Crohn's Disease
Phase 3 Upadacitinib Study for Children With Active Crohn’s Disease
This study is investigating the efficacy, safety, and drug levels of oral upadacitinib in children ages 2–17 with moderately to severely active Crohn’s disease and an inadequate response, intolerance, or contraindication to specified treatments.
Registry title: Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
12 recruiting U.S. sites ↓Study at a glance
- Age
- 2 Years–17 Years
- Treatment
- Upadacitinib
- Design
- Randomized · Quadruple
- Central study contact
- ABBVIE CALL CENTER844-663-3742abbvieclinicaltrials@abbvie.com
- Sponsor
- AbbVie
Research question
How effective and safe is oral upadacitinib for children with moderately to severely active Crohn’s disease, and how does the drug behave in their bodies?
Participant snapshot
Who the study is looking for
- The study is looking for children ages 2–17 with Crohn’s disease.
- The study is looking for participants who weigh at least 10 kilograms at screening and baseline.
- The study is looking for moderately to severely active disease confirmed by Pediatric Crohn’s Disease Activity Index and endoscopic findings.
- The study is looking for participants with an inadequate response, loss of response, intolerance, or medical contraindication to corticosteroids, immunomodulators, and/or biologic therapy.
- The study is enrolling through listed sites in multiple countries; the study team must confirm whether a particular site is currently accepting participants.
Participation overview
What participation may involve
Participants receive oral upadacitinib during a 12-week induction phase. Subsequent treatment depends on clinical response and may include blinded maintenance, extended open-label treatment, rescue therapy, or a long-term extension. What participation may involve: - Take an upadacitinib extended-release tablet once daily or oral solution twice daily, at approximately the same time each day and with or without food. - Attend regular weekly or monthly hospital or clinic visits. - Complete medical assessments, blood tests, side-effect checks, and questionnaires. - Complete assessments of Crohn’s disease activity and endoscopic response or remission at reported study time points. - Have a 30-day safety follow-up after discontinuing from the study at any time. The registry describes a 12-week induction phase, a 52-week maintenance phase, and an open-label extension lasting up to 156 weeks, plus 30 days of safety follow-up after discontinuation. The registry says visits are regular and may occur weekly or monthly at a hospital or clinic, but it does not give the visit-by-visit schedule.
Study interventions
What participants may receive or do
- Upadacitinib: Participants receive upadacitinib as an oral solution or extended-release tablet. The registry describes once-daily tablets or twice-daily oral solution, with some doses based on body weight.
Study design
How the comparison works
This phase 3 treatment study begins with open-label upadacitinib induction, followed by randomized, blinded maintenance for responders and then an open-label long-term extension. Participants who have a clinical response after the 12-week induction phase are randomly assigned to upadacitinib Dose B or Dose C for the 52-week maintenance phase. Induction and the long-term extension are open label. During maintenance, the participant, care provider, investigator, and outcomes assessor are masked to whether Dose B or Dose C is assigned. The blinded maintenance phase compares two upadacitinib dose groups, Dose B and Dose C. No placebo arm is listed; every registry arm includes upadacitinib.
Reported activities
Procedures and tests
- Screening colonoscopy to confirm Crohn’s disease and exclude current infection, colonic dysplasia, and malignancy.
- Central review of the Simple Endoscopic Score for Crohn’s Disease to document mucosal inflammation.
- Pediatric Crohn’s Disease Activity Index assessments covering symptoms, functioning, laboratory measures, growth, abdominal and perirectal findings, and disease outside the intestine.
- Blood tests during study follow-up.
- Medical assessments and checks for side effects.
- Participant questionnaires.
- Endoscopic assessments used to evaluate response and remission at reported outcome time points.
Eligibility highlights
Details that may affect whether you contact the study
These are selected highlights, not a complete eligibility check. Exact criteria remain in the full registry record below.
Common requirements
- Participants must be 2 through 17 years old.
- Weight must be at least 10 kilograms at both screening and baseline.
- Crohn’s disease must be moderate to severe, with a Pediatric Crohn’s Disease Activity Index above 30 and centrally reviewed endoscopic evidence meeting the specified score threshold.
- The Crohn’s disease diagnosis must be documented before baseline, confirmed by screening colonoscopy, and supported by appropriate biopsy documentation; current infection, colonic dysplasia, and malignancy must be excluded.
- Participants must have had an inadequate response, loss of response, intolerance, or medical contraindication to corticosteroids, immunomodulators, and/or biologic therapy.
- In the United States and South Korea, participants must specifically have had an inadequate response, loss of response, or intolerance to at least one anti-tumor necrosis factor treatment.
Possible reasons someone may not be able to join
- A Crohn’s disease diagnosis before age 2 excludes participation.
