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 : April 25, 2023
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|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 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||2 Years to 17 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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).
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
|Contact: Amgen Call Centeremail@example.com|
|Other Study ID Numbers:||
2019-002787-27 ( EudraCT Number )
|First Posted:||August 27, 2020 Key Record Dates|
|Last Update Posted:||April 25, 2023|
|Last Verified:||April 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.|
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
|Time Frame:||Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.|
|Access Criteria:||Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||Yes|
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