A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Crohn's Disease (CD)

Crohn's Disease (CD) Clinical Trial

Official title:

A Randomized, Double-Blind, Phase 3 Study to Evaluate the Efficacy and Safety of Vedolizumab Intravenous as Maintenance Therapy in Pediatric Subjects With Moderately to Severely Active Crohn's Disease Who Achieved Clinical Response Following Open-Label Vedolizumab Intravenous Therapy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04779320
• Other study ID #: MLN0002-3025
• Secondary ID: 2020-004301-31jR
• Status: Recruiting
• Phase: Phase 3
• Start date: April 30, 2022
• Est. completion date: November 30, 2024

Study information

• Verified date: April 2024
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Vedolizumab is a medicine that helps to reduce inflammation and pain in the digestive system. In this study, children and teenagers with moderate to severe Crohn's disease will be treated with vedolizumab.

The main aim of the study is to check if participants achieve remission after treatment with the vedolizumab. Remission means symptoms improve or disappear and an endoscopy shows no signs of inflammation.

Participants will receive 3 infusions of vedolizumab over 6 weeks. Then, those who have a clinical response will receive either a high dose or low dose of vedolizumab once every 8 weeks. They will receive the same dose every time.

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 CD. The drug is tested and approved in adults in approximately 70 countries. Participants to be enrolled must have failed response to, lost response to, or been intolerant to at least 1 of the current standard of care (SOC) induction and maintenance therapies for CD including exclusive and/or partial enteral nutrition therapy, immunomodulators (e.g., azathioprine [AZA], 6-mercaptopurine [6-MP], methotrexate [MTX]), and tumor necrosis factor-alpha (TNF-α) antagonists.

The study will enroll approximately 120 patients.

During the Induction Period participants will receive 3 doses of vedolizumab IV infusion at Day 1, Week 2, and Week 6 based on their weight at Baseline as:

• Participants 10 to 15 kg, Vedolizumab 150 mg

• Participants >15 to <30 kg, Vedolizumab 200 mg

• Participants ≥30 kg, Vedolizumab 300 mg

At Week 14, participants who achieve clinical response will be randomly assigned (by chance, like flipping a coin) in a 1:1 ratio to one of the 2 double-blind dose groups (high dose and low dose), stratified by previous exposure/failure to TNF-α antagonists therapy or naive to TNF-α antagonists therapy, and by weight groups. Participants will receive vedolizumab IV infusions every 8 weeks (Q8W) up to Week 46 during the Maintenance Period as follows:

• Participants ≥30 kg, Vedolizumab 300 mg (High dose) or 150 mg (Low dose)

• Participants >15 to <30 kg, Vedolizumab 200 mg (High dose) 100 mg (Low dose)

• Participants 10 to 15 kg, Vedolizumab 150 mg (High dose) or 100 mg (Low dose)

The dose will remain blinded to the participant and study doctor during the study (unless there is an urgent medical need). All participants will be administered vedolizumab via IV infusion. In participants who demonstrate lack of maintenance of clinical response during the Maintenance Period the dose will be escalated in a blinded fashion to the high dose in their weight group based on the weight at the time of the worsening of disease. In addition one-time rescue therapy with corticosteroids is allowed during Maintenance Period.

This multi-center trial will be conducted worldwide. After the Week 54, participants may be eligible to continue receiving vedolizumab in extension study MLN0002-3029. Participants who do not maintain corticosteroid-free clinical response at week 54 will undergo an end-of-study (EOS) or ET visit, and a safety visit 18 weeks after the last dose of vedolizumab followed by 2 years of long term follow-up (up to 104 weeks), in addition these participants will then be eligible to enter study MLN0002-3029 for an observational LTFU period of 2 years after the last dose of study drug.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: November 30, 2024
• Est. primary completion date: November 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

Main Inclusion Criteria:

1. The participants has moderately to severely active CD, unresponsive or intolerant to their current standard of care (SOC).

2. The participants weigh =10 kg at the time of screening and enrollment into the study.

3. Participants with Crohn's disease (CD) diagnosed at least 1 month before screening. Participants with moderately to severely active CD defined by a Pediatric Crohn's Disease Activity Index (PCDAI) >30 and an 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.

