Study of Oral Upadacitinib and Subcutaneous/Intravenous Tocilizumab to Evaluate Change in Disease Activity, Adverse Events and how Drug Moves Through the Body of Pediatric and Adolescent Participants With Active Systemic Juvenile Idiopathic Arthritis.

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] - Immune System Diseases [C20]

Trial duration

19 Mar 2024 → ongoing

Decision date (initial)

2024-11-11

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Abbvie Inc

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic

The study objective is to assess efficacy, safety, and pharmacokinetics of upadacitinib with a tocilizumab reference arm in children and adolescent subjects from 1 to < 18 years old with active sJIA

Secondary objectives 1

1. The secondary efficacy objectives are to assess the proportion of subjects who achieve the dichotomous secondary endpoints and the mean of continuous endpoints as specified in Section 3.3 of the protocol, based on the ITT population.

Conditions and MedDRA coding

juvenile idiopathic arthritis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-236245-PIP20-23

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. Subjects and/or their legally authorized representative must be able to understand and be willing to adhere to all protocol requirements and voluntarily sign and date an informed consent (and assent for minors as required by applicable regulation), approved by an independent ethics committee (IEC)/institutional review board (IRB), prior to the initiation of any screening or study-specific procedures. Demographic and Laboratory Assessments
2. Male or female subjects, ages 1 to < 18 years old at Baseline. Note: For Cohort 1, subjects must be ages 2 to < 18 years old in countries where SC tocilizumab is not approved for sJIA.
3. Subjects must have total body weight of 10 kg or higher at the time of Screening.
4. Laboratory values Subjects must meet all of the following criteria within the screening period prior to the first dose of study drug: Serum aspartate transaminase (AST) and alanine aminotransferase (ALT) < 2.0 × upper limit of normal (ULN) for age and sex; Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2 by modified Schwartz equation for Total white blood cell count (WBC) ≥ 3,000/iL; Absolute neutrophil count (ANC) ≥ 2,000/iL; Platelet count ≥ 100,000/iL; Absolute lymphocyte count (ALC) ≥ 750/iL; Hemoglobin ≥ 9 g/dL.

Exclusion criteria 3

1. For Cohort 1, subjects must not have had previous treatment with any IL-6 inhibitor. For Cohort 2, subjects must have an intolerance or inadequate response to an IL-6 inhibitor as judged by the investigator.
2. Subjects must not have uncontrolled severe systemic disease and/or impeding or active MAS within 1 month prior to Baseline.
3. Subjects must have inadequate response to previous treatment with nonsteroidal anti-inflammatory drugs (NSAIDs) and/or systemic glucocorticoids, used per local guidelines and as judged by the investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint only applies to Cohort 1. The primary endpoint is achievement of adapted sJIA ACR 30 response at Week 12 (defined as absence of fever [> 38°C] in the previous 1 week preceding evaluation and improvement of ≥ 30% in ≥ 3 of the 6 variables of the JIA core set [defined in Section 3.3], with no more than 1 variable worsening by > 30%).

Secondary endpoints 1

1. The secondary endpoints only apply to Cohort 1. Secondary Endpoint(s) 1. Achievement of adapted sJIA ACR 50/70/90/100 response at Week 12 2. Change from Baseline in each core adapted sJIA ACR component at Week 12 · Number of joints with active arthritis · Number of joints with limitation of motion · Childhood Health Assessment Questionnaire-Disability Index (CHAQ-DI) · Patient/Parent Global Assessment (Pt/ParentGA) · Physician's global assessment of disease activity (PhGA) · High-sensitivity CRP

Investigational products

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

Test 2

Comparator 3

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 6

Locations

8 EU/EEA countries · 15 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 89 files

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

Application history

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