NCT04528082RecruitingPhase 3
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)
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Amgen
- Enrollment
- 60
- Locations
- 54
- Primary Endpoint
- Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 2 Years to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
- •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).
Arms & Interventions
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.
Intervention: Apremilast (Drug)
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.
Intervention: Apremilast (Drug)
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.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
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 Outcomes
- Complete Response Rate for Genital Ulcers(Week 12)
- 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)
- Change from Week 0 to Week 12 in Disease Activity(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)(Week 12)
- Change from Week 0 to Week 12 on the Short Form Survey (SF-10)(Week 0 to Week 12)
- Number of Participants with a Treatment-emergent Adverse Event(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)(Up to Week 56)
- Change from Week 0 to Week 52 in Tanner Staging(Week 0 to Week 52)
- Change in Body Weight Measurements(Week 0 to Week 56)
- Change in Height Measurements(Week 0 to Week 56)
- Change in Body Mass Index (BMI)(Week 0 to Week 56)
- Plasma Concentrations of Apremilast(Up to Week 52)
- Taste and Acceptability of Apremilast(Week 0 and Week 2)
- Proportion of Participants Who Require Protocol-prohibited Medications due to Worsening of Behçet's Disease(Week 0 to Week 12)
Investigators
Study Sites (54)
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