Beginning to Assess an Appropriate CONtrol for Oral Food Challenges in Alpha-Gal Syndrome (CoFAR-13) - BeACON4AG

Официальное название

Beginning to Assess an Appropriate CONtrol for Oral Food Challenges in Alpha-Gal Syndrome (CoFAR-13)

Обзор

This is a multisite, randomized, double-blind, controlled cross-over trial with detailed characterization of participants with varying clinical sub-phenotypes of Alpha-gal Syndrome (AGS) who are then evaluated by oral food challenges with alpha-gal Knock Out (KO) pork versus Wild Type (WT) pork. Each participant will serve as their own control and ingest both alpha-gal-KO and WT pork on different days, with a minimum of an 8-day washout period between oral food challenges. The primary objective is to determine whether the odds of positive challenges among participants with suspected alpha-gal syndrome are lower with alpha-gal Knock Out (KO) pork as compared to Wild Type (WT) pork during Double Blind Food Challenge (DBFC)

Вмешательства

• Диагностический тест DBFC with 150g of Alpha-gal Knock Out (KO) pork
  Each participant will serve as their own control and ingest both alpha-gal-KO and WT pork on different days, with a minimum of an 8-day washout period between oral food challenges
• Диагностический тест DBFC with 150g of Wild Type (WT) pork
  Each participant will serve as their own control and ingest both alpha-gal-KO and WT pork on different days, with a minimum of an 8-day washout period between oral food challenges

Первичные конечные точки

• Positive Double Blind Food Challenge (DBFC) result to 150 grams of either alpha-gal knockout (KO) pork or wild type (WT) pork. [Срок оценки: At Visit 1 (within 2 months of screening) or Visit 2 (between 8 and 30 days of Visit 1)]

Вторичные конечные точки (3)

• Alpha-gal sub-phenotype as determined by symptoms resulting from Double Blind Food Challenge (DBFC) to wild-type (WT) pork. [Срок оценки: At Visit 1 (within 2 months of screening) or Visit 2 (between 8 and 30 days of Visit 1)]
• Gastrointestinal Symptom Rating Scale - Irritable Bowel Syndrome (GSRS-IBS) scores in response to 150 grams of Knock Out (KO) or Wild Type (WT) pork at each challenge visit [Срок оценки: At 7 days following completion of Double Blind Food Challenge (DBFC) at Visit 1 (within 2 months of screening) or Visit 2 (between 8 and 30 days of Visit 1)]
• Urticaria Activity Score (UAS7) in response to 150 grams of Knock Out (KO) or Wild Type (WT) pork at each challenge visit. [Срок оценки: At 7 days following completion of Double Blind Food Challenge (DBFC) at Visit 1 (within 2 months of screening) or Visit 2 (between 8 and 30 days of Visit 1)]

Критерии участия

Критерии включения

• Able to understand and provide informed consent and/or have a parent or guardian able to understand and provide informed consent. Participants 12-17 years old must be able to provide assent
• Participants who are willing to complete study procedures and follow-up
• Have clinical history consistent with Alpha-gal Syndrome (AGS)
• Have alpha-gal-specific Immunoglobin E (IgE) >= 0.1KU/L at screening
• Report reaction consistent with AGS within the previous 24 months (prior to enrollment)
• If with reproductive potential, agree to be abstinent or use an US Food and Drug Administration (FDA) approved method of birth control for the duration of the study
• Willing to be trained on the proper use of a device for the self-administration of epinephrine (i.e., autoinjector or intra-nasal spray) and willing to have two doses available for the duration of the study

Критерии исключения

• Unwilling to consume pork (native/wild type or genetically modified to no longer contain alpha-gal) during Double Blind Food Challenge (DBFC)
• Have underlying health conditions suggesting that participants should not undergo an oral food challenge, including conditions that may be exacerbated by oral food challenge or treatment with epinephrine (e.g., history of ischemic heart disease, uncontrolled asthma, or cardiac arrhythmia)
• Likely to move away from the study site prior to completion of follow up or do not have regular access to a phone
• Have a history of severe anaphylaxis in response to pork or alpha-gal ingestion, defined as neurologic compromise or requiring intubation or cardiovascular compromise defined as myocardial infarction or other clinically significant cardiovascular events
• Unwilling or unable to stop use of medications that may interfere with oral food challenge interpretation, reaction, or treatment for 5 half-lives prior to the challenge (e.g., antihistamines, beta blockers, Non-Steroidal Anti-Inflammatory Drugs (NSAIDs)
• Have used systemic immunomodulators (including biologics and monoclonal antibody therapy) within 6 months before study entry
• Have underlying health conditions that may increase the chance of false positive challenges (e.g., inflammatory bowel disease, chronic urticaria, systemic mastocytosis, and mast cell activating syndrome)
• Have used systemic steroids within 4 weeks of study entry
• Have used any investigational agent within 6 months of study entry
• Are on build-up of immunotherapy such as Antigen-based Immunotherapy (AIT), Oral Immunotherapy (OIT), Sublingual Immunotherapy (SLIT), or Epicutaneous Immunotherapy (EPIT)
• Have reacted to mammalian products with onset of symptoms within 30 minutes of oral ingestion
• Have current, diagnosed, mental illness or current, diagnosed or self-reported drug or alcohol abuse that, in the opinion of the investigator, would interfere with the participant's ability to comply with study requirements
• Are pregnant or plan to become pregnant during the course of the study
• Have past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements, and/or may impact the quality or interpretation of the data obtained from the study

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Перекрёстный дизайн

Маскирование

Двойное слепое

Основная цель

Диагностика

Центры проведения

США · 4 центра

• Arkansas Children's Hospital Research Institute: Department of Pediatrics, Allergy & Immun — Little Rock
• North Carolina Children's Hospital: Department of Pediatrics, Division of Allergy, Immunol — Chapel Hill
• Vanderbilt University Medical Center: Division of Pediatric Allergy, Immunology, and Pulmo — Nashville
• University of Virginia Health System: Division of Asthma & Immunology — Charlottesville

Идентификаторы

NCT: NCT07611435 · DAIT CoFAR-13

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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