Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease (BEAN)
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|ClinicalTrials.gov Identifier: NCT04528082|
Recruitment Status : Recruiting
First Posted : August 27, 2020
Last Update Posted : March 11, 2022
|Condition or disease||Intervention/treatment||Phase|
|Behçet Disease||Drug: Apremilast Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||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.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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)|
|Actual Study Start Date :||September 9, 2021|
|Estimated Primary Completion Date :||February 23, 2028|
|Estimated Study Completion Date :||January 1, 2029|
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.
Participants will receive apremilast orally.
Other Name: Otezla®
Placebo Comparator: Placebo to Apremilast
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.
Participants will receive apremilast orally.
Other Name: Otezla®
Participants will receive the matching placebo orally.
- Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12) [ Time Frame: Week 0 to Week 12 ]
- Number of Oral Ulcers from Week 0 to Week 12 [ Time Frame: Week 0 to Week 12 ]
- Change from Week 0 to Week 12 in the Pain of Oral Ulcers [ Time Frame: Week 0 to Week 12 ]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).
- Complete Response Rate for Oral Ulcers [ Time Frame: Week 12 ]Complete response rate for oral ulcers is defined as the proportion of participants who are oral ulcer free at Week 12.
- 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 ]
- Complete Response Rate for Genital Ulcers [ Time Frame: Week 12 ]Complete response rate for genital ulcers is defined as the proportion of participants (with genital ulcers at week 0) who are genital ulcer free at Week 12.
- Change from Week 0 to Week 12 in Disease Activity [ Time Frame: Week 0 to Week 12 ]Disease activity is measured by Behçet's Disease Current Activity (BDCAF) scores. The BDCAF consists of 3 component scores: the Behçet's Disease Current Activity Index (BDCAI) score, the Patient's Perception of Disease Activity, and the Clinician's Overall Perception of Disease Activity. The BDCAI score ranges from 0 to 12. A higher score indicates higher level of disease activity (worsening), and a negative change from baseline indicates improvement. The Patient's Perception of Disease Activity and the Clinician's Overall Perception of Disease Activity were assessed by the subject and the clinician, respectively, using a scale of 1 to 7, where a higher score indicates a higher level of disease activity.
- Proportion of Participants at Week 12 Who Have New-onset or Recurrence of Behçet's-related Manifestations (Other than Oral and Genital Ulcers) [ Time Frame: Week 12 ]
- Change from Week 0 to Week 12 on the Short Form Survey (SF-10) [ Time Frame: Week 0 to Week 12 ]The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning.
- Number of Participants with a Treatment-emergent Adverse Event [ Time Frame: Up to Week 56 ]
- Occurrence, Severity, and Frequency of Suicide/Suicide-related Ideations and Behaviors as Assessed by the Columbia Suicide Severity Rating Scale (C-SSRS) [ Time Frame: Up to Week 56 ]
The C-SSRS is an assessment tool that evaluates suicidal ideation and behavior. Number of participants with suicidal ideation or behavior is defined as the number of participants who answer "yes" at any time during the study (up to end of safety follow-up, Week 56) to one of the 10 categories:
Category 1: Wish to be dead Category 2: Non-specific active suicidal thoughts Category 3: Active suicidal ideation with any methods (not plan) without intent to act Category 4: Active suicidal ideation with some intent to act, without specific plan Category 5: Active suicidal ideation with specific plan and intent Category 6: Preparatory acts or behavior Category 7: Aborted attempt Category 8: Interrupted attempt Category 9: Actual attempt (non-fatal) Category 10: Completed suicide
- Change from Week 0 to Week 52 in Tanner Staging [ Time Frame: Week 0 to Week 52 ]Tanner Staging of sexual development assessment will be used to assess sexual maturity. Tanner Staging assessment consists of 3 domains (pubic hair, breast development, and other changes) for girls and 4 domains (pubic hair, penis development, testes development, and other changes) for boys. Stages range from 1-5, with 1 indicating preadolescent and 5 adult.
- Change in Body Weight Measurements [ Time Frame: Week 0 to Week 56 ]
- Change in Height Measurements [ Time Frame: Week 0 to Week 56 ]
- Change in Body Mass Index (BMI) [ Time Frame: Week 0 to Week 56 ]BMI assessed as weight/(height/100)^2
- Plasma Concentrations of Apremilast [ Time Frame: Up to Week 52 ]
- Taste and Acceptability of Apremilast [ Time Frame: Week 0 and Week 2 ]Taste and acceptability will be assessed using a questionnaire with a 7-point faces Likert Scale, with 1 ranging from "super bad" to 7 "super good" and questions to determine whether the participants are able to take the treatment medication.
- Proportion of Participants Who Require Protocol-prohibited Medications due to Worsening of Behçet's Disease [ Time Frame: Week 0 to Week 12 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04528082
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