Study to Assess Adverse Events, Change in Disease Activity, and How Oral Upadacitinib Moves Through the Body of Pediatric Participants With Moderately to Severely Active Ulcerative Colitis.

Ulcerative Colitis Clinical Trial

Official title:

A Phase 3 Multicenter Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Upadacitinib With Open-Label Induction, Randomized, Double-Blind Maintenance and Open-Label Long-Term Extension in Pediatric Subjects With Moderately to Severely Active Ulcerative Colitis and Inadequate Response, Intolerance, or Medical Contraindications to Corticosteroids, Immunosuppressants, and/or Biologic Therapy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05782907
• Other study ID #: M14-658
• Secondary ID: 2022-501788-41-0
• Status: Recruiting
• Phase: Phase 3
• Start date: November 6, 2023
• Est. completion date: October 31, 2033

Study information

• Verified date: May 2024
• Source: AbbVie
• Contact: ABBVIE CALL CENTER
• Phone: 844-663-3742
• Email: abbvieclinicaltrials[@]abbvie.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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 safe and effective Upadacitinib is in treating pediatric participants with UC. Adverse events and change in disease activity will be assessed. Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active UC and is being developed for moderate- to severely active UC in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: an 8-week open-label induction phase which means that the study doctor and patients know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 44-week double-blind maintenance phase meaning that neither the participants nor the study doctors will know which dose of upadacitinib will be given(UPA Dose B or Dose C). Period 2 is a 260 week open-label extension (OLE) of Period 1. Approximately 110 pediatric participants with moderate to severely active UC will be enrolled at up to 100 sites worldwide. Participants will receive upadacitinib oral tablets once daily or oral solution twice daily at approximately the same time each day, with or without food. Participants will be followed up for 30 days after each phase (i.e. after induction, maintenance, OLE) and only if a participant doesn't continue into the next phase. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 110
• Est. completion date: October 31, 2033
• Est. primary completion date: August 12, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

Inclusion Criteria:

• Active UC with an AMS of 5 to 9 points and endoscopic subscore of 2 to 3.
• Demonstrate an inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy.

Exclusion Criteria:

• Partcipants with previous exposure to JAK inhibitors (e.g., tofacitinib, baricitinib, filgotinib, upadacitinib).
• Females who are pregnant, breastfeeding, or considering becoming pregnant during the study and for approximately 30 days after the last dose of study drug.

