Apremilast Study in Children with Active Juvenile Psoriatic Arthritis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Amgen Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

31 Mar 2021 → ongoing

Decision date (initial)

2024-04-08

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Amgen Inc.

External identifiers

EU CT number

2023-503435-17-00

EudraCT number

2019-002788-88

WHO UTN

U1111-1295-2072

ClinicalTrials.gov

NCT04804553

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Safety

Estimate the efficacy of apremilast compared with placebo in the treatment of Juvenile Psoriatic Arthritis in pediatric subjects 5 to less than 18 years of age

Secondary objectives 9

1. Estimate effect of apremilast compared to placebo on pain in pediatric subjects (5 to less than 18 years) with JPsA
2. Estimate effect of apremilast compared to placebo on ACR Pedi 20/50/70/90 response, respective core components, and on juvenile arthritis disease activity in pediatric subjects with JPsA
3. Estimate impact on HRQoL in pediatric subjects with JPsA
4. Estimate efficacy of apremilast compared to placebo for overall JPsA related disease activity in pediatric subjects with JPsA
5. Estimate effect of apremilast compared to placebo on psoriatic arthritis flares in pediatric subjects with JPsA
6. Estimate effect of apremilast compared to placebo on psoriatic skin disease in pediatric subjects with JPsA
7. Estimate safety and tolerability of apremilast compared with placebo in pediatric subjects with JPsA
8. Characterize pharmacokinetics of apremilast in pediatric subjects with JPsA
9. Evaluate taste and acceptability of apremilast tablet and suspension in pediatric subjects with JPsA

Conditions and MedDRA coding

Juvenile psoriatic arthritis

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-000715-PIP02-11

Plan to share IPD

Yes

IPD plan description

De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request. Information on IPD sharing Access Criteria, Time Frame and Supporting Information Type is available at ClinicalTrials.gov (https://clinicaltrials.gov/study/NCT04804553) and at Amgen Clinical Trials portal (http://www.amgen.com/datasharing).

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Male or Female subjects 5 to less than 18 years of age at the time of randomization.
2. Confirmed diagnosis of JPsA according to the International League of Associations for Rheumatology (ILAR) Edmonton Revision (Petty, 2001) classification criteria of at least 6 months duration: •Arthritis and psoriasis, OR •Arthritis with at least 2 of the following: dactylitis; nail pitting or onycholysis; psoriasis in a first-degree relative
3. Active disease: at least 3 active joints
4. Inadequate response (at least 2 months) or intolerance to at least 1 DMARD, (which may include MTX or biologic agents).
5. Other protocol-defined inclusion criteria apply.

Exclusion criteria 4

1. Rheumatic autoimmune disease other than PsA, including, but not limited to: systemic lupus erythematosus, mixed connective tissue disease, scleroderma, polymyositis, or fibromyalgia.
2. Prior history of or current inflammatory joint disease other than PsA (eg, gout, reactive arthritis, rheumatoid arthritis, ankylosing spondylitis, Lyme disease).
3. Exclusions per ILAR Edmonton Revision (Edmonton, 2001) criteria for JPsA include: rthritis in an HLA-B27-positive male with arthritis onset after 6 years of age; Ankylosing spondylitis, sacroiliitis with inflammatory bowel disease, Reiter's syndrome, acute anterior uveitis, or a history of one of these disorders in a first-degree relative; History of IgM rheumatoid factor on at least 2 occasions at least 3 months apart; Presence of systemic JIA
4. Other protocol-defined exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of participants achieving ACR Pedi 30 from baseline (week 0) to week 16

Secondary endpoints 7

1. Change in subject's assessment of pain (a)
2. Number of participants achieving ACR Pedi 20/50/70/90 (a)
3. Change in Childhood Heath Assessment Questionnaire (CHAQ) (a)
4. Change in Juvenile Arthritis Disease Activity Score (JADAS) (a)
5. Refer to protocol for fully list of secondary endpoints.
6. PASI-75 response at week 16 for subjects with a baseline psoriasis BSA equal or more than 3% (a)
7. Number of participants who experience PsA flares (a)

Investigational products

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

Test 4

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Amgen Inc.

Sponsor organisation

Amgen Inc.

Address

1 Amgen Center Drive

City

Thousand Oaks

Postcode

91320-1799

Country

United States

Scientific contact point

Organisation

Amgen Inc.

Contact name

Medical Information

Public contact point

Organisation

Amgen Inc.

Contact name

Medical Information

Third parties 11

Locations

12 EU/EEA countries · 36 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Documents 280 files

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

Application history

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