Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease
ConditionBehçet Disease
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-17
SponsorAmgen
About this trial
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
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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
Design model
Parallel
Treatments tested in this trial
Apremilast
DrugParticipants will receive apremilast orally.
Placebo
DrugParticipants will receive the matching placebo orally.
Treatment groups
60 Participants
are divided into 2 treatment groupsGroup A: ApremilastExperimental treatment 1 intervention
Group B: Placebo to ApremilastPlacebo comparator 2 interventions
Trial outcomes
Primary outcomes
1
Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12)
Time frame
Week 0 to Week 12Secondary outcomes
1
Number of Oral Ulcers from Week 0 to Week 12
Time frame
Week 0 to Week 122
Change from Week 0 to Week 12 in the Pain of Oral Ulcers in Participants 5 Years of Age and Older
Pain of oral ulcers will be measured by a visual analog scale (VAS). The participants will be asked to place a vertical line on a 100 mm VAS at the point that represents the severity of oral ulcer pain. The scale will range from "no pain" (left hand boundary) to "worst possible pain" (right hand boundary).
Time frame
Week 0 to Week 123
Complete Response Rate for Oral Ulcers
Complete response rate for oral ulcers is defined as the proportion of participants who are oral ulcer free at Week 12.
Time frame
Week 124
Proportion of Participants at Week 12 Whose Number of Oral Ulcers is Reduced by Greater Than or Equal to 50% from Week 0
Time frame
Week 0 to Week 12 Source ClinicalTrials.gov