4. 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)-a antagonist therapy (eg, infliximab, adalimumab). This includes participants who are dependent on corticosteroids or exclusive or partial enteral nutrition to control symptoms and who are experiencing worsening of disease in the moderate-to-severe range when attempting to wean off corticosteroids or discontinue exclusive enteral nutrition.

5. 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. Participants with vaccinations that are up-to-date based on the countrywide accepted schedule of childhood vaccines.

Main Exclusion Criteria:

1. Participants who have received either (1) an investigational biologic (other than those listed in Exclusion Criterion #1) within 60 days or 5 half-lives before screening (whichever is longer); or (2) an approved biologic or biosimilar agent within 2 weeks before the first dose of study drug or at any time during the screening period.

2. Participants with active cerebral/meningeal disease, signs/symptoms or history of progressive multifocal leukoencephalopathy (PML) or any other major neurological disorders including stroke, multiple sclerosis, brain tumor or neurodegenerative disease.

3. The participants had a clinically significant infection (eg, pneumonia, pyelonephritis, coronavirus disease 2019 [COVID-19]) within 30 days prior to first dose of study drug.

4. The participants has received any live vaccinations within 30 days prior to first dose.

5. Participants who currently require surgical intervention or are anticipated to require surgical intervention for CD during this study.

6. 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.

7. Participants with a current diagnosis of indeterminate colitis.

8. Participants with clinical features suggesting monogenic very early-onset inflammatory bowel disease.

9. Active or latent tuberculosis (TB), as evidenced by a diagnostic TB test performed within 30 days of screening or during the screening Period that is positive, defined as:

• Positive QuantiFERON test or 2 successive indeterminate QuantiFERON tests, OR

• A TB skin test reaction =5 mm.

10. Participants with evidence of positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). Hepatitis B virus (HBV) immune participants(i.e., hepatitis B surface antigen [HBsAg]-negative and hepatitis B antibody-positive) may, however, be included.

Note: If a participant tests negative for HBsAg, but positive for HBcAb, the participant would be considered eligible if the absence of HBV DNA is confirmed by HBV DNA polymerase chain reaction reflex testing performed in the central laboratory.

11. Participants with chronic hepatitis C virus (HCV) (ie, positive HCV antibody [HCVAb] and HCV RNA).

Note: Participants who are HCVAb-positive without evidence of HCV RNA may be considered eligible (spontaneous viral clearance or previously treated and cured [defined as no evidence of HCV RNA at least 12 weeks before baseline]).

12. The participants has any identified congenital or acquired immunodeficiency (eg, common variable immunodeficiency, human immunodeficiency virus [HIV] infection, organ transplantation).

13. The participant has evidence of dysplasia or history of malignancy other than a successfully treated non-metastatic cutaneous squamous cell or basal cell carcinoma or localized carcinoma in situ of the cervix.

14. Participants with positive stool studies for ova and/or parasites or stool culture at screening visit.

15. Participants with positive Clostridioides difficile (C difficile) stool test at screening visit.

Other inclusion/exclusion criteria may apply.

Related Conditions & MeSH terms

• Crohn Disease
• Crohn's Disease (CD)

