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

Amgen

Status and phase: Enrolling; Phase 3

Conditions: Behçet Disease

Treatments: Drug: Placebo; Drug: Apremilast

Study type: Interventional

Funder types: Industry

Identifiers

NCT04528082

20190530

2019-002787-27 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 60 estimated patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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60 participants in 2 patient groups, including a placebo group

Apremilast

Experimental group

Description:

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.

Treatment:

Drug: Apremilast

Placebo to Apremilast

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo

Drug: Apremilast