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

Phase 3RecruitingNCT04528082

Amgen60 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Behçet Disease

Interventions

ApremilastDRUG

Participants will receive apremilast orally.

PlaceboDRUG

Participants will receive the matching placebo orally.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 17 Years

Medical Language ↔ Plain English

Key Inclusion Criteria * 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. * 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 * Behçet's disease-related active major organ involvement - pulmonary (eg, pulmonary artery aneurysm), vascular (eg, thrombophlebitis), gastrointestinal (eg, ulcers along the gastrointestinal tract), or CNS (eg, meningoencephalitis) manifestations, or 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).

Locations (27)

Outcomes

Primary Outcomes

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

Secondary Outcomes

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 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 12

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 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

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.

Time frame: Week 12

Change from Week 0 to Week 12 in Disease Activity

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.

Central Contacts

Amgen Call Center

CONTACT

866-572-6436 medinfo@amgen.com

Data from ClinicalTrials.gov

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Hospices Civils de Lyon Hopital Femme Mere Enfant

Bron, France

RECRUITING

Hopital Necker Enfants Malades

Paris, France

TERMINATED

Hopital Robert Debre

Paris, France

RECRUITING

Agia Sofia Children Hospital

Athens, Greece

RECRUITING

Attikon University General Hospital

Athens, Greece

RECRUITING

General Hospital of Thessaloniki Ippokrateio

Thessaloniki, Greece

RECRUITING

Meir Medical Center

Kfar Saba, Israel

RECRUITING

Ospedale Santissima Annunziata

Chieti, Italy

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, Italy

RECRUITING

Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano Pini

Milan, Italy

RECRUITING

...and 17 more locations

Time frame: Week 0 to Week 12

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)

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.

Time frame: Week 0 to Week 12

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)

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

Time frame: Up to Week 56

Change from Week 0 to Week 52 in Tanner Staging

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.

Time frame: Week 0 to Week 52

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)

BMI assessed as weight/(height/100)\^2

Time frame: Week 0 to Week 56

Plasma Concentrations of Apremilast

Time frame: Up to Week 52

Taste and Acceptability of Apremilast

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.

Time frame: Week 0 and Week 2

Proportion of Participants Who Require Protocol-prohibited Medications due to Worsening of Behçet's Disease

Time frame: Week 0 to Week 12