Intervention

Drug:
• Vedolizumab IV
Vedolizumab IV

Locations

Country	Name	City	State
Australia	Monash Health, Monash Medical Centre	Clayton	Victoria
Australia	Royal Children's Hospital Melbourne - PIN	Parkville	Victoria
Australia	Queensland Childrens Hospital	South Brisbane	Queensland
Australia	Children's Hospital at Westmead	Westmead	New South Wales
Belgium	UZ Antwerpen	Edegem	Antwerpen
Belgium	Universitair Ziekenhuis Brussel - PIN	Jette	Brussels
Belgium	UZ Leuven	Leuven	Vlaams Brabant
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	London Health Sciences Centre	London	Ontario
Canada	Centre Hospitalier Universitaire Sainte-Justine	Montreal	Quebec
Canada	British Columbia Children's Hospital	Vancouver	British Columbia
China	Beijing Children Hospital,Capital Medical University	Beijing	Beijing
China	The Children's Hospital Zhejiang UniversitySchool of Medicine	Hangzhou	Zhejiang
China	Children's Hospital of Fudan University	Shanghai	Shanghai
China	Henan Children's Hospital(Zhengzhou Children's Hospital)	Zhengzhou	Henan
Croatia	University Hospital Centre Split	Split
Croatia	Klinika Za Djecje Bolesti Zagreb	Zagreb	Grad Zagreb
Croatia	University Hospital Center Zagreb	Zagreb	Grad Zagreb
Czechia	Fakultni nemocnice Ostrava	Ostrava
Czechia	Fakultni nemocnice Kralovske Vinohrady	Prague	Praha, Hlavni Mesto
Czechia	Fakultni Thomayerova Nemocnice	Praha	Praha, Hlavni Mesto
Greece	Attikon University General Hospital	Athens	Attiki
Greece	Children's Hospital "Agia Sofia"	Athens
Greece	Ippokratio General Hospital of Thessaloniki	Thessaloniki
Greece	Ippokratio General Hospital of Thessaloniki	Thessaloniki
Hungary	Semmelweis Egyetem	Budapest
Hungary	Borsod-Abauj-Zemplen Megyei Kozponti Korhaz es Egyetemi Oktato Korhaz	Miskolc	Borsod-Abauj-Zemplen
Hungary	Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont	Szeged	Csongrad
Israel	Soroka University Medical Centre	Beer Sheba
Israel	Carmel Medical Center	Haifa
Israel	Rambam Medical Center - PPDS	Haifa
Israel	Hadassah Medical Center - PPDS	Jerusalem
Israel	Shaare Zedek Medical Center	Jerusalem
Israel	Tel Aviv Sourasky Medical Center PPDS	Jerusalem	Yerushalayim
Israel	Schneider Childrens Medical Center of Israel Petah Tikvah PIN	Petah Tikva	HaMerkaz
Italy	Azienda USL di Bologna	Bologna	Emilia-Romagna
Italy	ASST di Monza - Azienda Ospedaliera San Gerardo	Monza	Lombardia
Italy	AOU dell'Universita degli Studi della Campania Luigi Vanvitelli	Napoli	Campania
Italy	Azienda Ospedaliera Universitaria Federico II	Napoli	Campania
Italy	Universita degli Studi di Padova	Padova	Veneto
Italy	Sapienza University of Rome	Roma	Lazio
Japan	Juntendo University Hospital	Bunkyo-Ku	Tokyo
Japan	Japanese Red Cross Kumamoto Hospital	Kumamoto-shi	Kumamoto
Japan	Kurume University Hospital	Kurume-Shi	Hukuoka
Japan	National Center for Child Health and Development	Setagaya-Ku	Tokyo
Korea, Republic of	Kyungpook National University Chilgok hospital	Daegu	Daegu Gwang'yeogsi
Korea, Republic of	Gachon University Gil Medical Center	Incheon	Incheon Gwang'yeogsi
Korea, Republic of	Seoul National University Hospital	Seongnam
Korea, Republic of	Samsung Medical Center - PPDS	Seoul
Lithuania	Hospital of Lithuanian University of Health Sciences Kaunas Clinics	Kaunas	Kauno Apskritis
Lithuania	Vilnius University Hospital Santaros Klinikos	Vilnius	Vilniaus Apskritis
Poland	Copernicus Podmiot Leczniczy Sp. z o.o.	Gdansk	Pomorskie
Poland	Gornoslaskie Centrum Zdrowia Dziecka Im. Sw. Jana Pawla II Spsk Nr 6 Sum W Katowicach	Katowice	Slaskie
Poland	Uniwersytecki Szpital Dzieciecy	Krakow	Malopolskie
Poland	Instytut Centrum Zdrowia Matki Polki	Lodz	Lodzkie
Poland	SPZOZ Centralny Szpital Kliniczny UM w Lodzi	Lodz
Poland	Korczowski Bartosz, Gabinet Lekarski	Rzeszow	Podkarpackie
Poland	Twoja Przychodnia SCM	Szczecin	Zachodniopomorskie
Poland	Instytut Pomnik Centrum Zdrowia Dziecka	Warszawa	Mazowieckie
Poland	WIP Warsaw IBD Point Profesor Kierkus	Warszawa	Mazowieckie
Poland	Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu	Wroclaw	Dolnoslaskie
Slovakia	Detska fakultna nemocnica s poliklinikou Banska Bystrica	Banska Bystrica
Slovakia	Narodny ustav detskych chorob	Bratislava
Spain	Hospital Sant Joan de Deu - PIN	Esplugues de Llobregat	Barcelona
Spain	Hospital Infantil Universitario Nino Jesus - PIN	Madrid
Spain	Hospital Regional Universitario de Malaga - Hospital Materno Infantil	Malaga
Spain	Hospital de Sagunto	Sagunto	Valencia
Spain	Hospital Universitario Virgen del Rocio - PPDS	Sevilla
United Kingdom	Birmingham Children's Hospital NHS Foundation Trust	Birmingham	West Midlands
United Kingdom	Noahs Ark Childrens Hospital for Wales - PPDS - PIN	Cardiff	South Glamorgan
United Kingdom	Barts Health NHS Trust	London
United Kingdom	Great Ormond Street Hospital (GOSH)	London	London, City Of
United Kingdom	Kings College Hospital	London	London, City Of
United Kingdom	Royal Manchester Children's Hospital - PPDS	Manchester
United States	Childrens Center For Digestive Healthcare	Atlanta	Georgia
United States	Johns Hopkins University	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital For Children	Indianapolis	Indiana
United States	I.H.S Health LLC	Kissimmee	Florida
United States	Cedars Sinai Medical Center	Los Angeles	California
United States	MNGI Digestive Health, PA	Minneapolis	Minnesota
United States	Goryeb Children's Hospital	Morristown	New Jersey
United States	The Steven and Alexandra Cohen Childrens Medical Center of New York - BRANY - PPDS	New Hyde Park	New York
United States	Advocate Children's Hospital Park Ridge	Park Ridge	Illinois
United States	Phoenix Childrens Hospital	Phoenix	Arizona
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Hasbro Children's Hospital	Providence	Rhode Island
United States	Carilion Children's Tanglewood Center	Roanoke	Virginia
United States	Mayo Clinic - PIN	Rochester	Minnesota
United States	University of Rochester Medical Center PPDS	Rochester	New York
United States	Rady Childrens Hospital San Diego - PIN	San Diego	California
United States	University of California San Francisco	San Francisco	California
United States	Stony Brook University Medical Center	Stony Brook	New York
United States	SUNY Upstate Medical Center	Syracuse	New York

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  China,  Croatia,  Czechia,  Greece,  Hungary,  Israel,  Italy,  Japan,  Korea, Republic of,  Lithuania,  Poland,  Slovakia,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants With Clinical Remission at Week 54 Based on Pediatric Crohn's Disease Activity Index (PCDAI) Score =10	Clinical remission is defined by PCDAI score =10. The PCDAI was specifically designed for use in children. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: hematocrit (HCT) (adjusted for age and sex), erythrocyte sedimentation rate (ESR), and albumin level. The PCDAI score ranges from 0 to 100, with higher scores indicating more active disease. A score of <10 will be consistent with inactive disease, 11 to 30 will indicate mild disease, and >30 will indicate moderate to severe disease. A decrease of 12.5 points is taken as evidence of improvement.	Week 54
Primary	Percentage of Participants With Endoscopic Response at Week 54 Based on Simple Endoscopic Score for Crohn's Disease [SES-CD] Score	Endoscopic response is defined as at least a 50% reduction in SES-CD score from Baseline. The overall SES-CD score ranges from 0 to 56 and is the sum of 4 variables (ie, size of ulcers [cm], ulcerated surface, affected surface [%], and presence of narrowing) across 5 bowel segments (ie, rectum, descending and sigmoid colon, transverse colon, ascending colon, and ileum). Each variable is coded from 0 to 3 based on severity, where 0 is none or not severe and 3 is the most severe case, with the sum of the scores for each variable ranging from 0 to 15, except for presence of narrowing. Presence of narrowing ranges from 0 to 11 since a severity of 3 represents a narrowing which a colonoscope cannot be passed and, thus, can only be observed once among the bowel segments. The segmental SES-CD score is the sum of the 4 variables for each bowel segment and can range from 0 to 12, where each individual variable score ranges from 0 to 3.	Week 54
Secondary	Percentage of Participants with Clinical and Endoscopic Remission at Week 14 Based on Both PCDAI Score and SES-CD Score	Clinical and endoscopic remission is where participant achieves both clinical and endoscopic remission. Clinical remission is defined by PCDAI score =10. Endoscopic remission is defined by SES-CD score of =4 with at least a 2-point reduction from Baseline and no sub-score >1. PCDAI includes child-specific item: height velocity variable as well as three laboratory parameters: HCT, ESR, albumin level. PCDAI score ranges from 0 to 100, with higher scores indicating more active disease. The SES-CD score ranges from 0 to 56 and is the sum of 4 variables, size of ulcers [cm], ulcerated surface, affected surface [%], and presence of narrowing) across 5 bowel segments (ie, rectum, descending and sigmoid colon, transverse colon, ascending colon, and ileum), where higher scores indicate more severe disease.	Week 14
Secondary	Percentage of Participants with Clinical and Endoscopic Remission at Week 54 Based on Both PCDAI Score and SES-CD Score	Clinical and endoscopic remission is where participant achieves both clinical and endoscopic remission. Clinical remission is defined by PCDAI score =10. Endoscopic remission is defined by SES-CD score of =4 with at least a 2-point reduction from Baseline and no sub-score >1. PCDAI includes child-specific item: height velocity variable as well as three laboratory parameters: HCT, ESR, albumin level. PCDAI score ranges from 0 to 100, with higher scores indicating more active disease. The SES-CD score ranges from 0 to 56 and is the sum of 4 variables, size of ulcers [cm], ulcerated surface, affected surface [%], and presence of narrowing) across 5 bowel segments (ie, rectum, descending and sigmoid colon, transverse colon, ascending colon, and ileum), where higher scores indicate more severe disease.	Week 54
Secondary	Percentage of Participants with Sustained Clinical and Endoscopic Remission at Week 54	Sustained clinical and endoscopic remission is where a participant achieved clinical and endoscopic remission based on PCDAI and SES-CD scores at Weeks 14 and 54. Clinical remission is defined by PCDAI score =10. Endoscopic remission is defined as =4 with at least a 2-point reduction from Baseline and no sub-score >1 by SES-CD. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score ranges from 0 to 100, with higher scores indicating more active disease. The SES-CD score ranges from 0 to 56 and is the sum of 4 variables, size of ulcers [cm], ulcerated surface, affected surface [%], and presence of narrowing) across 5 bowel segments (ie, rectum, descending and sigmoid colon, transverse colon, ascending colon, and ileum), where higher scores indicate more severe disease.	Week 54
Secondary	Percentage of Participants with Corticosteroid-free Remission at Week 54 Based on PCDAI Score	Corticosteroid-free clinical remission is where participants achieves corticosteroid-free clinical remission based on PCDAI at Week 54 and has been off corticosteroids at least 12 weeks prior to and at Week 54. Clinical remission is defined by PCDAI score =10. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score ranges from 0 to 100, with higher scores indicating more active disease.	Week 54
Secondary	Percentage of Participants with Sustained Endoscopic Remission Based on SES-CD Score	Sustained endoscopic remission is where participants achieves endoscopic remission based on SES-CD =4 with at least a 2-point reduction from Baseline and no sub-score >1. The SES-CD evaluates 4 endoscopic variables (Size of ulcers, Ulcerated surface, Affected surface and Presence of narrowing). The score for each endoscopic variable is sum of values obtained for each segment. The SES-CD total is the sum of the four endoscopic variable scores from 0 to 56, where higher scores indicate more severe disease.	Week 14