- An active abdominal or perianal abscess excludes participation.
- Symptomatic bowel strictures exclude participation.
- An ostomy or ileoanal pouch excludes participation.
- Bowel resection within the three months before baseline, or a history of more than three bowel resections, excludes participation.
- A current diagnosis of ulcerative colitis, indeterminate colitis, or monogenic inflammatory bowel disease excludes participation.
- Fulminant colitis or toxic megacolon excludes participation.
- A history of specified gastrointestinal perforation, diverticulitis, or a significantly increased perforation risk in the investigator’s judgment excludes participation.
- A current primary immune deficiency excludes participation.
- Conditions that could interfere with drug absorption, including short bowel syndrome or gastric bypass surgery, may exclude participation; prior gastric banding or segmentation alone does not.
Important unknowns
What the record does not make clear
- The registry describes regular weekly and monthly clinic visits but does not provide a visit-by-visit schedule or explain how frequency changes across phases.
- The record does not clearly state which current Crohn’s disease medicines may continue during the study.
- The record does not report medication washout requirements or timing.
- The registry mentions open-label Dose C rescue therapy during maintenance but does not fully describe when rescue begins, how it is monitored, or what happens if it does not work.
- The record requires screening colonoscopy and reports endoscopic outcomes at Weeks 12 and 64, but it does not clearly provide the full endoscopy schedule or preparation and sedation details.
- The record does not state which study-related treatments, tests, or other care are paid for or billed to insurance.
- The record does not state whether participants or families receive compensation.
- The record does not describe reimbursement or support for travel, lodging, meals, or childcare.
- The record says visits occur at a hospital or clinic but does not say whether any visits or assessments may be completed remotely.
- The record does not explain whether upadacitinib may remain available after study treatment ends.
- The overall study is recruiting and many listed sites are recruiting, but some sites are completed; the record does not guarantee that a particular site or age and weight cohort remains open.
Before contacting the site
Questions for the study team
- What is the full visit schedule for induction, maintenance, and extension, and how long does each visit usually take?
- Which current Crohn’s disease medicines must stop, may continue, or may change during each study phase?
- How many colonoscopies or other endoscopic procedures are required, and what preparation, anesthesia, or sedation is involved?
- What criteria trigger extended induction, rescue treatment, discontinuation, or movement into the long-term extension?
- Which study-related treatments, tests, travel expenses, and other costs are covered, and is compensation available?
- Can any visits or assessments be completed remotely or coordinated with a local clinician?
- Is the nearest listed site currently enrolling the relevant age and weight group?
- What treatment and follow-up options are available after study treatment ends?
Before changing care
Questions for your gastroenterologist
- How stable is the child’s Crohn’s disease now, and what are the medical risks of changing current treatment for screening or study participation?
- What approved treatment alternatives remain, and how do their expected burdens and risks compare with the study procedures?
- Are there concerns in the child’s medical history—such as strictures, abscesses, surgery, infection, immune deficiency, or absorption problems—that should be discussed with the research team?
- How should the gastroenterologist and research team coordinate disease monitoring, urgent symptoms, rescue treatment, and routine care?
This plain-language digest is provided by the Aidy clinical trials API. It may omit details and is not medical advice or an eligibility decision. Review the full registry record and confirm details with the study team.
Source record
Full registry record
The sections below preserve the study information supplied through ClinicalTrials.gov, including complete descriptions, criteria, outcomes, and locations.
About this study
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.
Study design and administration
- Organization
- AbbVie
- Organization class
- Industry
- Organization study ID
- M14-671
- Lead sponsor
- AbbVie
- Sponsor class
- Industry
- Enrollment type
- Estimated
- Allocation
- Randomized
- Intervention model
- Sequential
- Primary purpose
- Treatment
- Masking
- Quadruple
- Who is masked
- Participant, Care Provider, Investigator, Outcomes Assessor
- Standard age groups
- Child
Study arms
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.
Interventions: Drug: Upadacitinib
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)
Interventions: Drug: Upadacitinib
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)
Interventions: Drug: Upadacitinib
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.
Interventions: Drug: Upadacitinib
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.