Related Conditions & MeSH terms

• Colitis
• Colitis, Ulcerative
• Ulcer
• Ulcerative Colitis

Intervention

Drug:
• Upadacitinib
Oral Solution/ Tablets

Locations

Country	Name	City	State
Australia	Perth Children's Hospital /ID# 254727	Nedlands	Western Australia
Australia	Queensland Children's Hospital /ID# 261032	South Brisbane	Queensland
Australia	Children's Hospital at Westmead /ID# 255556	Westmead
Belgium	Universitair Ziekenhuis Leuven /ID# 251185	Leuven	Vlaams-Brabant
Bulgaria	UMHAT Sveti Georgi /ID# 251949	Plovdiv
Bulgaria	Specialized Hospital For Active Treatment Of Children Diseases Prof. Ivan Mitev /ID# 251285	Sofiya
Bulgaria	UMHAT Sveta Marina /ID# 251948	Varna
France	CHU Bordeaux - Hopital Pellegrin /ID# 253182	Bordeaux	Gironde
France	HCL - Hopital Femme Mere Enfant /ID# 251502	Bron CEDEX	Rhone
France	AP-HP - Hopital Necker /ID# 251658	Paris
France	Hopitaux de Paris (AP-HP) - Hopital Robert Debre - CHU /ID# 252069	Paris
France	CHU Toulouse - Hopital Paule de Viguier /ID# 252070	Toulouse
Greece	Agia Sofia Hospital /ID# 250697	Athens	Attiki
Greece	University General Hospital of Heraklion PA.G.N.I /ID# 250696	Heraklion	Kriti
Hungary	Semmelweis Egyetem /ID# 251083	Budapest
Hungary	Debreceni Egyetem-Klinikai Kozpont /ID# 251835	Debrecen	Hajdu-Bihar
Israel	Shaare Zedek Medical Center /ID# 254832	Jerusalem	Yerushalayim
Israel	Schneider Children's Medical Center /ID# 254833	Petah Tikva	HaMerkaz
Italy	Azienda Ospedaliero Universitaria Meyer /ID# 251624	Florence	Firenze
Italy	Sapienza University /ID# 251625	Rome	Roma
Japan	Tokyo Medical And Dental University Hospital /ID# 251929	Bunkyo-ku	Tokyo
Japan	Tokyo Metropolitan Children's Medical Center /ID# 252477	Fuchu City	Tokyo
Japan	Osaka Women's and Children's Hospital /ID# 252397	Izumi-Shi	Osaka
Japan	Tsujinaka Hospital Kashiwanoha /ID# 251930	Kashiwa-shi	Chiba
Japan	Kurume University Hospital /ID# 251927	Kurume-shi	Fukuoka
Japan	Osaka General Medical Center /ID# 253678	Osaka-shi	Osaka
Japan	Saitama Children's Medical Center /ID# 252362	Saitama-shi	Saitama
Japan	Hokkaido P.W.F.A.C. Sapporo-Kosei General Hospital /ID# 251928	Sapporo-shi	Hokkaido
Japan	Miyagi Children's Hospital /ID# 251931	Sendai-shi	Miyagi
Japan	National Center for Child Health and Development /ID# 251926	Setagaya-ku	Tokyo
Korea, Republic of	Kyungpook National University Chilgok Hospital /ID# 252663	Daegu
Korea, Republic of	Samsung Medical Center /ID# 252023	Seoul
Korea, Republic of	Seoul National University Hospital /ID# 252024	Seoul
Netherlands	Universitair Medisch Centrum Groningen /ID# 252003	Groningen
New Zealand	Christchurch Hospital /ID# 254703	Christchurch	Canterbury
New Zealand	Starship Child Health /ID# 254702	Grafton	Auckland
Poland	Gastromed Sp. z o.o /ID# 251290	Torun	Kujawsko-pomorskie
Poland	Instytut Pomnik - Centrum Zdrowia Dziecka /ID# 251289	Warszawa	Mazowieckie
Spain	Hospital Sant Joan de Deu /ID# 251194	Esplugues de Llobregat	Barcelona
Spain	Hospital Arquitecto Marcide - Complejo Hospitalario Universitario de Ferrol /ID# 252105	Ferrol	A Coruna
Spain	Hospital Regional Universitario de Malaga /ID# 251193	Malaga
Taiwan	National Taiwan University Hospital /ID# 251650	Taipei City
Taiwan	Linkou Chang Gung Memorial Hospital /ID# 251654	Taoyuan City
United Kingdom	Barts Health NHS Trust /ID# 251917	London	London, City Of
United Kingdom	Sheffield Children's Hospital NHS Foundation Trust /ID# 251600	Sheffield	England
United Kingdom	University Hospital Southampton NHS Foundation Trust /ID# 252097	Southampton	Hampshire
United States	Children's Hospital Colorado - Aurora /ID# 250110	Aurora	Colorado
United States	Levine Children's Hospital /ID# 250131	Charlotte	North Carolina
United States	UH Cleveland Medical Center /ID# 250134	Cleveland	Ohio
United States	Arkansas Children's Hospital /ID# 250106	Little Rock	Arkansas
United States	The Mount Sinai Hospital /ID# 250141	New York	New York
United States	Children's Specialty Group /ID# 256966	Norfolk	Virginia
United States	UCSF Benioff Children's Hospital - Oakland /ID# 255067	Oakland	California
United States	Phoenix Children's Hospital /ID# 250135	Phoenix	Arizona
United States	Nemours/Alfred duPont Hospital for Children /ID# 255483	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
AbbVie

