomnik Centrum Zdrowia Dziecka
state: Mazowieckie
city: Rzeszow
country: Poland
facility: Korczowski Bartosz, Gabinet Lekarski
state: Podkarpackie
city: Katowice
country: Poland
facility: Gornoslaskie Centrum Zdrowia Dziecka Im. Sw. Jana Pawla II Spsk Nr 6 Sum W Katowicach
state: Slaskie
city: Szczecin
country: Poland
facility: Twoja Przychodnia SCM
state: Zachodniopomorskie
city: Lodz
country: Poland
facility: SPZOZ Centralny Szpital Kliniczny UM w Lodzi
city: Bratislava
country: Slovakia
facility: Narodny ustav detskych chorob
city: London
country: United Kingdom
facility: Great Ormond Street Hospital (GOSH)
state: London, City Of
city: Cardiff
country: United Kingdom
facility: Noahs Ark Childrens Hospital for Wales - PPDS - PIN
state: South Glamorgan
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital NHS Foundation Trust
state: West Midlands
city: London
country: United Kingdom
facility: Barts Health NHS Trust
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital - PPDS
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: