Clinical trial NCT05995353: A Study to Assess Adverse Events, Change in Disease Activity, and How Intravenous and Subcutaneous Risankizumab Moves Through the Body of Pediatric Participants With Moderately to Severely Active Crohn's Disease
Official title: A Phase 3, Multi-Center Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Risankizumab With Open-Label Induction, Randomized Double-Blind Maintenance, and Long-Term Extension Periods in Pediatric Subjects (2 to < 18 Years of Age) With Moderately to Severely Active Crohn's Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Crohn's Disease
Keywords: Crohn's Disease
Risankizumab
Skyrizi
Pediatrics
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2023-08-16
Enrollment: 110
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: Crohn's Disease (CD) is a gastrointestinal disease that can cause chronic diarrhea with or without gross bleeding, abdominal pain, weight loss, and fever. This study will assess the pharmacokinetics, efficacy, and safety of risankizumab in pediatric participants with moderately to severely active CD aged 2 to \< 18 years old who have had intolerance or inadequate response to other therapies.

Risankizumab is an approved drug for adults with plaque psoriasis, psoriatic arthritis, and CD and is being developed for the treatment of CD in pediatrics. This study is comprised of 3 cohorts that may participate in 3 substudies (SS). Cohort 1 will enroll participants with ages from 6 to less than 18 years. Cohort 2 will enroll participants with ages from 2 to less than 6 years. Cohort 3 will enroll participants with ages from 2 to less than 18 years. SS1 is an open-label induction period where participants will receive a weight-based induction regimen of risankizumab. SS2 is a double-blind maintenance period where participants will be randomized to receive 1 of 2 doses of weight-based induction regimen of risankizumab. SS3 is an open-label extension period where participants will receive risankizumab based off of their response in SS2. Around 110 pediatric participants with CD will be enrolled at around 100 sites worldwide.

Participants in SS1 will receive risankizumab intravenously during the 12-week induction period. Participants in SS2 will receive risankizumab subcutaneously during the 52-week randomized maintenance period. Participants in SS3 will receive risankizumab subcutaneously during the 208-week open label period. Participants will be followed-up for approximately 140 days.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.


Eligibility criteria: Inclusion Criteria:

* Pediatric individuals, 2 to \< 18 years old
* Must have moderately to severely active CD, as defined by the PCDAI score \> 30 assessed at Baseline
* Must have endoscopic evidence of mucosal inflammation as documented by the SES-CD of ≥ 6 for ileocolonic or colonic disease (or SES-CD of ≥ 4 for isolated ileal disease)
* Demonstrated intolerance or inadequate response to one or more of the following categories of drugs: aminosalicylates (This drug class is not sufficient for eligibility for subjects in France, Italy, Netherlands, Spain, and Sweden), oral locally acting corticosteroids, systemic steroids (prednisone or equivalent), IMMs, and/or biologic therapies

Exclusion Criteria:

* History of hereditary fructose intolerance (a rare genetic condition) or an allergic reaction or significant sensitivity to constituents of the study drug (and its excipients) and/or other products in the same class
* Any of the following medical disorders:

  1. Current diagnosis of ulcerative colitis, indeterminate colitis, or monogenic IBD.
  2. A diagnosis of CD prior to 2 years of age.
  3. A diagnosis or suspected diagnosis of a primary immunodeficiency.
  4. Currently known complications of CD such as:

     * Active abscess (abdominal or perianal);
     * Symptomatic bowel strictures;
     * \> 2 missing segments of the following 5 segments: terminal ileum, right colon, transverse colon, sigmoid and left colon, and rectum;
     * Fulminant colitis;
     * Toxic megacolon;
     * Or any other manifestation that might require surgery while enrolled in the study.
  5. Ostomy or ileoanal pouch.
  6. Diagnosis of short gut or short bowel syndrome.
  7. Surgical bowel resection within the past 3 months prior to Baseline (excluding gastrointestinal surgeries which are not bowel resections such as appendectomy or ostomy closure), or a history of \>3 bowel resections.

