临床试验/NCT05926765

NCT05926765进行中（未招募）2 期

A Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia

MeiraGTx, LLC56 个研究点分布在 3 个国家目标入组 290 人开始时间: 2023年6月13日最近更新: 2026年7月24日

适应症

干预措施

试验速览

阶段: 2 期
状态: 进行中（未招募）
发起方: MeiraGTx, LLC
入组人数: 290
试验地点: 56
主要终点: Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验相关资讯

研究概览

简要总结

This study will assess the efficacy and safety of bilateral intra-parotid administration of AAV2-hAQP1 in adults with Grade 2 or Grade 3 radiation-induced late xerostomia.

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, Investigator, Outcomes Assessor)

入排标准

年龄范围: 18 Years 至 —（Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Completed beam radiation therapy for head and neck cancer at least 3 years prior to the first screening visit
•No history of recurrent head and neck cancer, parotid gland cancer, or a second primary cancer, except for treated basal cell or squamous cell carcinoma of the skin or in situ cervical carcinoma
•An unstimulated whole saliva flow rate (mL/min) >0 (i.e., at least one drop of saliva in the collection tube)
•A stimulated whole saliva flow rate (mL/min) within a specified range after mechanical stimulation by chewing
•Average screening XQ Total Score at or above a specified threshold
•No evidence of head and neck cancer, defined as a negative otolaryngology exam and a negative computed tomography (CT) scan of the head, neck, and chest with contrast. If a participant has had a magnetic resonance imaging (MRI) study, CT scan, positron emission tomography (PET), or fluorodeoxyglucose-positron emission tomography (FDG-PET) scan of the head, neck, and chest within 6 months of signing the informed consent form (and at least 3 years after the completion of radiotherapy), then that scan may be used for eligibility determination and a CT scan at screening will not be required. If the CT of the neck captures images from the forehead down to the neck, no CT of the head is required.
•Either received treatment with one or more prescription sialagogues and elected to discontinue therapy or, in consultation with their physician, elected not to initiate such treatment
•Participants taking a prescription sialagogue (specifically, pilocarpine or cevimeline) must stop that medication at least 2 weeks prior to Screening and be willing to refrain from taking such medications for the duration of the study
•Participants who require medication for an underlying medical condition that is known to affect salivary output must be on stable doses of such medications for at least one month prior to the first screening visit

排除标准

•History of recurrent head and neck cancer, parotid gland cancer, or a second primary cancer, except for treated basal cell or squamous cell carcinoma of the skin or in situ cervical carcinoma
•History of systemic autoimmune disease affecting the salivary glands (e.g., Sjogren's disease)
•Currently using systemic immunosuppressive medication(s) (e.g., corticosteroids or biologics) or treated with one within 4 weeks of the first screening visit. Note: Topical, inhaled, or intranasal corticosteroids are permitted.
•Active viral infection with Epstein-Barr virus (EBV), defined as a positive anti-VCA IgM. In the event a potential participant has a positive anti-VCA IgM, they may be rescreened 2-4 months later at which time a positive Epstein-Barr Virus Nuclear Antigen (EBNA) will be considered as evidence of resolved EBV infection.
•Evidence of active Hepatitis C virus (HCV) infection
•Evidence of human immunodeficiency virus (HIV) infection
•Diagnosis of myasthenia gravis
•Personal or family history of acute or chronic angle-closure glaucoma (ACG), or at increased risk of developing ACG, or had prophylactic treatment to reduce the risk of developing ACG
•Known allergy or hypersensitivity to glycopyrrolate
•Current smokers or history of smoking within the preceding 3 years (includes vaping with tobacco additives)
•Current alcohol misuse or a history of the same within the preceding 3 years, as defined by local guidance. In North America, an average intake for men of more than 14 drinks per week, and for women more than 7 drinks per week, consistent with the US National Institute of Alcohol Abuse and Alcoholism. In the UK, an average intake of more than 14 units per week for both men and women, consistent with the UK Chief Medical Officers' Low Risk Drinking Guidelines.
•Poorly controlled diabetes (hemoglobin A1c >7%)

研究组 & 干预措施

Cohort 1: AAV2-hAQP1 Group 1

Experimental

Eligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland

干预措施: AAV2-hAQP1 Concentration 1 (Genetic)

Cohort 1: AAV2-hAQP1 Group 2

Experimental

Eligible participants will receive up to 3 mL of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland

干预措施: AAV2-hAQP1 Concentration 2 (Genetic)

Cohort 1: Placebo group

Placebo Comparator

Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland

干预措施: Placebo (Other)

Cohort 2: AAV2-hAQP1 Group 3

Experimental

Eligible participants will receive up to 3 mL of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland

干预措施: AAV2-hAQP1 Concentration 3 (Genetic)

Cohort 2: AAV2-hAQP1 Group 4

Experimental

Eligible participants will receive up to 3 mL of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland

干预措施: AAV2-hAQP1 Concentration 4 (Genetic)

Cohort 2 Placebo group

Placebo Comparator

Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland

干预措施: Placebo (Other)

结局指标

主要结局

Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score

时间窗: 12 months

The XQ consists of 8 symptom-specific questions the participant rates from 0 (not present) to 10 (worst possible). The XQ Total Score is the sum of all individual item ratings and ranges from 0 to 80.

Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score

时间窗: 12 months

次要结局

Change from Baseline to Month 12 in unstimulated whole saliva flow rate(12 months)
Change from Baseline to Month 12 in unstimulated whole saliva flow rate(12 months)
The number of participants with treatment-emergent adverse events (AEs) and serious adverse events (SAEs)(from Baseline to Month 12)

研究者

发起方

MeiraGTx, LLC

申办方类型

Industry

责任方

Sponsor

研究点 (56)

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