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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06332534
Registration number
NCT06332534
Ethics application status
Date submitted
19/03/2024
Date registered
27/03/2024
Titles & IDs
Public title
Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
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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 Crohn's Disease and Inadequate Response, Intolerance, or Medical Contraindications to Corticosteroids, Immunosuppressants, and/or Biologic Therapy
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Secondary ID [1]
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M14-671
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Crohn's Disease
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Condition category
Condition code
Oral and Gastrointestinal
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Inflammatory bowel disease
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Oral and Gastrointestinal
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Crohn's disease
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Upadacitinib
Experimental: Period 1: Open Label Induction Phase (Dose A) - All participants in the open label induction phase of Period 1 will receive upadacitinib Dose A for 12 weeks based on body weight.
Experimental: Period 1: Double-Blind Maintenance Phase (Dose B) - Clinical responders per PCDAI at the end of open label induction phase of Period 1 will be randomly assigned to receive Dose B or C for 52 weeks (oral solution dose will be based on body weight)
Experimental: Period 1: Double-Blind Maintenance Phase (Dose C) - Clinical responders per PCDAI at the end of open label induction phase of Period 1 will be randomly assigned to receive either upadacitinib Dose C or B for 52 weeks (oral solution dose will be based on body weight)
Experimental: Period 2: Open Label Long-Term Extension Phase Cohort 1 - Participants receiving double-blind maintenance therapy with upadacitinib Dose B or upadacitinib Dose C daily in Period 1 who complete the Week 64 visit will receive upadacitinib Dose B daily for up to 156 weeks.
Experimental: Period 2: Open Label Long-Term Extension Phase Cohort 2 - Participants who were receiving rescue therapy with open-label upadacitinib Dose C during maintenance phase in Period 1 and completed the Week 64 visit will continue to receive upadacitinib Dose C daily for up to 156 weeks.
Experimental: Period 2: Open Label Long-Term Extension Phase Cohort 3 - Participants who did not achieve clinical response per PCDAI at Week 12 of Period 1 will receive an extended treatment with open-label upadacitinib Dose C daily for an additional 12 weeks. If they are responders after 12 weeks extended treatment, they will continue, otherwise they may be discontinued at the discretion of the investigator
Treatment: Drugs: Upadacitinib
Oral Solution/ Extended-Release Tablets
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of participants who achieved clinical response per the Pediatric Crohn's Disease Activity Index (PCDAI) at Week 12, with clinical remission per the PCDAI at Week 64
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Assessment method [1]
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PCDAI is an index used to measure disease activity of pediatric patients with Crohn's disease assessing abdominal pain, stool frequency, patient functioning, hematocrit, erythrocyte sedimentation rate, albumin, weight, height, abdomen, perirectal disease, and extraintestinal manifestations. It ranges from 0 to 100; higher scores indicate more active disease. Clinical remission was defined as PCDAI = 10.
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Timepoint [1]
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At Week 64
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Primary outcome [2]
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Achievement of endoscopic response at Week 64 in participants who achieved clinical response per PCDAI at Week 12.
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Assessment method [2]
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Endoscopic response is defined as \> 50% reduction in Simple Endoscopic Score for Crohn's Disease (SES-CD) score from Baseline (or for participants with a Baseline SES-CD of 4, at least a 2-point reduction from Baseline), as scored by a central reader.
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Timepoint [2]
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At Week 64
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Primary outcome [3]
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Number of Participants with Adverse Events
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Assessment method [3]
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An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. The investigator assessed the relationship of each event to the use of study drug as either probably related, possibly related, probably not related or not related.
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Timepoint [3]
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Through Week 156
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Secondary outcome [1]
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Achievement of clinical remission per PCDAI
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Assessment method [1]
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Clinical remission per PCDAI is defined as PCDAI = 10.
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Timepoint [1]
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Week 12
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Secondary outcome [2]
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Achievement of endoscopic response
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Assessment method [2]
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Endoscopic response is defined as \> 50% reduction in SES-CD score from Baseline (or for participants with a Baseline SES-CD of 4, at least a 2-point reduction from Baseline), as scored by a central reader.
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Timepoint [2]
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Week 12
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Secondary outcome [3]
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Achievement of endoscopic remission
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Assessment method [3]
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Endoscopic remission is defined as SES-CD = 4 with at least a 2-point reduction from Baseline and no subscore \> 1, as scored by a central reader
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Timepoint [3]
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Week 12
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Secondary outcome [4]
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Achievement of clinical response per PCDAI
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Assessment method [4]
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Clinical response is defined as reduction in PCDAI of = 15 points from Baseline
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Timepoint [4]
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Week 12
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Secondary outcome [5]
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Achievement of clinical response per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12
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Assessment method [5]
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Timepoint [5]
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Week 64
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Secondary outcome [6]
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Achievement of endoscopic remission at Week 64 in participants who achieved clinical response per PCDAI at Week 12
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Assessment method [6]
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Endoscopic remission is defined as SES-CD = 4 with at least a 2-point reduction from Baseline and no subscore \> 1, as scored by a central reader
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Timepoint [6]
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Week 64
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Secondary outcome [7]
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Achievement of corticosteroid (CS)-free clinical remission per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12
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Assessment method [7]
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CS-free clinical remission per PCDAI: clinical remission per PCDAI and not receiving corticosteroids at Week 12 (for Period 1 induction endpoint\[s\]); or for at least 90 days prior to the study visit at which endpoint is assessed (for Period 1 maintenance endpoint\[s\] and Period 2 endpoint\[s\]).
