Phase 3, Open-label Apremilast Pediatric Long-term Extension Study in Children with Oral Ulcers Associated With Behçet’s Disease or Juvenile Psoriatic Arthritis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Amgen Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

3 Oct 2019 → ongoing

Decision date (initial)

2024-01-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Amgen Inc.

External identifiers

EU CT number

2023-503433-21-00

EudraCT number

2022-003024-41

WHO UTN

U1111-1295-2162

ClinicalTrials.gov

NCT05767047

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

Evaluate the long-term safety of apremilast in subjects 2 years of age or older with oral ulcers associated with Behçets disease or 5 years of age or older with active JPsA that have completed Study 20190530 or Study 20190529

Conditions and MedDRA coding

Behçet’s disease is a chronic, relapsing, multisystem inflammatory disorder of unknown etiology characterized by 4 major symptoms (oral aphthous ulcers, genital ulcers, skin lesions, and ocular lesions) and occasionally by 5 minor symptoms (arthritis, gastrointestinal ulcers, epididymitis, vascular lesions, and central nervous system [CNS] symptoms) (Cho, 2012). Juvenile idiopathic arthritis is a heterogeneous group of chronic inflammatory disorders that initially presents in children by the age of 16 years. It refers to arthritis of at least 6 weeks duration of unknown etiology that begins in children less than 16 years old. Juvenile idiopathic arthritis has an annual incidence of 2 to 20 cases per 100 000 population and a prevalence of 16 to 150 cases per 100 000 population. It is an autoimmune disorder marked by abnormalities of immune responses (Adelowo, 2010).

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-000715-PIP02-11, EMEA-000715-PIP05-13

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/NCT05767047) 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. Subject’s legally authorized representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
2. Subject must be < 18 years of age at the time the informed consent document is signed and have completed week 52 (Apremilast Active TreatmenPhase) of Study 20190529 (from a country without another mechanism for post-trial access to apremilast for JPsA) or Study 20190530, where drug is not approved for the subject’s pediatric therapeutic indication in their country.
3. Subject must have an age and sex specific body mass index (BMI) value no lower in range than the 5th percentile on the Centers for Disease Control (CDC) growth chart (Appendix 11.7) for children and adolescents (CDC, 2000) at enrollment.
4. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
5. Subject must have acceptable benefit/risk for continued treatment with apremilast.

Exclusion criteria 6

1. Answer “Yes” to any question on the Columbia-Suicide Severity Rating Scale (C-SSRS) at the week 52 visit on Study 20190529 (subjects from France and Turkey sites only) or Study 20190530.
2. Scheduled surgery or other interventions that would interrupt the subject’s participation in the study.
3. Female subjects of childbearing potential (for the purpose of this study, a female subject is considered of childbearing potential if she is ³ 12 years old or has reached menarche, whichever occurred first) unwilling to use protocol specified method of contraception see Appendix 5 (Section 11.5) during treatment and for an additional 30 days after the last dose of investigational product.
4. Female subjects planning to become pregnant while on study through 30 days after the last dose of investigational product.
5. Female subjects of childbearing potential with a positive pregnancy test assessed at week 0 by a highly sensitive urine or serum pregnancy test.
6. Subject has known sensitivity to any of the products to be administered during dosing.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. Adverse events: Type, frequency, severity, and relationship to apremilast
2. Columbia-Suicide Severity rating Scale (C-SSRS)
3. Tanner Staging
4. Body weight, height, and body mass index (BMI)
5. Vital signs and laboratory parameters

Investigational products

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

Test 3

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 2

Locations

3 EU/EEA countries · 12 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 35 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