Behçets syndrome Silk Road Disease
Conditions
Interventions
Week 0 to week 12 - double-blind, placebo-controlled treatment phase:
- one group will receive apremilast depending on weight (10 mg BID for * 12 kg
to
Sponsors
Amgen
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: Main inclusion criteria: - Male or Female participants 2 to
Exclusion criteria
Exclusion criteria: Main 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), and central nervous system (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 * Subjects with mild BD-related ocular lesions not requiring systemic immunosuppressive therapy are allowed * Subjects 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) - Receipt of concomitant immune modulating therapy (except oral or topical corticosteroids, which must have been tapered during screening as appropriate and discontinued prior to randomization) within specified time based on type of drug. - History or evidence of any other clinically significant disorder, condition or disease that could pose a risk to subject safety or interfere with the study evaluation, procedures or completion. - Female subject who is (or plans to become) pregnant or breastfeeding. - Female subject of child bearing potential unwilling to use 1 highly effective method of contraception. Exclusion criteria are described in more detail in section 5.2 of the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome of the study is to assess the area under the curve for the number of oral ulcers from Week 0 through Week 12 (AUC W0-W12). | — |
Secondary
| Measure | Time frame |
|---|---|
| * Number of oral ulcers from week 0 to week 12. * Change from week 0 to week 12 in the pain of oral ulcers as measured by a visual analog scale (VAS) * Complete response rate for oral ulcers defined as the proportion of subjects who are oral ulcer free at week 12 * Proportion of subjects at week 12 whose number of oral ulcers is reduced by * 50% from week 0 * Complete response rate for genital ulcers defined as the proportion of subjects (with genital ulcers at week 0) who are genital ulcer free at week 12. * Change from week 0 to week 12 in disease activity as measured by Behçet*s Disease Current Activity Form (BDCAF) scores * Proportion of subjects at week 12 who have new-onset (ie, absent at baseline) or recurrence (ie, with history) of Behçet*s-related manifestations (other than oral and genital ulcers) * Change from week 0 to week 12 on the SF-10 (10-item short form survey). * Type, frequency, severity, and relationship to investigational product of adverse events (including malignancies and serious/systemic infections), clinical laboratory tests, vital signs, and physical examination from signing of the informed consent/assent forms through week 52 and the 30 day posttreatment safety follow-up phase. * Occurrence, severity, and frequency of suicide/suicide-related ideations and behaviors as assessed by the C-SSRS at select visits from week 0 through week 52 and the 30 day posttreatment safety follow-up visit. * Tanner staging of sexual development assessment of sexual maturity at week 0 and week 52 (or early termination). * Body weight, height, and BMI at week 0 (baseline) through week 52 and the 30 day posttreatment safety follow-up visit. * Plasma concentrations of apremilast will be summarized by visit and dosing regimen. * Taste and acceptability will be assessed using a 7-point faces Likert Scale and a parent assessment of the subject, including whether the subject was able to swallow the tablet at week 0 and week 2. * Prop | — |
Countries
Netherlands
Outcome results
None listed