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Timepoint [7]
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Week 64
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Eligibility
Key inclusion criteria
* Weight at Screening and Baseline must be = 10 kg
* Moderate to severe CD defined as PCDAI > 30 and endoscopic evidence of mucosal inflammation as documented by a centrally read SES-CD of >/ 6 (or SES-CD of >/4 for isolated ileal disease) excluding the presence of narrowing component.
* Documented diagnosis of CD prior to Baseline, confirmed by colonoscopy during the screening period, with exclusion of current infection, colonic dysplasia and/or malignancy. Appropriate documentation of biopsy results consistent with the diagnosis of CD, in the assessment of the investigator, must be available
* Demonstrated an inadequate response, loss of response, or intolerance to corticosteroids, IMMs, and/or biologic therapy or in whom use of those therapies is medically contraindicated. For participants in the US, participants must have demonstrated an inadequate response, loss or response, or intolerance to one or more anti-TNFs (tumor necrosis factor).
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Minimum age
2
Years
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* History of:
* A diagnosis of CD prior to 2 years of age.
* Currently known complications of CD such as:
* Active abscess (abdominal or perianal);
* Symptomatic bowel strictures;
* More than 2 missing segments of the following 5 intestinal segments: terminal ileum, right colon, transverse colon, sigmoid and left colon, and rectum;
* Ostomy or ileoanal pouch;
* Surgical bowel resection within the past 3 months prior to Baseline, or a history of more than 3 bowel resections.
* Japan participants only: positive result of beta-D-glucan or two consecutive indeterminate results of beta-D-glucan during the Screening period (screening for Pneumocystis jiroveci infection)
* History of any of the following:
* Current diagnosis of UC, indeterminate colitis, or monogenic IBD;
* Fulminant colitis or toxic megacolon;
* Gastrointestinal perforation (other than due to appendicitis or mechanical injury), diverticulitis, or significantly increased risk for GI perforation per investigator judgment including history of volvulus and/or intussusception (telescoping of bowels);
* Current diagnosis of any primary immune deficiency
* Conditions that could interfere with drug absorption including but not limited to short bowel syndrome or gastric bypass surgery; subjects with a history of gastric banding/segmentation are not excluded.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Other
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
26/07/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
20/12/2034
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Actual
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Sample size
Target
110
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NT
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Recruitment hospital [1]
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Children's Hospital at Westmead /ID# 262350 - Westmead
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment outside Australia
Country [1]
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Japan
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State/province [1]
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Chiba
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Country [2]
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Japan
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State/province [2]
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Fukuoka
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Country [3]
0
0
Japan
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State/province [3]
0
0
Miyagi
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Country [4]
0
0
Japan
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State/province [4]
0
0
Osaka
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Country [5]
0
0
Japan
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State/province [5]
0
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Saga
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Country [6]
0
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Japan
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State/province [6]
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Saitama
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Country [7]
0
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Japan
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State/province [7]
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Tokyo
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Country [8]
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Japan
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State/province [8]
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Toyama
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Country [9]
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Korea, Republic of
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State/province [9]
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Seoul Teugbyeolsi
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Country [10]
0
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New Zealand
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State/province [10]
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Canterbury
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Country [11]
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Puerto Rico
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State/province [11]
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Bayamon
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Country [12]
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Puerto Rico
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State/province [12]
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Dorado
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Country [13]
0
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Taiwan
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State/province [13]
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Taipei
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
AbbVie
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Crohn's disease (CD) is a long-lasting disease that causes severe inflammation (redness, swelling), in the digestive tract, most often affecting the bowels. It can cause many different symptoms including abdominal pain, diarrhea, tiredness, and weight loss. This study will assess how safe and effective oral Upadacitinib is in treating moderately to severely active Crohn's Disease in pediatric participants aged 2 to 18 years old who have had inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy. Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active CD and is being developed for moderate- to severely active CD in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: a 12-week open-label induction phase which means that the study doctor and participants know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 52-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 156-week open-label extension of Period 1. Approximately 110 pediatric participants with moderate to severely active CD will be enrolled at approximately 92 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 have a safety follow up for 30 days after discontinuation from any time point within the study. There may be higher treatment burden for participants in this trial compared to their standard of care (due to study procedures). Participants will attend regular (weekly, monthly) 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.
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Trial website
https://clinicaltrials.gov/study/NCT06332534
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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ABBVIE INC.
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Address
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AbbVie
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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ABBVIE CALL CENTER
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Address
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Country
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Phone
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844-663-3742
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
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When will data be available (start and end dates)?
For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
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Available to whom?
To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/abbvie/
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06332534