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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

AbbVie Deutschland GmbH & Co. KG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

11 Sep 2024 → ongoing

Decision date (initial)

2024-08-05

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

AbbVie Inc.

External identifiers

EU CT number

2023-509618-12-00

ClinicalTrials.gov

NCT06332534

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Safety

To demonstrate efficacy of upadacitinib based on a higher rate of participants who achieve the co-primary endpoints of clinical remission per Pediatric Crohn's Disease Activity Index (PCDAI) and endoscopic response at Week 64 in pediatric participants based on the modified intent-to-treat population (consisting of all participants who achieved clinical response per PCDAI at Week 12 and were randomized into the maintenance phase) of at least the upadacitinib 30 mg adult equivalent dose when compared to external placebo

Conditions and MedDRA coding

Crohn's Disease (CD)

Regulatory references

Scientific advice from competent authorities

Pharmaceuticals And Medical Devices Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-001741-PIP03-16

Plan to share IPD

Yes

IPD plan description

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. To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/ For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Weight at Screening and Baseline must be ≥ 10 kg
2. 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.
3. 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
4. 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 and South Korea, participants must have demonstrated an inadequate response, loss of response, or intolerance to one or more anti-TNFs (tumor necrosis factor).

Exclusion criteria 9

1. History of: A diagnosis of CD prior to 2 years of age.
2. Currently known complications of CD such as: Active abscess (abdominal or perianal);
3. Currently known complications of CD such as: Symptomatic bowel strictures
4. Currently known complications of CD such as: More than 2 missing segments of the following 5 intestinal segments: terminal ileum, right colon, transverse colon, sigmoid and left colon, and rectum
5. Currently known complications of CD such as: Surgical bowel resection within the past 3 months prior to Baseline, or a history of more than 3 bowel resections.
6. History of Fulminant colitis or toxic megacolon
7. History of 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)
8. Current diagnosis of any primary immune deficiency
9. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Achievement of 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
2. Achievement of endoscopic response at Week 64 in participants who achieved clinical response per PCDAI at Week 12.
3. Number of Participants with Adverse Events

Secondary endpoints 7

1. Achievement of clinical remission per PCDAI at week 12
2. Achievement of endoscopic response at week 12
3. Achievement of endoscopic remission at week 12
4. Achievement of clinical response per PCDAI at week 12
5. Achievement of clinical response per PCDAI at week 64 in participants who achieved clinical response per PCDAI at Week 12
6. Achievement of endoscopic remission at Week 64 in participants who achieved clinical response per PCDAI at Week 12
7. Achievement of corticosteroid (CS)-free clinical remission per PCDAI at Week 64 in participants who achieved clinical response per PCDAI at Week 12

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Auxiliary 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

AbbVie Deutschland GmbH & Co. KG

Sponsor organisation

AbbVie Deutschland GmbH & Co. KG

Address

Knollstrasse

City

Ludwigshafen Am Rhein

Postcode

67061

Country

Germany

Scientific contact point

Organisation

AbbVie Deutschland GmbH & Co. KG

Contact name

Global Clinical Trials Helpdesk

Public contact point

Organisation

AbbVie Deutschland GmbH & Co. KG

Contact name

Global Clinical Trials Helpdesk

Third parties 8

Locations

7 EU/EEA countries · 25 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Corrective measures 1 · Art. 77 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 136 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications