Upadacitinib for Crohn's Disease
(U-EMPOWER Trial)
Recruiting in Palo Alto (17 mi)
+68 other locations
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: AbbVie
Disqualifiers: Ulcerative colitis, Abscess, Bowel strictures, others
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 3 Jurisdictions
Trial Summary
What is the purpose of this trial?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.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study doctor to understand how your current treatments might interact with the trial.
How is the drug Upadacitinib different from other treatments for Crohn's disease?
Upadacitinib is unique because it is an oral medication that selectively inhibits Janus kinase 1 (JAK-1), a protein involved in inflammation, and it has shown promise in treating Crohn's disease, especially in patients who have not responded to other treatments. It is already approved for ulcerative colitis and rheumatoid arthritis, making it a novel option for Crohn's disease.
12345Eligibility Criteria
This trial is for children aged 2-18 with moderate to severe Crohn's Disease who haven't had success with, or can't use, standard treatments like corticosteroids and biologics. They must weigh at least 10 kg and have a confirmed diagnosis of CD with specific endoscopic findings.Inclusion Criteria
Weight at Screening and Baseline must be ≥ 10 kg
My Crohn's disease is moderate to severe with a PCDAI score over 30 and confirmed inflammation.
My condition didn't improve or worsened with standard treatments, or I can't tolerate them.
+1 more
Exclusion Criteria
I do not have conditions like short bowel syndrome or have had gastric bypass surgery that could affect drug absorption.
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)
I have been diagnosed with a primary immune deficiency.
+3 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Open-Label Induction
Participants receive upadacitinib Dose A for 12 weeks based on body weight
12 weeks
Weekly visits (in-person)
Double-Blind Maintenance
Participants receive either upadacitinib Dose B or Dose C for 52 weeks
52 weeks
Monthly visits (in-person)
Open-Label Long-Term Extension
Participants continue to receive upadacitinib for up to 156 weeks
156 weeks
Monthly visits (in-person)
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
1 visit (in-person)
Participant Groups
The trial tests Upadacitinib's safety and effectiveness in these kids over two periods: first a known-dose phase for 12 weeks, then a year where the dose is unknown to them. It ends with an open-label extension for up to three years.
6Treatment groups
Experimental Treatment
Group I: Period 2: Open Label Long-Term Extension Phase Cohort 3Experimental Treatment1 Intervention
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
Group II: Period 2: Open Label Long-Term Extension Phase Cohort 2Experimental Treatment1 Intervention
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.
Group III: Period 2: Open Label Long-Term Extension Phase Cohort 1Experimental Treatment1 Intervention
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.
Group IV: Period 1: Open Label Induction Phase (Dose A)Experimental Treatment1 Intervention
All participants in the open label induction phase of Period 1 will receive upadacitinib Dose A for 12 weeks based on body weight.
Group V: Period 1: Double-Blind Maintenance Phase (Dose C)Experimental Treatment1 Intervention
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)
Group VI: Period 1: Double-Blind Maintenance Phase (Dose B)Experimental Treatment1 Intervention
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)
Upadacitinib is already approved in European Union, United States, Canada for the following indications:
🇪🇺 Approved in European Union as Rinvoq for:
- Rheumatoid arthritis
- Psoriatic arthritis
- Atopic dermatitis
- Ankylosing spondylitis
- Ulcerative colitis
- Crohn's disease
🇺🇸 Approved in United States as Rinvoq for:
- Rheumatoid arthritis
- Psoriatic arthritis
- Atopic dermatitis
- Ankylosing spondylitis
- Ulcerative colitis
- Crohn's disease
🇨🇦 Approved in Canada as Rinvoq for:
- Rheumatoid arthritis
- Psoriatic arthritis
- Atopic dermatitis
- Ankylosing spondylitis
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
OSF St. Francis Medical Center /ID# 262192Peoria, IL
Indiana University Health Riley Hospital for Children /ID# 262215Indianapolis, IN
MNGI Digestive Health, P. A. /ID# 262204Minneapolis, MN
UH Cleveland Medical Center /ID# 262188Cleveland, OH
More Trial Locations
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Who Is Running the Clinical Trial?
AbbVieLead Sponsor
References
Multicentre Real-world Experience of Upadacitinib in the Treatment of Crohn's Disease. [2023]Upadacitinib is a selective Janus kinase inhibitor approved for the management of ulcerative colitis and is under evaluation for the management of Crohn's disease [CD] in Phase 3 clinical trials.
Upadacitinib Is Effective and Safe in Both Ulcerative Colitis and Crohn's Disease: Prospective Real-World Experience. [2023]Upadacitinib is a novel selective Janus kinase 1 inhibitor that has shown efficacy in the treatment of moderate to severe ulcerative colitis (UC) and Crohn's disease (CD), and has received Food and Drug Administration approval for UC. We report a large real-world experience with upadacitinib in UC and CD.
Upadacitinib Induction and Maintenance Therapy for Crohn's Disease. [2023]Upadacitinib, an oral selective Janus kinase (JAK) inhibitor, is under investigation for the treatment of Crohn's disease.
Upadacitinib Is Safe and Effective for Crohn's Disease: Real-World Data from a Tertiary Center. [2023]Upadacitinib is a selective JAK-1 inhibitor approved for the treatment of rheumatoid arthritis and more recently, ulcerative colitis. Phase II trials demonstrated that upadacitinib induces endoscopic remission in patients with moderate-to-severe Crohn's disease. However, real-world data are lacking. We present a short report on our experience with off-label upadacitinib in patients with CD at a tertiary center. In this cohort of medically refractory patients with CD, treatment with upadacitinib resulted in subjective and objective responses in 25 and 42% of patients, respectively. Even at doses that are considered lower than currently being studied for CD, upadacitinib was associated with a favorable benefit-to-risk profile.
Upadacitinib Was Efficacious and Well-tolerated Over 30 Months in Patients With Crohn's Disease in the CELEST Extension Study. [2022]The long-term efficacy and safety of upadacitinib was evaluated in an open-label extension (OLE) of a phase II, double-blind, randomized trial of patients with Crohn's disease.