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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05782907




Registration number
NCT05782907
Ethics application status
Date submitted
13/03/2023
Date registered
24/03/2023
Date last updated
31/05/2024

Titles & IDs
Public title
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.
Scientific 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
Secondary ID [1] 0 0
2022-501788-41-00
Secondary ID [2] 0 0
M14-658
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ulcerative Colitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 0 0 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Upadacitinib

Experimental: Period 1- Open Label Induction Phase - All participants in open label induction phase of Period 1 will receive upadacitinib Dose A for 8 weeks based on body weight.

Experimental: Period 1- Double Blind Maintenance Phase - Clinical responders at the end of open label induction phase of Period 1 will be randomly assigned to receive either upadacitinib Dose B or Dose C for 44 weeks based on body weight.

Experimental: Period 2- Open Label Long Term Extension Phase Arm A - Clinical non-responders outside of US after Period 1 induction phase will receive upadacitinib Dose A daily for 8 week extended induction phase in open label long term extension (OLE) Period 2. Clinical responders from extended induction phase in OLE will receive upadacitinib Dose B daily for up to 252 weeks in OLE period 2.

Experimental: Period 2- Open Label Long Term Extension Phase Arm B - Clinical non-responders in US after Period 1 induction phase or clinical responders with loss of response during maintenance phase will receive upadacitinib Dose B daily for up to 260 weeks in OLE Period 2.

Experimental: Period 2- Long Term Extension Phase Arm C - Clinical responders who complete Period 1 through Week 52 will receive upadacitinib Dose C daily for up to 260 weeks in OLE Period 2.


Treatment: Drugs: Upadacitinib
Oral Solution/ Tablets
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Achieving Adapted Mayo score (AMS) Clinical Remission (Period 1)
Timepoint [1] 0 0
Week 8
Primary outcome [2] 0 0
Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Responders per AMS (Period 1)
Timepoint [2] 0 0
Week 52
Secondary outcome [1] 0 0
Percentage of Participants Achieving Endoscopic Improvement (Period 1)
Timepoint [1] 0 0
Week 8
Secondary outcome [2] 0 0
Percentage of Participants Achieving Partial Mayo Score (PMS) Clinical Remission (Period 1)
Timepoint [2] 0 0
Week 8
Secondary outcome [3] 0 0
Percentage of Participants Achieving AMS Clinical Response (Period 1)
Timepoint [3] 0 0
Week 8
Secondary outcome [4] 0 0
Percentage of Participants Achieving Endoscopic Improvement Among Week 8 Responders per AMS (Period 1)
Timepoint [4] 0 0
Week 52
Secondary outcome [5] 0 0
Percentage of Participants Achieving PMS Clinical Remission Among Week 8 Responders per AMS (Period 1)
Timepoint [5] 0 0
Week 52
Secondary outcome [6] 0 0
Percentage of Participants Achieving AMS Clinical Response Among Week 8 Responders per AMS (Period 1)
Timepoint [6] 0 0
Week 52
Secondary outcome [7] 0 0
Percentage of Participants Achieving PMS Clinical Response Among Week 8 Clinical Responders per AMS (Period 1)
Timepoint [7] 0 0
Week 52
Secondary outcome [8] 0 0
Percentage of Participants Achieving Corticosteroid-Free AMS Clinical Remission Among Week 8 Responders per AMS (Period 1)
Timepoint [8] 0 0
Week 52
Secondary outcome [9] 0 0
Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Remitters per AMS (Period 1)
Timepoint [9] 0 0
Week 52

Eligibility
Key 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.
Minimum age
2 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/11/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/10/2033
Actual
Sample size
Target
110
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,WA
Recruitment hospital [1] 0 0
Queensland Children's Hospital /ID# 261032 - South Brisbane
Recruitment hospital [2] 0 0
Perth Children's Hospital /ID# 254727 - Nedlands
Recruitment hospital [3] 0 0
Children's Hospital at Westmead /ID# 255556 - Westmead
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment postcode(s) [2] 0 0
6009 - Nedlands
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Delaware
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
Belgium
State/province [11] 0 0
Vlaams-Brabant
Country [12] 0 0
Bulgaria
State/province [12] 0 0
Plovdiv
Country [13] 0 0
Bulgaria
State/province [13] 0 0
Sofiya
Country [14] 0 0
Bulgaria
State/province [14] 0 0
Varna
Country [15] 0 0
France
State/province [15] 0 0
Gironde
Country [16] 0 0
France
State/province [16] 0 0
Rhone
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
France
State/province [18] 0 0
Toulouse
Country [19] 0 0
Greece
State/province [19] 0 0
Attiki
Country [20] 0 0
Greece
State/province [20] 0 0
Kriti
Country [21] 0 0
Hungary
State/province [21] 0 0
Hajdu-Bihar
Country [22] 0 0
Hungary
State/province [22] 0 0
Budapest
Country [23] 0 0
Israel
State/province [23] 0 0
HaMerkaz
Country [24] 0 0
Israel
State/province [24] 0 0
Yerushalayim
Country [25] 0 0
Italy
State/province [25] 0 0
Firenze
Country [26] 0 0
Italy
State/province [26] 0 0
Roma
Country [27] 0 0
Japan
State/province [27] 0 0
Chiba
Country [28] 0 0
Japan
State/province [28] 0 0
Fukuoka
Country [29] 0 0
Japan
State/province [29] 0 0
Hokkaido
Country [30] 0 0
Japan
State/province [30] 0 0
Miyagi
Country [31] 0 0
Japan
State/province [31] 0 0
Osaka
Country [32] 0 0
Japan
State/province [32] 0 0
Tokyo
Country [33] 0 0
Japan
State/province [33] 0 0
Saitama
Country [34] 0 0
Korea, Republic of
State/province [34] 0 0
Daegu
Country [35] 0 0
Korea, Republic of
State/province [35] 0 0
Seoul
Country [36] 0 0
Netherlands
State/province [36] 0 0
Groningen
Country [37] 0 0
New Zealand
State/province [37] 0 0
Auckland
Country [38] 0 0
New Zealand
State/province [38] 0 0
Canterbury
Country [39] 0 0
Poland
State/province [39] 0 0
Kujawsko-pomorskie
Country [40] 0 0
Poland
State/province [40] 0 0
Mazowieckie
Country [41] 0 0
Spain
State/province [41] 0 0
A Coruna
Country [42] 0 0
Spain
State/province [42] 0 0
Barcelona
Country [43] 0 0
Spain
State/province [43] 0 0
Malaga
Country [44] 0 0
Taiwan
State/province [44] 0 0
Taipei
Country [45] 0 0
Taiwan
State/province [45] 0 0
Taoyuan City
Country [46] 0 0
United Kingdom
State/province [46] 0 0
England
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Hampshire
Country [48] 0 0
United Kingdom
State/province [48] 0 0
London, City Of

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
AbbVie
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05782907
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
ABBVIE INC.
Address 0 0
AbbVie
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
ABBVIE CALL CENTER
Address 0 0
Country 0 0
Phone 0 0
844-663-3742
Fax 0 0
Email 0 0
abbvieclinicaltrials@abbvie.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05782907