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

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)

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.

Key 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.

Key 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).