A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia
AQUAx2
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
1 other identifier
interventional
276
3 countries
33
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Jun 2023
Typical duration for phase_2
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 13, 2023
CompletedFirst Submitted
Initial submission to the registry
June 22, 2023
CompletedFirst Posted
Study publicly available on registry
July 3, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2026
February 2, 2026
January 1, 2026
3.5 years
June 22, 2023
January 30, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score
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 Outcomes (2)
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
Study Arms (6)
Cohort 1: AAV2-hAQP1 Group 1
EXPERIMENTALEligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Cohort 1: AAV2-hAQP1 Group 2
EXPERIMENTALEligible participants will receive up to 3 mL of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Cohort 1: Placebo group
PLACEBO COMPARATOREligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
Cohort 2: AAV2-hAQP1 Group 3
EXPERIMENTALEligible participants will receive up to 3 mL of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Cohort 2: AAV2-hAQP1 Group 4
EXPERIMENTALEligible participants will receive up to 3 mL of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Cohort 2 Placebo group
PLACEBO COMPARATOREligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
Interventions
Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Administration of diluent via Stensen's duct to each parotid gland
Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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%)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- MeiraGTx, LLClead
Study Sites (33)
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234, United States
City of Hope
Duarte, California, 91010, United States
Miami Cancer Institute at Baptist Health South Florida
Miami, Florida, 33176, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21231, United States
Tufts University School of Dental Medicine
Boston, Massachusetts, 02111, United States
Brigham and Women's Hospital
Boston, Massachusetts, 02120, United States
Henry Ford Health
Detroit, Michigan, 48202, United States
University of Missouri
Columbia, Missouri, 65212, United States
Washington University - St. Louis
St Louis, Missouri, 63110, United States
Erie County Medical Center
Buffalo, New York, 14215, United States
UNC-Chapel Hill
Chapel Hill, North Carolina, 27514, United States
Atrium Health
Charlotte, North Carolina, 28203, United States
Penn State
Hershey, Pennsylvania, 17033, United States
Alleghany General Hospital
Pittsburgh, Pennsylvania, 15212, United States
Johnson City Medical Center
Johnson City, Tennessee, 37604, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
Houston Methodist
Houston, Texas, 77030, United States
Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North
Greater Sudbury, Ontario, P3E 5J1, Canada
Hopital Fleurimont, CIUSSS de l'Estrie-CHUS
Québec, J1H 5HE, Canada
Princess Margaret Cancer Centre
Toronto, M5G 2C4, Canada
CIUSSS-MCQ (Trois-Rivières, QC)
Trois-Rivières, G8Z 3R9, Canada
Addenbrooke's Hospital
Cambridge, United Kingdom
Cardiff and Vale NHS Trust - Head & Neck Services
Cardiff, CF14 4XW, United Kingdom
Ninewells Hospital & Medical School
Dundee, DD1 9SY, United Kingdom
Glasgow Royal Infirmary
Glasgow, LS2 9LU, United Kingdom
Leeds Dental Institute
Leeds, LS2 9LU, United Kingdom
Western General
London, EH4 2XU, United Kingdom
Guys Hospital
London, SE1 9RT, United Kingdom
The Royal Marsden
London, SW3 6JJ, United Kingdom
University College London Hospitals NHS Foundation Trust
London, WC1E 6DG, United Kingdom
Nottingham University Hospitals NHS Trust
Nottingham, NG7 2UH, United Kingdom
York Hospital
York, YO31 8HE, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 22, 2023
First Posted
July 3, 2023
Study Start
June 13, 2023
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
December 1, 2026
Last Updated
February 2, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share