Primary outcomes:
measure: Cohort 3 (Substudy 2): Percentage of Participants Achieving Pediatric Crohn's Disease Activity Index (PCDAI) Clinical Remission
timeFrame: At 64 weeks
measure: Cohort 3 (Substudy 2): Percentage of Participants Achieving Endoscopic Response per Simple Endoscopic Score for Crohn's Disease (SES-CD)
timeFrame: At 64 weeks
measure: Cohorts 1 & 2: Maximum Observed Serum Concentration (Cmax) of Risankizumab
timeFrame: Up to approximately Week 64
measure: Cohorts 1 & 2: Time to Cmax (Tmax) of Risankizumab
timeFrame: Up to approximately 64 weeks
measure: Cohorts 1 & 2: Area Under the Serum Concentration-Time Curve Over the Dosing Interval (AUCtau) of Risankizumab
timeFrame: Up to approximately 64 weeks
Secondary outcomes:
measure: Cohort 3 (Substudy 1): Percentage of Participants Achieving PCDAI Clinical Remission
timeFrame: At 12 weeks
measure: Cohort 3 (Substudy 1): Percentage of Participants Achieving Endoscopic Response per SES-CD
timeFrame: At 12 weeks
measure: Cohort 3 (Substudy 1): Percentage of Participants Achieving Endoscopic Remission per SES-CD
timeFrame: At 12 weeks
measure: Cohort 3 (Substudy 2): Percentage of Participants Achieving Endoscopic Remission per SES-CD
timeFrame: At 64 weeks
measure: Cohort 3 (Substudy 2): Percentage of Participants Achieving Corticosteroid-Free Clinical Remission per PCDAI
timeFrame: At 64 weeks
measure: Cohorts 1 & 2 (Substudy 2): Percentage of Participants Achieving PCDAI Clinical Remission
timeFrame: At 64 weeks
measure: Cohorts 1 & 2 (Substudy 2): Percentage of Participants Achieving Endoscopic Response per SES-CD
timeFrame: At 64 weeks
measure: Cohorts 1 & 2 (Substudy 1): Percentage of Participants Achieving PCDAI Clinical Remission
timeFrame: At 12 weeks
measure: Cohorts 1 & 2 (Substudy 1): Percentage of Participants Achieving Endoscopic Response per SES-CD
timeFrame: At 12 weeks
measure: Cohorts 1 & 2 (Substudy 1): Percentage of Participants Achieving Endoscopic Remission per SES-CD
timeFrame: At 12 weeks
measure: Cohorts 1 & 2 (Substudy 2): Percentage of Participants Achieving Endoscopic Remission per SES-CD
timeFrame: At 64 weeks
measure: Cohorts 1 & 2 (Substudy 2): Percentage of Participants Achieving Corticosteroid-Free Clinical Remission per PCDAI
timeFrame: At 64 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital /ID# 255766
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital /ID# 255762
state: Arkansas
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital - Oakland /ID# 258327
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado - Aurora /ID# 255764
state: Colorado
city: Orlando
country: United States
facility: Arnold Palmer Hospital for Children Center Digestive Health & Nutrition - Orland /ID# 255437
state: Florida
city: Indianapolis
country: United States
facility: Indiana University Health Riley Hospital for Children /ID# 256454
state: Indiana
city: Boston
country: United States
facility: Massachusetts General Hospital /ID# 255767
state: Massachusetts
city: Minneapolis
country: United States
facility: MNGI Digestive Health, P. A. /ID# 255366
state: Minnesota
city: Morristown
country: United States
facility: Goryeb Children's Hospital /ID# 256452
state: New Jersey
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai /ID# 254880
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic - Cleveland /ID# 256453
state: Ohio
city: Edegem
country: Belgium
facility: Uza /Id# 255114
state: Antwerpen
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires UCL Saint-Luc /ID# 255108
state: Bruxelles-Capitale
city: Jette
country: Belgium
