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)

20190530

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.

2021-09-09

2027-08-23

2028-07-01

Recruiting

Early phase I

60

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).

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': '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.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 60, 'type': 'ESTIMATED'}}

2023-12-08