A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia (AQUAx2)
July 23, 2026 updated by: MeiraGTx, LLC
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
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.
Study Overview
Status
Active, not recruiting
Study Type
Interventional
Enrollment (Actual)
290
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Québec, Canada, J1H 5HE
- Hopital Fleurimont, CIUSSS de l'Estrie-CHUS
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Toronto, Canada, M5G 2C4
- Princess Margaret Cancer Centre
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Trois-Rivières, Canada, G8Z 3R9
- CIUSSS-MCQ (Trois-Rivières, QC)
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Ontario
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Greater Sudbury, Ontario, Canada, P3E 5J1
- Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North
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Cambridge, United Kingdom
- Addenbrooke'S Hospital
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Cardiff, United Kingdom, CF14 4XW
- Cardiff and Vale NHS Trust - Head & Neck Services
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Dundee, United Kingdom, DD1 9SY
- Ninewells Hospital & Medical School
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Glasgow, United Kingdom, LS2 9LU
- Glasgow Royal Infirmary
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Leeds, United Kingdom, LS2 9LU
- Leeds Dental Institute
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London, United Kingdom, SE1 9RT
- Guys Hospital
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London, United Kingdom, SW3 6JJ
- The Royal Marsden
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London, United Kingdom, EH4 2XU
- Western General
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London, United Kingdom, WC1E 6DG
- University College London Hospitals NHS Foundation Trust
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Nottingham, United Kingdom, NG7 2UH
- Nottingham University Hospitals NHS Trust
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York, United Kingdom, YO31 8HE
- York Hospital
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Arizona
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Gilbert, Arizona, United States, 85234
- Banner MD Anderson Cancer Center
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California
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Duarte, California, United States, 91010
- City of Hope
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Florida
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Miami, Florida, United States, 33176
- Miami Cancer Institute at Baptist Health South Florida
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa
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Maryland
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Baltimore, Maryland, United States, 21231
- Johns Hopkins Hospital
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Massachusetts
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Boston, Massachusetts, United States, 02120
- Brigham and Women's Hospital
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Boston, Massachusetts, United States, 02111
- Tufts University School of Dental Medicine
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Michigan
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Detroit, Michigan, United States, 48202
- Henry Ford Health
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Missouri
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Columbia, Missouri, United States, 65212
- University of Missouri
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St Louis, Missouri, United States, 63110
- Washington University - St. Louis
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New York
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Buffalo, New York, United States, 14215
- Erie County Medical Center
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North Carolina
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Chapel Hill, North Carolina, United States, 27514
- UNC-Chapel Hill
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Charlotte, North Carolina, United States, 28203
- Atrium Health
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Pennsylvania
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Hershey, Pennsylvania, United States, 17033
- Penn State
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Pittsburgh, Pennsylvania, United States, 15212
- Alleghany General Hospital
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Tennessee
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Johnson City, Tennessee, United States, 37604
- Johnson City Medical Center
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Texas
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Dallas, Texas, United States, 75390
- UT Southwestern Medical Center
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Houston, Texas, United States, 77030
- Houston Methodist
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Description
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%)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: 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
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Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
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Experimental: 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
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Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
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Placebo Comparator: Cohort 1: Placebo group
Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
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Administration of diluent via Stensen's duct to each parotid gland
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Experimental: 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
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Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland
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Experimental: 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
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Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland
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Placebo Comparator: Cohort 2 Placebo group
Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
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Administration of diluent via Stensen's duct to each parotid gland
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score
Time Frame: 12 months
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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.
|
12 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Change from Baseline to Month 12 in unstimulated whole saliva flow rate
Time Frame: 12 months
|
Change from Baseline to Month 12 in unstimulated whole saliva flow rate (mL/min)
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12 months
|
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The number of participants with treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Time Frame: from Baseline to Month 12
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from Baseline to Month 12
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 13, 2023
Primary Completion (Estimated)
June 1, 2027
Study Completion (Estimated)
June 1, 2027
Study Registration Dates
First Submitted
June 22, 2023
First Submitted That Met QC Criteria
June 22, 2023
First Posted (Actual)
July 3, 2023
Study Record Updates
Last Update Posted (Actual)
July 24, 2026
Last Update Submitted That Met QC Criteria
July 23, 2026
Last Verified
July 1, 2026
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MGT-AQP1-201
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.