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

Официальное название

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)

Обзор

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.

Вмешательства

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

Первичные конечные точки

• Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12) [Срок оценки: Week 0 to Week 12]

Вторичные конечные точки (12)

• Number of Oral Ulcers from Week 0 to Week 12 [Срок оценки: 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 [Срок оценки: Week 0 to Week 12]
• Complete Response Rate for Oral Ulcers [Срок оценки: 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 [Срок оценки: Week 0 to Week 12]
• Complete Response Rate for Genital Ulcers [Срок оценки: Week 12]
• Change from Week 0 to Week 12 in Disease Activity [Срок оценки: 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) [Срок оценки: Week 12]
• Change from Week 0 to Week 12 on the Short Form Survey (SF-10) [Срок оценки: Week 0 to Week 12]
• Number of Participants with a Treatment-emergent Adverse Event [Срок оценки: 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) [Срок оценки: Up to Week 56]
• Change from Week 0 to Week 52 in Tanner Staging [Срок оценки: Week 0 to Week 52]
• Change in Body Weight Measurements [Срок оценки: Week 0 to Week 56]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Испания · 6 центров

• Hospital Universitario Virgen del Rocio — Seville
• Hospital Universitari Vall d Hebron — Barcelona
• Hospital Sant Joan de Deu — Esplugues de Llobregat
• Hospital Universitari i Politecnic La Fe — Valencia
• Hospital Universitario Ramon y Cajal — Madrid
• Hospital Universitario La Paz — Madrid

Turkey (Türkiye) · 6 центров

• Hacettepe Universitesi Tip Fakultesi Hastanesi — Ankara
• Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi — Istanbul
• Istanbul Universitesi Cerrahpasa Tip Fakultesi — Istanbul
• Umraniye Egitim ve Arastirma Hastanesi — Istanbul
• Dokuz Eylul Universitesi Tip Fakultesi Hastanesi — Izmir
• Erciyes Universitesi Tip Fakultesi Hastanesi — Kayseri

Италия · 4 центра

• Ospedale Santissima Annunziata — Chieti
• IRCCS Istituto Giannina Gaslini — Genova
• Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetan — Milan
• IRCCS Ospedale Pediatrico Bambino Gesu — Roma

Франция · 3 центра

• Hospices Civils de Lyon Hopital Femme Mere Enfant — Bron
• Hopital Necker Enfants Malades — Paris
• Hopital Robert Debre — Paris

Греция · 3 центра

• Agia Sofia Children Hospital — Athens
• Attikon University General Hospital — Athens
• General Hospital of Thessaloniki Ippokrateio — Thessaloniki

Великобритания · 3 центра

• Birmingham Childrens Hospital — Birmingham
• Alder Hey Childrens Hospital — Liverpool
• John Radcliffe Hospital — Oxford

Израиль · 1 центр

• Meir Medical Center — Kfar Saba

Швейцария · 1 центр

• Centre Hospitalier Universitaire Vaudois — Lausanne

Идентификаторы

NCT: NCT04528082 · 20190530 · 2023-503436-40-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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