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Bulgaria,  France,  Greece,  Hungary,  Israel,  Italy,  Japan,  Korea, Republic of,  Netherlands,  New Zealand,  Poland,  Spain,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants Achieving Adapted Mayo score (AMS) Clinical Remission (Period 1)	The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The AMS is a composite of the following subscores: stool frequency subscore (SFS), rectal bleeding subscore (RBS) and endoscopy subscore (MES). AMS ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS < or = 2, with SFS < or = 1 and not higher than baseline, RBS of 0, and MES < or = 1.	Week 8
Primary	Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Responders per AMS (Period 1)	The Mayo score is a tool designed to measure disease activity for ulcerative colitis. AMS ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS = 2, with SFS = 1 and not higher than Baseline, RBS of 0, and MES = 1.	Week 52
Secondary	Percentage of Participants Achieving Endoscopic Improvement (Period 1)	Endoscopic Improvement is defined as MES < or = 1.	Week 8
Secondary	Percentage of Participants Achieving Partial Mayo Score (PMS) Clinical Remission (Period 1)	The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The PMS is a composite of the following subscores: SFS, RBS and physician's global assessment (PGA). The PMS ranges from 0 to 9 with higher scores representing more severe disease. PMS clinical remission is defined as a PMS < or = 2 and no individual subscore > 1.	Week 8
Secondary	Percentage of Participants Achieving AMS Clinical Response (Period 1)	The adapted mayo score (AMS) is a composite of the following subscores: SFS, RBS and MES. AMS clinical response is defined as decrease in AMS by > or = 2 points and > or = 30% from baseline with a decrease in RBS of > or = 1 or an absolute RBS of 0 or 1.	Week 8
Secondary	Percentage of Participants Achieving Endoscopic Improvement Among Week 8 Responders per AMS (Period 1)	Endoscopic Improvement is defined as MES of < or = 1. The AMS is a composite of the following subscores: SFS, RBS and MES.	Week 52
Secondary	Percentage of Participants Achieving PMS Clinical Remission Among Week 8 Responders per AMS (Period 1)	The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The PMS ranges from 0 to 9 with higher scores representing more severe disease. PMS clinical remission is defined as a PMS < or = 2 and no individual subscore > 1. The AMS is a composite of the following subscores: SFS, RBS and MES.	Week 52
Secondary	Percentage of Participants Achieving AMS Clinical Response Among Week 8 Responders per AMS (Period 1)	The AMS is a composite of the following subscores: SFS, RBS and MES. AMS clinical response is defined as decrease in AMS by > or = 2 points and > or = 30% from baseline with a decrease in RBS of > or = 1 or an absolute RBS of 0 or 1.	Week 52
Secondary	Percentage of Participants Achieving PMS Clinical Response Among Week 8 Clinical Responders per AMS (Period 1)	The AMS is a composite of the following subscores: SFS, RBS and endoscopy MES. PMS clinical response is defined as decrease in PMS by > or = 2 points and > or = 30% from baseline with a decrease in RBS > or = 1 or an absolute RBS of 0 or 1.	Week 52
Secondary	Percentage of Participants Achieving Corticosteroid-Free AMS Clinical Remission Among Week 8 Responders per AMS (Period 1)	Corticosteroid-free AMS clinical remission is defined as being in AMS clinical remission and free of corticosteroids for >= 90 days immediately preceeding the timepoint of endpoint assessment. The AMS is a composite of the following subscores: SFS, RBS and MES.	Week 52
Secondary	Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Remitters per AMS (Period 1)	The AMS is a composite of the following subscores: SFS, RBS and MES. Adapted Mayo score ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS < or = 2, with SFS < or = 1 and not higher than baseline, RBS of 0, and MES < or = 1.	Week 52

External Links

•   This clinical study may be evaluating a usage that is not currently FDA approved. Please see US Prescribing Information for approved uses.