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

Behçet Disease Clinical Trial

— BEAN  

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04528082
• Other study ID #: 20190530
• Secondary ID: 2019-002787-27
• Status: Recruiting
• Phase: Phase 3
• Start date: September 9, 2021
• Est. completion date: July 1, 2028

Study information

• Verified date: December 2023
• Source: Amgen
• Contact: Amgen Call Center
• Phone: 866-572-6436
• Email: medinfo[@]amgen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: July 1, 2028
• Est. primary completion date: August 23, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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).

Related Conditions & MeSH terms

• Behçet Disease
• Behcet Syndrome
• Oral Ulcer

Intervention

Drug:
• Apremilast
Participants will receive apremilast orally.
• Placebo
Participants will receive the matching placebo orally.

Locations

Country	Name	City	State
France	Hospices Civils de Lyon Hopital Femme Mere Enfant	Bron cedex
France	Hopital Necker Enfants Malades	Paris
France	Hopital Robert Debre	Paris
Greece	Agia Sofia Children Hospital	Athens
Greece	Attikon University General Hospital	Athens
Greece	General Hospital of Thessaloniki Ippokrateio	Thessaloniki
Israel	Meir Medical Center	Kfar Saba
Italy	Ospedale Santissima Annunziata	Chieti
Italy	IRCCS Istituto Giannina Gaslini	Genova
Italy	Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano Pini	Milano
Italy	IRCCS Ospedale Pediatrico Bambino Gesu	Roma
Spain	Hospital Universitari Vall d Hebron	Barcelona	Cataluña
Spain	Hospital Sant Joan de Deu	Esplugues de Llobregat	Cataluña
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Universitario Ramon y Cajal	Madrid
Spain	Hospital Universitario Virgen del Rocio	Sevilla	Andalucía
Spain	Hospital Universitari i Politecnic La Fe	Valencia	Comunidad Valenciana
Switzerland	Centre Hospitalier Universitaire Vaudois	Lausanne
Turkey	Hacettepe Universitesi Tip Fakultesi Hastanesi	Ankara
Turkey	Istanbul Universitesi Cerrahpasa Tip Fakultesi	Istanbul
Turkey	Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi	Istanbul
Turkey	Umraniye Egitim ve Arastirma Hastanesi	Istanbul
Turkey	Dokuz Eylul Universitesi Tip Fakultesi Hastanesi	Izmir
Turkey	Erciyes Universitesi Tip Fakultesi Hastanesi	Kayseri
United Kingdom	Birmingham Childrens Hospital	Birmingham
United Kingdom	Alder Hey Childrens Hospital	Liverpool

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

Countries where clinical trial is conducted

France,  Greece,  Israel,  Italy,  Spain,  Switzerland,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12)		Week 0 to Week 12
Secondary	Number of Oral Ulcers from Week 0 to Week 12		Week 0 to Week 12
Secondary	Change from Week 0 to Week 12 in the Pain of Oral Ulcers	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).	Week 0 to Week 12
Secondary	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.	Week 12
Secondary	Proportion of Participants at Week 12 Whose Number of Oral Ulcers is Reduced by Greater Than or Equal to 50% from Week 0		Week 0 to Week 12
Secondary	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.	Week 12
Secondary	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.	Week 0 to Week 12
Secondary	Proportion of Participants at Week 12 Who Have New-onset or Recurrence of Behçet's-related Manifestations (Other than Oral and Genital Ulcers)		Week 12
Secondary	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.	Week 0 to Week 12
Secondary	Number of Participants with a Treatment-emergent Adverse Event		Up to Week 56
Secondary	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	Up to Week 56
Secondary	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.	Week 0 to Week 52
Secondary	Change in Body Weight Measurements		Week 0 to Week 56
Secondary	Change in Height Measurements		Week 0 to Week 56
Secondary	Change in Body Mass Index (BMI)	BMI assessed as weight/(height/100)^2	Week 0 to Week 56
Secondary	Plasma Concentrations of Apremilast		Up to Week 52
Secondary	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.	Week 0 and Week 2
Secondary	Proportion of Participants Who Require Protocol-prohibited Medications due to Worsening of Behçet's Disease		Week 0 to Week 12

External Links

•   AmgenTrials clinical trials website