A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis

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 Open-Label Long-Term Extension Periods in Pediatric Subjects (2 to < 18 Years of Age) With Moderately to Severely Active Ulcerative Colitis

Overview

Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). This study will assess how Risankizumab moves through the body as well as how safe and effective it is in treating pediatric participants with moderate to severely active UC. Adverse events and change in disease activity will be assessed. Risankizumab is an approved medication for moderate to severe UC in multiple countries and is being developed for the treatment of UC 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 maintenance regimen of risankizumab. SS3 is an open-label extension period where participants will receive risankizumab based off of their response in SS2. Around 120 pediatric participants with UC will be enrolled at around 80 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.

Interventions

• Drug Risankizumab
  Risankizumab intravenous (IV) infusion
• Drug Risankizumab
  Risankizumab subcutaneous (SC) injection

Primary outcome measures

• PK Lead-In Cohort 1: Maximum Observed Serum Concentration (Cmax) [Time frame: At Week 64]
• PK Lead-In Cohort 2: Maximum Observed Serum Concentration (Cmax) [Time frame: At Week 64]
• PK Lead-In Cohort 1: Time to Maximum Serum Concentration (Tmax) [Time frame: At Week 64]
• PK Lead-In Cohort 2: Time to Maximum Serum Concentration (Tmax) [Time frame: At Week 64]
• PK Lead-In Cohort 1: Area Under the Serum Concentration-Time Curve Over the Dosing Interval (AUCtau) [Time frame: At Week 64]
• PK Lead-In Cohort 2: Area Under the Serum Concentration-Time Curve Over the Dosing Interval (AUCtau) [Time frame: At Week 64]
• Expansion Cohort 3: Achievement of Clinical Remission per Modified Mayo Score (mMS) Among Week 12 Clinical Responders per mMS [Time frame: At Week 64]
• Number of Participants With Adverse Events [Time frame: Up to 292 Weeks]

Secondary outcome measures (12)

• PK Lead-In Cohort 1: Achievement of clinical remission per mMS among Week 12 responders per mMS [Time frame: At Week 64]
• PK Lead-In Cohort 2: Achievement of clinical remission per mMS among Week 12 responders per mMS [Time frame: At Week 64]
• PK Lead-In Cohort 1: Achievement of clinical remission per mMS [Time frame: At Week 12]
• PK Lead-In Cohort 2: Achievement of clinical remission per mMS [Time frame: At Week 12]
• PK Lead-In Cohort 1: Achievement of clinical response per mMS [Time frame: At Week 12]
• PK Lead-In Cohort 2: Achievement of clinical response per mMS [Time frame: At Week 12]
• PK Lead-In Cohort 1: Achievement of endoscopic improvement [Time frame: At Week 12]
• PK Lead-In Cohort 2: Achievement of endoscopic improvement [Time frame: At Week 12]
• PK Lead-In Cohort 1: Symptomatic response per partial mMS [Time frame: At Week 12]
• PK Lead-In Cohort 2: Symptomatic response per partial mMS [Time frame: At Week 12]
• PK Lead-In Cohort 1: Achievement of clinical response per mMS among Week 12 responders per mMS [Time frame: At Week 64]
• PK Lead-In Cohort 2: Achievement of clinical response per mMS among Week 12 responders per mMS [Time frame: At Week 64]

Eligibility criteria

Inclusion criteria

• Active ulcerative colitis (UC) with an modified Mayo Score (mMS) of 5 to 9 points and endoscopic subscore of 2 to 3 (confirmed by central reader).
• Demonstrated intolerance or inadequate response (IR) to one or more of the following categories of drugs:

aminosalicylates (except in countries where failure of this drug class is not sufficient for eligibility), oral locally acting corticosteroids, systemic steroids (prednisone or equivalent), immunomodulators (IMMs), and/or biologic therapies, as outlined in the protocol.

\- Subjects must have a documented history of UC for at least 3 months prior to Baseline, confirmed by colonoscopy during the screening period, with exclusion of current infection, colonic dysplasia and/or malignancy. Documentation of pathology results consistent with the diagnosis of UC must be available.