facility: Universitair Ziekenhuis Brussel /ID# 255109
state: Bruxelles-Capitale
city: Leuven
country: Belgium
facility: Universitair Ziekenhuis Leuven /ID# 255098
state: Vlaams-Brabant
city: Bruxelles
country: Belgium
facility: Hospital Universite Enfants Reine Fabiola /ID# 255112
city: Liege
country: Belgium
facility: Groupe Sante CHC - Clinique du MontLegia /ID# 255620
city: Plovdiv
country: Bulgaria
facility: UMHAT Sveti Georgi /ID# 255386
city: Sofiya
country: Bulgaria
facility: Specialized Hospital For Active Treatment Of Children Diseases Prof. Ivan Mitev /ID# 255384
city: Varna
country: Bulgaria
facility: UMHAT Sveta Marina /ID# 256358
city: Calgary
country: Canada
facility: Alberta Children's Hospital /ID# 255357
state: Alberta
city: Vancouver
country: Canada
facility: BC Children's Hospital /ID# 255359
state: British Columbia
city: London
country: Canada
facility: London Health Sciences Center- University Hospital /ID# 258598
state: Ontario
city: Beijing
country: China
facility: Beijing Children's Hospital /ID# 256081
state: Beijing
city: Beijing
country: China
facility: Peking University Third Hospital /ID# 255876
state: Beijing
city: Guangzhou
country: China
facility: Guangzhou Medical University Affiliated Women and Children's Medical Center /ID# 255428
state: Guangdong
city: Guangzhou
country: China
facility: The Sixth Affiliated Hospital of Sun Yat-sen University /ID# 270589
state: Guangdong
city: Zhengzhou
country: China
facility: Henan Children's Hospital Zhengzhou Children's Hospital /ID# 255562
state: Henan
city: Changsha
country: China
facility: Hunan Children's Hospital /ID# 255610
state: Hunan
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital /ID# 255564
state: Jiangxi
city: Shenyang
country: China
facility: Shengjing Hospital of China Medical University /ID# 255563
state: Liaoning
city: Shanghai
country: China
facility: Children's Hospital of Shanghai /ID# 255531
state: Shanghai
city: Shanghai
country: China
facility: Ruijin Hospital, Shanghai Jiaotong University School of Medicine /ID# 255688
state: Shanghai
city: Prague
country: Czechia
facility: Fakultní nemocnice v Motole /ID# 256547
state: Praha 5
city: Praha
country: Czechia
facility: Vseobecna fakultni nemocnice v Praze /ID# 256096
city: Tours
country: France
facility: CHRU Tours - Hopital Gatien de Clocheville /ID# 255052
state: Centre-Val De Loire
city: Bordeaux
country: France
facility: CHU Bordeaux - Hopital Pellegrin /ID# 257060
state: Nouvelle-Aquitaine
city: Bron CEDEX
country: France
facility: Hospices Civils de Lyon - Hôpital Femme Mère Enfant /ID# 255443
state: Rhone
city: Paris
country: France
facility: AP-HP - Hopital Necker /ID# 255608
city: Toulouse
country: France
facility: CHU Toulouse - Hopital Paule de Viguier /ID# 255609
city: Muenchen
country: Germany
facility: Dr. von Haunerschen Kinderspital /ID# 255577
state: Bayern
city: Muenster
country: Germany
facility: Universitaetsklinikum Muenster /ID# 256762
state: Nordrhein-Westfalen
city: Petah Tikva
country: Israel
facility: Schneider Children's Medical Center /ID# 254950
state: HaMerkaz
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center /ID# 254951
state: Yerushalayim
city: Genoa
country: Italy
facility: IRCCS Istituto Giannina Gaslini /ID# 255262
state: Genova
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II /ID# 255045
state: Napoli
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria Gaetano Martino /ID# 255044
city: Obu
country: Japan
facility: Aichi Children'S Health And Medical Center /ID# 272085
state: Aichi
city: Kashiwa-shi
country: Japan
facility: Tsujinaka Hospital Kashiwanoha /ID# 268409