Interventions: Drug: Upadacitinib
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
Interventions: Drug: Upadacitinib
Interventions
Drug
Upadacitinib
Oral Solution/ Extended-Release Tablets
Eligibility
2 Years–17 Years
All
Not accepted
Inclusion criteria (4)
- Weight at Screening and Baseline must be \>= 10 kgRegistry-derived · unreviewed
- Moderate to severe Crohn's Disease (CD) defined as Pediatric Crohn's Disease Activity Index (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.Registry-derived · unreviewed
- 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 availableRegistry-derived · unreviewed
- Demonstrated an inadequate response, loss of response, or intolerance to corticosteroids, immunomodulators (IMMs), and/or biologic therapy or in whom use of those therapies is medically contraindicated. For participants in the US and South Korea, participants must have demonstrated an inadequate response, loss of response, or intolerance to one or more anti-TNFs (tumor necrosis factor).Registry-derived · unreviewed
Exclusion criteria (15)
- History of:Registry-derived · unreviewed
- A diagnosis of CD prior to 2 years of age.Registry-derived · unreviewed
- Currently known complications of CD such as:Registry-derived · unreviewed
- Active abscess (abdominal or perianal);Registry-derived · unreviewed
- Symptomatic bowel strictures;Registry-derived · unreviewed
- More than 2 missing segments of the following 5 intestinal segments: terminal ileum, right colon, transverse colon, sigmoid and left colon, and rectum;Registry-derived · unreviewed
- Ostomy or ileoanal pouch;Registry-derived · unreviewed
- Surgical bowel resection within the past 3 months prior to Baseline, or a history of more than 3 bowel resections.Registry-derived · unreviewed
- 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)Registry-derived · unreviewed
- History of any of the following:Registry-derived · unreviewed
- Current diagnosis of ulcerative colitis (UC), indeterminate colitis, or monogenic inflammatory bowel disease (IBD);Registry-derived · unreviewed
- Fulminant colitis or toxic megacolon;Registry-derived · unreviewed
- Gastrointestinal (GI) 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);Registry-derived · unreviewed
- Current diagnosis of any primary immune deficiencyRegistry-derived · unreviewed
- 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.Registry-derived · unreviewed
This information can identify a possible match, conflict, or item needing confirmation. Only the study team can determine eligibility.
Study outcomes
Primary outcome
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
Time frame: At Week 64
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.
Primary outcome
Achievement of endoscopic response at Week 64 in participants who achieved clinical response per PCDAI at Week 12.
Time frame: At Week 64
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.
Primary outcome
Number of Participants with Adverse Events
Time frame: Through Week 156
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.
Secondary outcome
Achievement of clinical remission per PCDAI
Time frame: Week 12
Clinical remission per PCDAI is defined as PCDAI ≤ 10.
Secondary outcome
Achievement of endoscopic response
Time frame: Week 12
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.
Secondary outcome
Achievement of endoscopic remission
Time frame: Week 12
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
Secondary outcome
Achievement of clinical response per PCDAI
Time frame: Week 12
Clinical response is defined as reduction in PCDAI of ≥ 15 points from Baseline
Secondary outcome
Achievement of clinical response per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12
Time frame: Week 64
Secondary outcome
Achievement of endoscopic remission at Week 64 in participants who achieved clinical response per PCDAI at Week 12
Time frame: Week 64
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
Secondary outcome
Achievement of corticosteroid (CS)-free clinical remission per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12
Time frame: Week 64
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\[s\]).
Recruiting locations in the United States
UCSF Benioff Children's Hospital - Oakland /ID# 262217
RecruitingOakland, California, 94609, United States
Lucile Packard Children's Hospital /ID# 262193
RecruitingPalo Alto, California, 94304, United States
Children's Hospital Colorado - Aurora /ID# 262207
RecruitingAurora, Colorado, 80045, United States
Connecticut Children's Medical Center - Hartford /ID# 262256
RecruitingHartford, Connecticut, 06106, United States
OSF St. Francis Medical Center /ID# 262192
RecruitingPeoria, Illinois, 61637-0001, United States
Indiana University Health Riley Hospital for Children /ID# 262215
RecruitingIndianapolis, Indiana, 46202, United States
Boston Children's Hospital /ID# 262191
RecruitingBoston, Massachusetts, 02115, United States
MNGI Digestive Health, P. A. /ID# 262204
RecruitingMinneapolis, Minnesota, 55413-2195, United States
Icahn School of Medicine at Mount Sinai /ID# 262216
RecruitingNew York, New York, 10029, United States
Univ NC Chapel Hill /ID# 262198
RecruitingChapel Hill, North Carolina, 27514-4220, United States
UH Cleveland Medical Center /ID# 262188
RecruitingCleveland, Ohio, 44106, United States
Children's Hospital of Philadelphia - Main /ID# 262197
RecruitingPhiladelphia, Pennsylvania, 19104-4319, United States
This study also lists 73 locations outside the United States. They are not shown here.
Central study contacts
Registry dates
- First posted
- Mar 27, 2024
- Primary completion
- Jun 2027
- Overall completion
- Dec 2034
Trial information comes from ClinicalTrials.gov and may change. Confirm current status, eligibility, and site details with the study team. Aidy does not provide medical advice or determine eligibility.