A phase 2 randomized controlled trial using biologics to improve multi OIT outcomes (COMBINE): design, rationale, and methods

使用生物制剂改善多种口服免疫疗法疗效的 II 期随机对照试验 (COMBINE):设计、原理和方法

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Abstract

BACKGROUND: Food allergy remains a serious public health concern associated with significantly lowered quality of life and the risk of potentially life-threatening allergic reactions. While oral immunotherapy (OIT) has consistently demonstrated efficacy in the desensitization of multi-food allergic patients, many patients undergoing such treatment are burdened by dose-related side effects that can hinder their compliance and the overall efficacy of OIT. Recent efforts to improve upon OIT have begun to evaluate the concomitant use of biologics such as omalizumab and dupilumab with OIT for their ability to selectively inhibit pathways involved in the underlying pathology of food allergy. METHODS: Herein, we detail the clinical trial design, rationale, and methods for a Phase 2 randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of omalizumab and/or dupilumab therapy in combination with participant-specific multi-food (mOIT) in patients aged 4-55 years, with multi-food allergy that includes peanut. In this two-arm superiority trial, participants will be randomized (5:5:1) to (1) omalizumab/placebo-dupilumab with mOIT (n = 50), (2) omalizumab/dupilumab with mOIT (n = 50), or (3) a mechanistic-only arm of placebo-omalizumab/dupilumab with mOIT (n = 10). Double-blind placebo-controlled food challenges (DBPCFCs) will be used to assess desensitization to ≥1,043 mg cumulative protein at Week 32, after which all treatment is to be discontinued. A follow-up assessment of sustained unresponsiveness via DBPCFCs will be conducted at Week 44. CONCLUSION: This trial tests the hypothesis that adding dupilumab to omalizumab-facilitated mOIT will increase the likelihood of inducing sustained unresponsiveness and decrease mOIT-related adverse events.

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