state: Chiba
city: Kurume-shi
country: Japan
facility: Kurume University Hospital /ID# 268418
state: Fukuoka
city: Maebashi-shi
country: Japan
facility: Gunma University Hospital /ID# 270560
state: Gunma
city: Kumamoto-shi
country: Japan
facility: Japanese Red Cross Kumamoto Hospital /ID# 268586
state: Kumamoto
city: Izumi-Shi
country: Japan
facility: Osaka Women's and Children's Hospital /ID# 268419
state: Osaka
city: Saitama-shi
country: Japan
facility: Saitama Children's Medical Center /ID# 268410
state: Saitama
city: Bunkyo-ku
country: Japan
facility: Institute of Science Tokyo Hospital /ID# 269175
state: Tokyo
city: Fuchu-shi
country: Japan
facility: Tokyo Metropolitan Children's Medical Center /ID# 268415
state: Tokyo
city: Setagaya-ku
country: Japan
facility: National Center for Child Health and Development /ID# 268420
state: Tokyo
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital /ID# 255318
state: Seoul Teugbyeolsi
city: Seoul
country: Korea, Republic of
facility: Yonsei University Health System Severance Hospital /ID# 256976
state: Seoul Teugbyeolsi
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center /ID# 255284
state: Seoul Teugbyeolsi
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Chilgok Hospital /ID# 255817
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC, locatie AMC /ID# 254827
state: Noord-Holland
city: Torun
country: Poland
facility: Gastromed Sp. z o.o /ID# 255939
state: Kujawsko-pomorskie
city: Warszawa
country: Poland
facility: Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 255938
state: Mazowieckie
city: Dorado
country: Puerto Rico
facility: Puerto Rico Health Institute /ID# 255071
city: San Juan
country: Puerto Rico
facility: Clinical Research Puerto Rico /ID# 266479
city: Ferrol
country: Spain
facility: Hospital Arquitecto Marcide - Complejo Hospitalario Universitario de Ferrol /ID# 255614
state: A Coruna
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Nino Jesus /ID# 255012
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga /ID# 257553
city: Solna
country: Sweden
facility: Karolinska University Hospital Solna /ID# 255240
state: Stockholms Lan
city: Stockholm
country: Sweden
facility: Sodersjukhuset /ID# 255239
state: Stockholms Lan
city: Göteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset /ID# 255236
state: Vastra Gotalands Lan
city: Zurich
country: Switzerland
facility: Kinderspital Zurich - Eleonorenstiftung /ID# 255337
state: Zuerich
city: Bern
country: Switzerland
facility: Inselspital, Universitaetsspital Bern /ID# 255321
city: Taipei City
country: Taiwan
facility: National Taiwan University Hospital /ID# 255679
state: Taipei
city: Changhua City, Changhua County
country: Taiwan
facility: Changhua Christian Hospital /ID# 256082
city: Ankara
country: Turkey
facility: Gazi University Medical Faculty /ID# 255086
city: Istanbul
country: Turkey
facility: Sariyer Hamidiye Etfal Eğitim Ve Araştirma Hastanesi /ID# 257143
city: Istanbul
country: Turkey
facility: Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi /ID# 261020
city: Kocaeli
country: Turkey
facility: Kocaeli University Med Faculty /ID# 256922
city: Sheffield
country: United Kingdom
facility: Sheffield Children's Hospital NHS Foundation Trust /ID# 255758
state: England
city: London
country: United Kingdom
facility: Disc_Barts Health NHS Trust - The Royal London Hospital /ID# 255757
state: Greater London
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's NHS Foundation Trust /ID# 255759
Study officials:
affiliation: AbbVie
name: ABBVIE INC.
role: STUDY_DIRECTOR
References: