A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia

NCT ID: NCT05926765

Last Updated: 2025-12-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

276 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-13

Study Completion Date

2026-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Cohort 1: AAV2-hAQP1 Group 1

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

Group Type EXPERIMENTAL

AAV2-hAQP1 Concentration 1

Intervention Type GENETIC

Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Cohort 1: AAV2-hAQP1 Group 2

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

Group Type EXPERIMENTAL

AAV2-hAQP1 Concentration 2

Intervention Type GENETIC

Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Cohort 1: Placebo group

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Administration of diluent via Stensen's duct to each parotid gland

Cohort 2: AAV2-hAQP1 Group 3

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

Group Type EXPERIMENTAL

AAV2-hAQP1 Concentration 3

Intervention Type GENETIC

Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Cohort 2: AAV2-hAQP1 Group 4

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

Group Type EXPERIMENTAL

AAV2-hAQP1 Concentration 4

Intervention Type GENETIC

Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Cohort 2 Placebo group

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Administration of diluent via Stensen's duct to each parotid gland

Interventions

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AAV2-hAQP1 Concentration 1

Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Intervention Type GENETIC

AAV2-hAQP1 Concentration 2

Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Intervention Type GENETIC

Placebo

Administration of diluent via Stensen's duct to each parotid gland

Intervention Type OTHER

AAV2-hAQP1 Concentration 3

Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Intervention Type GENETIC

AAV2-hAQP1 Concentration 4

Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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%)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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MeiraGTx, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Banner MD Anderson Cancer Center

Gilbert, Arizona, United States

Site Status RECRUITING

City of Hope

Duarte, California, United States

Site Status RECRUITING

Miami Cancer Institute at Baptist Health South Florida

Miami, Florida, United States

Site Status RECRUITING

University of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Henry Ford Health

Detroit, Michigan, United States

Site Status RECRUITING

University of Missouri

Columbia, Missouri, United States

Site Status RECRUITING

Erie County Medical Center

Buffalo, New York, United States

Site Status RECRUITING

UNC-Chapel Hill

Chapel Hill, North Carolina, United States

Site Status RECRUITING

Atrium Health

Charlotte, North Carolina, United States

Site Status RECRUITING

Penn State

Hershey, Pennsylvania, United States

Site Status RECRUITING

Alleghany General Hospital

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Houston Methodist

Houston, Texas, United States

Site Status RECRUITING

Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North

Greater Sudbury, Ontario, Canada

Site Status RECRUITING

Hopital Fleurimont, CIUSSS de l'Estrie-CHUS

Québec, , Canada

Site Status RECRUITING

Princess Margaret Cancer Centre

Toronto, , Canada

Site Status RECRUITING

CIUSSS-MCQ (Trois-Rivières, QC)

Trois-Rivières, , Canada

Site Status RECRUITING

Addenbrooke's Hospital

Cambridge, , United Kingdom

Site Status RECRUITING

Ninewells Hospital & Medical School

Dundee, , United Kingdom

Site Status RECRUITING

Western General

London, , United Kingdom

Site Status RECRUITING

Guys Hospital

London, , United Kingdom

Site Status RECRUITING

Nottingham University Hospitals NHS Trust

Nottingham, , United Kingdom

Site Status RECRUITING

Countries

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United States Canada United Kingdom

Central Contacts

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MeiraGTx Clinical Project Manager

Role: CONTACT

646-860-7982

Facility Contacts

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Gabriela Rodriguez

Role: primary

Ellie Maghami

Role: primary

626-218-1133

Alessandro Villa

Role: primary

786-596-2000

McLean D Sunderland

Role: primary

Jessica Leitzel

Role: primary

Sharan Shah

Role: primary

Subramanian Karthikeyan

Role: primary

313-725-7859

Rebecca Schneider

Role: primary

Role: primary

Yumika Forney

Role: primary

919-962-8559

Jenene Noll

Role: primary

Carly Herbe

Role: primary

717-531-6822

Role: primary

Thomas Schlieve, DDS, MD

Role: primary

214-648-3034

Rejani Nair

Role: primary

346-238-4511

Role: primary

Cynthia Ladouceur

Role: primary

Michael Glogauer

Role: primary

Marie-Eve Caron

Role: primary

819-697-3333 ext. 63238

Amy Bates

Role: primary

Janet Douglas

Role: primary

01382 496685

Ashley Hay

Role: primary

0131 536 3162

Daisy Hubble

Role: primary

020 7188 2018

Jo Curtis

Role: primary

01159249924 ext. 80693

Kirsty Topham

Role: backup

Other Identifiers

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MGT-AQP1-201

Identifier Type: -

Identifier Source: org_study_id

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