[M14-671] Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects with Moderately to Severely Active Crohn's Disease

JRCT ID: jRCT2071240040

Registered date:24/07/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Crohn's Disease
• Date of first enrollment: 24/07/2024
• Target sample size: 110
• Countries of recruitment: Australia,Japan,Belgium,Japan,Brazil,Japan,Bulgaria,Japan,Canada,Japan,China,Japan,Czechia,Japan,France,Japan,Greece,Japan,Italy,Japan,New Zealand,Japan,Poland,Japan,Puerto Rico,Japan,South Korea,Japan,Spain,Japan,Taiwan,Japan,Turkey,Japan,United Kingdom,Japan,United States of America,Japan
• Study type: Interventional

Intervention(s)

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.

Period 1: Double-Blind Maintenance Phase (Dose B) ; Description: 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)

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)

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.

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.

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.

Outcome(s)

Primary Outcome

-Percentage of participants with clinical remission per the Pediatric Crohn's Disease Activity Index (PCDAI) at Week 64 in the participants who achieved clinical response per PCDAI at Week 12
-Achievement of endoscopic response at Week 64 in participants who achieved clinical response per PCDAI at Week 12.
-Number of Participants with Adverse Events

Secondary Outcome

-Achievement of clinical remission per PCDAI
-Achievement of endoscopic response at Week 12
-Achievement of endoscopic remission at Week 12
-Achievement of clinical response per PCDAI at Week 12
-Achievement of clinical response per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12
-Achievement of endoscopic remission at Week 64 in participants who achieved clinical response per PCDAI at Week 12
-Achievement of corticosteroid (CS)-free clinical remission per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: <= 17age old
• Gender: Both

Include 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).

Exclude 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.

Related Information

• Primary Sponsor: Otani Tetsuya
• Secondary ID(s): NCT06332534