Secondary	Percentage of Participants with Sustained Endoscopic Remission Based on SES-CD Score	Sustained endoscopic remission is where participants achieves endoscopic remission based on SES-CD =4 with at least a 2-point reduction from Baseline and no sub-score >1. The SES-CD evaluates 4 endoscopic variables (Size of ulcers, Ulcerated surface, Affected surface and Presence of narrowing). The score for each endoscopic variable is sum of values obtained for each segment. The SES-CD total is the sum of the four endoscopic variable scores from 0 to 56, where higher scores indicate more severe disease.	Week 54
Secondary	Percentage of Participants with Sustained Clinical Remission at Week 14 Based on PCDAI Score	Sustained Clinical Remission is where participants will achieve clinical remission based on PCDAI at Weeks 14 and 54. Clinical remission is defined by PCDAI score =10. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score will range from 0 to 100, with higher scores indicating more active disease.	Week 14
Secondary	Percentage of Participants with Sustained Clinical Remission at Week 54 Based on PCDAI Score	Sustained Clinical Remission is where participants will achieve clinical remission based on PCDAI at Weeks 14 and 54. Clinical remission is defined by PCDAI score =10. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score will range from 0 to 100, with higher scores indicating more active disease.	Week 54
Secondary	Serum Trough Concentrations of Vedolizumab Over Time		Predose and postdose at multiple time points (up to 54 weeks)
Secondary	Percentage of Participants With Positive Antivedolizumab Antibodies		Pre-dose (up to 54 weeks)
Secondary	Percentage of Participants With Positive Neutralizing Antivedolizumab Antibody Titers		Pre-dose (up to 54 weeks)
Secondary	Sustained Clinical Response at Week 14 Based on PCDAI Score	Sustained clinical response is where a participant achieve clinical response based on PCDAI score =30 and reduction of the PCDAI by =15 points from Baseline. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score will range from 0 to 100, with higher scores indicating more active disease.	Week 14
Secondary	Sustained Clinical Response at Week 54 Based on PCDAI Score	Sustained clinical response is where a participant achieve clinical response based on PCDAI score =30 and reduction of the PCDAI by =15 points from Baseline. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score will range from 0 to 100, with higher scores indicating more active disease.	Week 54
Secondary	Percentage of Participants with Clinical Remission at Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54	Clinical remission is defined by PCDAI score < 10. The PCDAI was specifically designed for use in children. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT (adjusted for age and sex), ESR, and albumin level. The PCDAI score will range from 0 to 100, with higher scores indicating more active disease.	Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Secondary	Percentage of Participants with Change in Baseline in Clinical Response at Weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54	Clinical Response is where participants achieves clinical response if PCDAI =30 with reduction in the PCDAI of =15 points from Baseline. The PCDAI includes a child-specific item: the height velocity variable as well as three laboratory parameters: HCT, ESR, and albumin level. The PCDAI score will range from 0 to 100, with higher scores indicating more active disease.	Baseline, weeks 2, 6, 10, 14, 22, 30, 38, 46, and 54
Secondary	Percentage of Participants with at Least One Adverse Event (AE), Serious Adverse Event (SAE), and AE of special interest (AESI)	An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have causal relationship with this treatment. AE can be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug whether it is considered related to drug. SAE is any untoward medical occurrence that at any dose: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to congenital anomaly/birth defect and/or is important medical event. An AESI (serious or non-serious) is one of scientific and medical concern specific to compound or program, for which ongoing monitoring and rapid communication by investigator. AESIs include- opportunistic infection, such as progressive multifocal leukoencephalopathy (PML), liver injury, malignancies, infusion-related reactions, hypersensitivity.	From first dose of study drug before each dose on dosing days through the Week 72
Secondary	Change from Baseline in Weight	Change from in Baseline in weight will be calculated as: Weight at each study visit (up to Week 54) - Weight at Baseline.	Baseline up to Week 54
Secondary	Change from Baseline in Linear Growth Z-score	Linear growth Z-score will be calculated as: Z-score = (observed value - median value of the reference population)/ standard deviation value of reference population.	Baseline up to Week 54
Secondary	Change from Baseline in Tanner Stages at Week 54	Tanner Stage is used to define physical measurements of sexual development based on external primary and secondary sex characteristics. Female and male participants are evaluated for breast development and genital development respectively and both genders for pubic hair distribution based on a 5-stage scale ranging from Stage I (prepubertal/preadolescent characteristics) to Stage V (mature or adult characteristics).	Week 54

External Links

•   To obtain more information about this study, click this link.