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

Official title

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study, Followed by an Active Treatment Phase to Evaluate the Efficacy and Safety of Apremilast in Children From 2 to Less Than 18 Years of Age With Active Oral Ulcers Associated With Behçet's Disease (BEAN)

Overview

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.

Interventions

• Drug Apremilast
  Participants will receive apremilast orally.
• Drug Placebo
  Participants will receive the matching placebo orally.

Primary outcome measures

• Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12) [Time frame: Week 0 to Week 12]

Secondary outcome measures (12)

• Number of Oral Ulcers from Week 0 to Week 12 [Time frame: 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 [Time frame: Week 0 to Week 12]
• Complete Response Rate for Oral Ulcers [Time frame: 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 [Time frame: Week 0 to Week 12]
• Complete Response Rate for Genital Ulcers [Time frame: Week 12]
• Change from Week 0 to Week 12 in Disease Activity [Time frame: Week 0 to Week 12]
• Proportion of Participants at Week 12 Who Have New-onset or Recurrence of Behçet's-related Manifestations (Other than Oral and Genital Ulcers) [Time frame: Week 12]
• Change from Week 0 to Week 12 on the Short Form Survey (SF-10) [Time frame: Week 0 to Week 12]
• Number of Participants with a Treatment-emergent Adverse Event [Time frame: Up to Week 56]
• Occurrence, Severity, and Frequency of Suicide/Suicide-related Ideations and Behaviors as Assessed by the Columbia Suicide Severity Rating Scale (C-SSRS) [Time frame: Up to Week 56]
• Change from Week 0 to Week 52 in Tanner Staging [Time frame: Week 0 to Week 52]
• Change in Body Weight Measurements [Time frame: Week 0 to Week 56]

Eligibility criteria

Inclusion criteria

• 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.

Exclusion criteria

• 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).

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

Spain · 6 centers

• Hospital Universitario Virgen del Rocio — Seville
• Hospital Universitari Vall d Hebron — Barcelona
• Hospital Sant Joan de Deu — Esplugues de Llobregat
• Hospital Universitari i Politecnic La Fe — Valencia
• Hospital Universitario Ramon y Cajal — Madrid
• Hospital Universitario La Paz — Madrid

Turkey (Türkiye) · 6 centers

• Hacettepe Universitesi Tip Fakultesi Hastanesi — Ankara
• Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi — Istanbul
• Istanbul Universitesi Cerrahpasa Tip Fakultesi — Istanbul
• Umraniye Egitim ve Arastirma Hastanesi — Istanbul
• Dokuz Eylul Universitesi Tip Fakultesi Hastanesi — Izmir
• Erciyes Universitesi Tip Fakultesi Hastanesi — Kayseri

Italy · 4 centers

• Ospedale Santissima Annunziata — Chieti
• IRCCS Istituto Giannina Gaslini — Genova
• Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetan — Milan
• IRCCS Ospedale Pediatrico Bambino Gesu — Roma

France · 3 centers

• Hospices Civils de Lyon Hopital Femme Mere Enfant — Bron
• Hopital Necker Enfants Malades — Paris
• Hopital Robert Debre — Paris

Greece · 3 centers

• Agia Sofia Children Hospital — Athens
• Attikon University General Hospital — Athens
• General Hospital of Thessaloniki Ippokrateio — Thessaloniki

United Kingdom · 3 centers

• Birmingham Childrens Hospital — Birmingham
• Alder Hey Childrens Hospital — Liverpool
• John Radcliffe Hospital — Oxford

Israel · 1 center

• Meir Medical Center — Kfar Saba

Switzerland · 1 center

• Centre Hospitalier Universitaire Vaudois — Lausanne

Identifiers

NCT: NCT04528082 · 20190530 · 2023-503436-40-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