Clinical trial NCT04528082: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Behçet Disease
Keywords: Behçet's Disease
Oral ulcers
Apremilast
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2020-08-27
Enrollment: 60
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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.
Primary purpose: TREATMENT

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.


Eligibility criteria: Key Inclusion Criteria

* Male or Female participants 2 to \< 18 years of age at randomization.
* Diagnosed with behçet's disease (BD) meeting the International Study Group for Behçet Disease (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.

Key Exclusion Criteria

* Behcet's disease-related active major organ involvement - pulmonary (eg, pulmonary artery aneurysm), vascular (eg, thrombophlebitis), gastrointestinal (eg, ulcers along the gastrointestinal tract), and central nervous system (CNS) (eg, meningoencephalitis) manifestations, and 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).

Primary outcomes:
measure: Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12)
timeFrame: Week 0 to Week 12
Secondary outcomes:
measure: Number of Oral Ulcers from Week 0 to Week 12
timeFrame: Week 0 to Week 12
measure: Change from Week 0 to Week 12 in the Pain of Oral Ulcers
timeFrame: Week 0 to Week 12
measure: Complete Response Rate for Oral Ulcers
timeFrame: Week 12
measure: Proportion of Participants at Week 12 Whose Number of Oral Ulcers is Reduced by Greater Than or Equal to 50% from Week 0
timeFrame: Week 0 to Week 12
measure: Complete Response Rate for Genital Ulcers
timeFrame: Week 12
measure: Change from Week 0 to Week 12 in Disease Activity
timeFrame: Week 0 to Week 12
measure: Proportion of Participants at Week 12 Who Have New-onset or Recurrence of Behçet's-related Manifestations (Other than Oral and Genital Ulcers)
timeFrame: Week 12
measure: Change from Week 0 to Week 12 on the Short Form Survey (SF-10)
timeFrame: Week 0 to Week 12
measure: Number of Participants with a Treatment-emergent Adverse Event
timeFrame: Up to Week 56
measure: Occurrence, Severity, and Frequency of Suicide/Suicide-related Ideations and Behaviors as Assessed by the Columbia Suicide Severity Rating Scale (C-SSRS)
timeFrame: Up to Week 56
measure: Change from Week 0 to Week 52 in Tanner Staging
timeFrame: Week 0 to Week 52
measure: Change in Body Weight Measurements
timeFrame: Week 0 to Week 56
measure: Change in Height Measurements
timeFrame: Week 0 to Week 56
measure: Change in Body Mass Index (BMI)
timeFrame: Week 0 to Week 56
measure: Plasma Concentrations of Apremilast
timeFrame: Up to Week 52
measure: Taste and Acceptability of Apremilast
timeFrame: Week 0 and Week 2
measure: Proportion of Participants Who Require Protocol-prohibited Medications due to Worsening of Behçet's Disease
timeFrame: Week 0 to Week 12
Other outcomes:
Locations:
city: Bron cedex
country: France
facility: Hospices Civils de Lyon Hopital Femme Mere Enfant
city: Paris
country: France
facility: Hopital Necker Enfants Malades
city: Paris
country: France
facility: Hopital Robert Debre
city: Athens
country: Greece
facility: Agia Sofia Children Hospital
city: Athens
country: Greece
facility: Attikon University General Hospital
city: Thessaloniki
country: Greece
facility: General Hospital of Thessaloniki Ippokrateio
city: Kfar Saba
country: Israel
facility: Meir Medical Center
city: Chieti
country: Italy
facility: Ospedale Santissima Annunziata
city: Genova
country: Italy
facility: IRCCS Istituto Giannina Gaslini
city: Milano
country: Italy
facility: Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano Pini
city: Roma
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino Gesu
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
state: Andalucía
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
state: Cataluña
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Deu
state: Cataluña
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
state: Comunidad Valenciana
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Lausanne
country: Switzerland
facility: Centre Hospitalier Universitaire Vaudois
city: Ankara
country: Turkey
facility: Hacettepe Universitesi Tip Fakultesi Hastanesi
city: Istanbul
country: Turkey
facility: Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
city: Istanbul
country: Turkey
facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi
city: Istanbul
country: Turkey
facility: Umraniye Egitim ve Arastirma Hastanesi
city: Izmir
country: Turkey
facility: Dokuz Eylul Universitesi Tip Fakultesi Hastanesi
city: Kayseri
country: Turkey
facility: Erciyes Universitesi Tip Fakultesi Hastanesi
city: Birmingham
country: United Kingdom
facility: Birmingham Childrens Hospital
city: Liverpool
country: United Kingdom
facility: Alder Hey Childrens Hospital
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: