A Double-blind, Placebo-controlled, Randomized Phase 3 Pivotal Trial to Assess the Efficacy and Safety of Peanut Epicutaneous Immunotherapy With Viaskin Peanut in Peanut-allergic Children
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 500
- 试验地点
- 31
- 主要终点
- Difference in Percentages of Treatment Responders at Month 12; Analyzed in the Overall Population
研究概览
简要总结
The PEPITES study evaluates the efficacy and safety of Viaskin Peanut 250 µg peanut protein to induce desensitization to peanut in peanut-allergic children 4 through 11 years of age after a 12-month treatment by epicutaneous immunotherapy (EPIT).
详细描述
This is a 12-month, Phase III, double-blind, placebo-controlled, randomized study to assess the efficacy and safety of Viaskin Peanut, dosed at 250µg peanut protein (per patch) in peanut-allergic children from 4 through 11 years of age.
The overall maximum study duration for each subject is approximately 61 weeks (6-week screening period, 12-month treatment period and 2-week follow-up period).
During the screening period, subjects will undergo a first screening visit and an entry double-blind, placebo-controlled food challenge (DBPCFC) to peanut to confirm their allergy and their entry peanut eliciting dose (ED). The starting dose of the challenge will be 1 mg peanut protein and will escalate up to a highest dose of 300mg peanut protein. Subjects who react at or below the dose of 300mg peanut protein are considered eligible.
Randomization of eligible subjects will occur in a 2:1 ratio to Viaskin Peanut dosed at 250µg peanut protein (active treatment) or placebo. Subjects will be stratified at randomization by their entry/screening DBPCFC ED in 1 of the following 2 strata and by study center:
- Stratum 1: children with a screening ED of 1mg, 3mg or 10mg;
- Stratum 2: children with a screening ED of 30mg, 100mg or 300mg.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 4 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female children aged 4 through 11 years;
- •Physician-diagnosis of peanut allergy or children with a well documented medical history of IgE-mediated symptoms after ingestion of peanut and currently following a strict peanut-free diet, but without a physician diagnosis;
- •Peanut-specific IgE level (ImmunoCAP system) >0.7 kU/L;
- •Positive peanut skin prick test (SPT) with a largest wheal diameter:
- •≥6 mm for children 4 through 5 years of age at Visit 1,
- •≥8 mm for children 6 years and above at Visit 1;
- •Positive DBPCFC at ≤300 mg peanut protein.
排除标准
- •History of severe anaphylaxis to peanut with any of the following symptoms: hypotension, hypoxia, neurological compromise (collapse, loss of consciousness or incontinence);
- •Generalized dermatologic disease
- •Diagnosis of mast cell disorders, including mastocytosis or uricaria pigmentosa as well as hereditary or idiopathic angioedema;
- •Diagnosis of asthma that fulfills any of the following criteria:
- •Uncontrolled persistent asthma as defined by National Asthma Education and Prevention Program Asthma guidelines 2007 or by Global Initiative for Asthma guidelines 2015,
- •Asthma treated with either a high daily high dose of inhaled corticosteroid or with a combination therapy of a medium or high daily dose of inhaled corticosteroid with a long acting inhaled β2 agonist or with a combination therapy of a high daily dose of inhaled corticosteroid with a long acting inhaled β2 agonist. Asthmatic subjects treated with a medium daily dose of inhaled corticosteroids are eligible. Intermittent asthmatic subjects who require intermittent use of inhaled corticosteroids for rescue are also eligible,
- •Two or more systemic corticosteroid courses for asthma in the past year or 1 oral corticosteroid course for asthma within 3 months prior to Visit 1, or during screening period,
- •Prior intubation/mechanical ventilation for asthma within 1 year prior to Visit 1, or during screening;
- •Receiving β-blocking agents, angiotensin-converting enzyme inhibitors, angiotensin-receptor blockers, calcium channel blockers or tricyclic antidepressant therapy;
- •Received anti-tumor necrosis factor drugs or anti-IgE drugs (such as omalizumab) or any biologic immunomodulatory therapy within 1 year prior to Visit 1, during screening period or during study participation;
- •Use of systemic long-acting corticosteroids within 12 weeks prior to Visit 1 and/or use of systemic short-acting corticosteroids within 4 weeks prior to Visit 1 or during screening;
- •Prior or concomitant history of any immunotherapy to any food;
- •Receiving or planning to receive any aeroallergen immunotherapy during their participation in the study. Aeroallergen immunotherapy must be discontinued at the time of Visit 1;
- •Any disorder in which epinephrine is contraindicated such as coronary artery disease, uncontrolled hypertension, or serious ventricular arrhythmias.
研究组 & 干预措施
Viaskin Peanut 250mcg
One Viaskin epicutaneous delivery system (patch) containing 250 µg of peanut protein applied on the skin for 24 hours (±4 hours of allowance) daily for a period of 12 months.
干预措施: Viaskin Peanut 250mcg (Biological)
Placebo
One Viaskin epicutaneous delivery system (patch) containing placebo applied on the skin for 24 hours (±4 hours of allowance) daily for a period of 12 months.
干预措施: Placebo (Biological)
结局指标
主要结局
Difference in Percentages of Treatment Responders at Month 12; Analyzed in the Overall Population
时间窗: At Month 12
The Double-Blind Placebo-Controlled Food Challenges (DBPCFCs) to determine Eliciting Dose (ED) were performed at screening and Month 12, with each challenge occurring over 2 days. The participant was gradually fed increasing amounts of standardized blinded oral formulas containing either peanut protein (during 1 of the 2 days of the challenge), or without any peanut protein (during the other day of the challenge). A participant was defined as a treatment responder if: * ED was ≥300 mg peanut protein at Month 12 DBPCFC (for screening ED subgroup 1), or * ED was ≥1,000 mg peanut protein at Month 12 DBPCFC (for screening ED subgroup 2). Participants with missing treatment response at Month 12 were imputed as non-responders. The percentage of treatment responders at Month 12 is presented. Analysis of the difference in response rates between treatment groups is presented in the subsequent statistical analysis table. Analysis was performed in the overall population.
次要结局
- Difference in Percentages of Treatment Responders at Month 12; Analyzed in Each Screening Eliciting Dose (ED) Subgroup(At Month 12)
- Cumulative Reactive Dose (CRD) of Peanut Protein at Baseline and Month 12(Baseline and Month 12)