Exclusion criteria

• Participants who have had a major surgery performed within 12 weeks prior to Baseline or planned during the conduct of the study (e.g., inguinal hernia repair, cholecystectomy, intestinal resection).
• Participants who have concurrent clinically significant medical conditions other than the indication being studied or any other reason that the investigator determines would interfere with the subject's participation in this study, would make the subject an unsuitable candidate to receive study treatment, or would put the subject at risk by participating in the study.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Sequential

Masking

Double blind

Primary purpose

Treatment

Study locations

United States · 11 centers

• Phoenix Children's Hospital /ID# 273015 — Phoenix
• Rady Children's Hospital /ID# 271873 — San Diego
• University of California San Francisco - Mission Bay /ID# 273022 — San Francisco
• Nicklaus Children's Hospital - Miami - Southwest 62nd Avenue /ID# 271585 — Miami
• Childrens Center For Digestive Health Care /ID# 273228 — Atlanta
• University of Chicago Medical Center /ID# 271588 — Chicago
• Goryeb Children's Hospital /ID# 271801 — Morristown
• University Hospitals Cleveland Medical Center /ID# 271831 — Cleveland
• … and 3 more centers

Japan · 6 centers

• Aichi Medical University Hospital /ID# 281613 — Nagakute
• Tsujinaka Hospital - Kashiwanoha /ID# 280303 — Kashiwa-shi
• Kagawa University Hospital /ID# 282010 — Kita-gun
• Osaka Women's and Children's Hospital /ID# 280789 — Izumi-Shi
• Tokyo Medical University Hospital /ID# 280850 — Shinjuku-ku
• Nagasaki University Hospital /ID# 281241 — Nagasaki

South Korea · 5 centers

• Pusan National University Yangsan Hospital /ID# 272769 — Yangsan
• Seoul National University Hospital /ID# 272852 — Seoul
• Kangbuk Samsung Hospital /ID# 273333 — Seoul
• Yonsei University Health System Severance Hospital /ID# 272894 — Seoul
• Samsung Medical Center /ID# 272862 — Seoul

Italy · 4 centers

• Fondazione di Religione e di Culto Casa Sollievo della Sofferenza /ID# 271889 — San Giovanni Rotondo
• Azienda Ospedaliera Universitaria Federico II - Naples /ID# 271895 — Naples
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS-Universita Cattolica /ID# 2729 — Rome
• Ospedale Infantile Burlo Garofolo /ID# 274442 — Trieste

Belgium · 3 centers

• Cliniques Universitaires UCL Saint-Luc /ID# 270123 — Brussels
• Hospital Universite Enfants Reine Fabiola /ID# 271860 — Brussels
• Centre Hospitalier Regional de la Citadelle /ID# 270459 — Liège

Brazil · 3 centers

• Galileo Medical Research /ID# 271817 — Juiz de Fora
• Hospital Pequeno Principe /ID# 271814 — Curitiba
• Hospital Sirio Libanes - Sao Paulo /ID# 271818 — São Paulo

Canada · 3 centers

• Alberta Children's Hospital /ID# 272635 — Calgary
• Edmonton Clinic Health Academy /ID# 271168 — Edmonton
• London Health Sciences Centre - Verspeeten Family Cancer Centre /ID# 271157 — London

Germany · 3 centers

• Medizinische Universitaet Lausitz - Carl Thiem /ID# 272023 — Cottbus
• Klinikum Kassel /ID# 271546 — Kassel
• Universitaetsklinikum Muenster - Albert Schweitzer Campus /ID# 271898 — Münster

Greece · 3 centers

• General Hospital of Chest Diseases of Athens SOTIRIA /ID# 270143 — Athens
• University General Hospital Attikon /ID# 272361 — Chaïdári
• General Hospital of Thessaloniki Hippokrateio /ID# 271939 — Thessaloniki

Serbia · 3 centers

• University Children's Hospital /ID# 269960 — Belgrade
• Institut za zdravstvenu zastitu majke i deteta Srbije Dr Vukan Cupic /ID# 270696 — Belgrade-Vračar
• Institute for Child and Youth Health Care of Vojvodina /ID# 269961 — Novi Sad

Sweden · 3 centers

• Karolinska University Hospital Solna /ID# 271679 — Solna
• Sodersjukhuset /ID# 271678 — Stockholm
• Sahlgrenska Universitetssjukhuset /ID# 271675 — Gothenburg

United Kingdom · 3 centers

• Addenbrookes Hospital /ID# 271489 — Cambridge
• Sheffield Children's Hospital NHS Foundation Trust /ID# 271490 — Sheffield
• Alder Hey Children's NHS Foundation Trust /ID# 271533 — Liverpool

China · 2 centers

• Henan Children's Hospital Zhengzhou Children's Hospital /ID# 278794 — Zhengzhou
• The First Hospital of Jilin University - Changchun /ID# 279290 — Changchun

Spain · 2 centers

• Hospital Teresa Herrera - CHUAC /ID# 271459 — A Coruña
• Hospital Universitario Puerta de Hierro - Majadahonda /ID# 271466 — Majadahonda

Taiwan · 2 centers

• Taichung Veterans General Hospital /ID# 269242 — Taichung
• National Taiwan University Hospital /ID# 269244 — Taipei

Identifiers

NCT: NCT07071519 · M19-751 · 2024-514695-41-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