Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease

NCT04528082 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: 201905302023-503436-40-00
• Study Type: interventional
• Target: 60
• Locations: 8 countries
• Sites: 27

Brief Summary

The aim of this study is to estimate the efficacy of apremilast compared to placebo in the treatment of oral ulcers in pediatric participants from 2 to \< 18 years of age with oral ulcers associated with Behçet's disease (BD) through week 12.

Conditions

Behçet Disease

Keywords

Behçet's Disease; Oral ulcers; Apremilast

Outcome Measures

Primary Outcomes (1)

• Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12)

  Week 0 to Week 12

Secondary Outcomes (17)

• Number of Oral Ulcers from Week 0 to Week 12

  Week 0 to Week 12

• Change from Week 0 to Week 12 in the Pain of Oral Ulcers in Participants 5 Years of Age and Older

  Week 0 to Week 12

• Complete Response Rate for Oral Ulcers

  Week 12

• Proportion of Participants at Week 12 Whose Number of Oral Ulcers is Reduced by Greater Than or Equal to 50% from Week 0

  Week 0 to Week 12

• Complete Response Rate for Genital Ulcers

  Week 12

Study Arms (2)

Apremilast

EXPERIMENTAL

Participants will receive apremilast orally in the double-blind 12 week treatment phase. Then the participants will continue to receive apremilast in the active 40 weeks treatment phase.

Drug: Apremilast

Placebo to Apremilast

PLACEBO COMPARATOR

Participants will receive the matching placebo orally in the double-blind 12 week treatment phase. Then the participants will receive apremilast in the active 40 weeks treatment phase.

Drug: Apremilast; Drug: Placebo

Interventions

Apremilast DRUG

Participants will receive apremilast orally.

Also known as: Otezla®

Apremilast; Placebo to Apremilast

Placebo DRUG

Participants will receive the matching placebo orally.

Placebo to Apremilast

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or Female participants 2 to \< 18 years of age at randomization.
• Diagnosed with BD meeting the ISGBD criteria at any time prior to the screening visit.
• Oral ulcers that occurred ≥ 3 times within the 12-month period prior to the screening visit.
• Participant must have ≥ 2 oral ulcers at both the screening visit and on day 1.
• Participant has had prior treatment with ≥ 1 non-biologic BD therapy, such as, but not limited to, topical corticosteroids or systemic treatment.

You may not qualify if:

• Behçet's disease-related active major organ involvement - pulmonary (eg, pulmonary artery aneurysm), vascular (eg, thrombophlebitis), gastrointestinal (eg, ulcers along the gastrointestinal tract), or CNS (eg, meningoencephalitis) manifestations, or ocular lesions (eg, uveitis) requiring immunosuppressive therapy; however:
• Previous major organ involvement is allowed if it occurred ≥1 year prior to the screening visit and is not active at time of enrollment
• Participants with mild BD-related ocular lesions not requiring systemic immunosuppressive therapy are allowed
• Participants with BD-related arthritis and BD-skin manifestations are also allowed.
• Previous exposure to biologic therapies for the treatment of BD oral ulcers, previous biologic exposure is allowed for other indications (including other manifestations of BD).

Sponsors & Collaborators

• Amgen: lead

Study Sites (27)

Hospices Civils de Lyon Hopital Femme Mere Enfant

Bron, 69677, France

RECRUITING

Hopital Necker Enfants Malades

Paris, 75015, France

TERMINATED

Hopital Robert Debre

Paris, 75019, France

RECRUITING

Agia Sofia Children Hospital

Athens, 11527, Greece

RECRUITING

Attikon University General Hospital

Athens, 12462, Greece

RECRUITING

General Hospital of Thessaloniki Ippokrateio

Thessaloniki, 54642, Greece

RECRUITING

Meir Medical Center

Kfar Saba, 4428164, Israel

RECRUITING

Ospedale Santissima Annunziata

Chieti, 66100, Italy

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

RECRUITING

Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano Pini

Milan, 20122, Italy

RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesu

Roma, 00165, Italy

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Andalusia, 41013, Spain

RECRUITING

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, 08035, Spain

RECRUITING

Hospital Sant Joan de Deu

Esplugues de Llobregat, Catalonia, 08950, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, 46026, Spain

RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

RECRUITING

Hospital Universitario La Paz

Madrid, 28046, Spain

TERMINATED

Centre Hospitalier Universitaire Vaudois

Lausanne, 1011, Switzerland

TERMINATED

Hacettepe Universitesi Tip Fakultesi Hastanesi

Ankara, 06100, Turkey (Türkiye)

RECRUITING

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

Istanbul, 34093, Turkey (Türkiye)

RECRUITING

Istanbul Universitesi Cerrahpasa Tip Fakultesi

Istanbul, 34098, Turkey (Türkiye)

RECRUITING

Umraniye Egitim ve Arastirma Hastanesi

Istanbul, 34764, Turkey (Türkiye)

RECRUITING

Dokuz Eylul Universitesi Tip Fakultesi Hastanesi

Izmir, 35330, Turkey (Türkiye)

TERMINATED

Erciyes Universitesi Tip Fakultesi Hastanesi

Kayseri, 38030, Turkey (Türkiye)

RECRUITING

Birmingham Childrens Hospital

Birmingham, B4 6NH, United Kingdom

TERMINATED

Alder Hey Childrens Hospital

Liverpool, L12 2AP, United Kingdom

RECRUITING

John Radcliffe Hospital

Oxford, OX3 7LD, United Kingdom

RECRUITING

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Behcet Syndrome; Oral Ulcer

Interventions

apremilast

Condition Hierarchy (Ancestors)

Mouth Diseases; Stomatognathic Diseases; Uveitis, Anterior; Panuveitis; Uveitis; Uveal Diseases; Eye Diseases; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vascular

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Central Study Contacts

Amgen Call Center

CONTACT

866-572-6436; medinfo@amgen.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The participants will be randomized to receive apremilast or placebo in the double-blind 12 week treatment phase. Then, the participants will all receive apremilast for a further 40 weeks in the active treatment phase. The participants will then complete the 30 days safety follow-up period after the last dose of apremilast in the active treatment phase. Participants who complete the 52 weeks of treatment may have the opportunity to continue receiving apremilast through an open-label extension study (NCT05767047) . The participants who choose not to participate in the optional open-label, long-term study will complete the 30 days safety follow-up period after the last dose of apremilast in the active treatment phase.

Study Record Dates

• First Submitted: August 24, 2020
• First Posted: August 27, 2020
• Study Start: September 9, 2021
• Primary Completion (Estimated): February 7, 2030
• Study Completion (Estimated): December 17, 2030
• Last Updated: December 1, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Locations

IT (4); ES (6); CH (1); TU (6); GR (3); GB (3); FR (3); IL (1)