Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid GlandsPhase 2AQUAx2NCT05926765

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

A Phase 2 study testing AAV2-hAQP1 Concentration 1, AAV2-hAQP1 Concentration 2 and AAV2-hAQP1 Concentration 3 in people with Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands, run by MeiraGTx, LLC at 33 sites in 3 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands
What is being tested?: AAV2-hAQP1 Concentration 1, AAV2-hAQP1 Concentration 2 and AAV2-hAQP1 Concentration 3
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 290 participants
Where: 33 sitesin 3 countries
Study dates: 13 Jun 2023to Jun 2027
Phase: Phase 2
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

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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

Who cannot join

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

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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%)

Trial locations and contacts

33 sites in 3 countries.

Country

City, postcode or hospital

33 sites in 3 countries

United States18 sites

Johns Hopkins Hospital
Baltimore, Maryland, 21231
Tufts University School of Dental Medicine
Boston, Massachusetts, 02111
Brigham and Women's Hospital
Boston, Massachusetts, 02120
Erie County Medical Center
Buffalo, New York, 14215
UNC-Chapel Hill
Chapel Hill, North Carolina, 27514
Atrium Health
Charlotte, North Carolina, 28203
University of Missouri
Columbia, Missouri, 65212
UT Southwestern Medical Center
Dallas, Texas, 75390
Henry Ford Health
Detroit, Michigan, 48202
City of Hope
Duarte, California, 91010
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234
Penn State
Hershey, Pennsylvania, 17033
Houston Methodist
Houston, Texas, 77030
University of Iowa
Iowa City, Iowa, 52242
Johnson City Medical Center
Johnson City, Tennessee, 37604
Miami Cancer Institute at Baptist Health South Florida
Miami, Florida, 33176
Alleghany General Hospital
Pittsburgh, Pennsylvania, 15212
Washington University - St. Louis
St Louis, Missouri, 63110

Other trials in United States

United Kingdom11 sites

Addenbrooke's Hospital
Cambridge
Cardiff and Vale NHS Trust - Head & Neck Services
Cardiff, CF14 4XW
Ninewells Hospital & Medical School
Dundee, DD1 9SY
Glasgow Royal Infirmary
Glasgow, LS2 9LU
Leeds Dental Institute
Leeds, LS2 9LU
Western General
London, EH4 2XU
Guys Hospital
London, SE1 9RT
The Royal Marsden
London, SW3 6JJ
University College London Hospitals NHS Foundation Trust
London, WC1E 6DG
Nottingham University Hospitals NHS Trust
Nottingham, NG7 2UH
York Hospital
York, YO31 8HE

Other trials in United Kingdom

Canada4 sites

Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North
Greater Sudbury, Ontario, P3E 5J1
Hopital Fleurimont, CIUSSS de l'Estrie-CHUS
Québec, J1H 5HE
Princess Margaret Cancer Centre
Toronto, M5G 2C4
CIUSSS-MCQ (Trois-Rivières, QC)
Trois-Rivières, G8Z 3R9

Other trials in Canada

About this study

Official title: 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.

Conditions: Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands
Being tested: AAV2-hAQP1 Concentration 1, AAV2-hAQP1 Concentration 2, Placebo, AAV2-hAQP1 Concentration 3, AAV2-hAQP1 Concentration 4
Medical subject headings: Lymphoma, Follicular

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AAV2-hAQP1 Concentration 1Genetic
Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
AAV2-hAQP1 Concentration 2Genetic
Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
PlaceboOther
Administration of diluent via Stensen's duct to each parotid gland
AAV2-hAQP1 Concentration 3Genetic
Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland
AAV2-hAQP1 Concentration 4Genetic
Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Study groups in NCT05926765
Study group	Role	What they receive
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 Receives: Genetic: AAV2-hAQP1 Concentration 1
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 Receives: Genetic: AAV2-hAQP1 Concentration 2
Cohort 1: Placebo group	Placebo comparator	Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland Receives: Other: Placebo
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 Receives: Genetic: AAV2-hAQP1 Concentration 3
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 Receives: Genetic: AAV2-hAQP1 Concentration 4
Cohort 2 Placebo group	Placebo comparator	Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 12 months

Secondary outcomes (2)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

13 Jun 2023Jun 2027

First posted3 Jul 2023
Study start13 Jun 2023
Primary completionJun 2027 (estimated)
Study completionJun 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid GlandsStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Investigator, Outcomes assessor
Enrolment: 290 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05926765
Sponsor's protocol ID: MGT-AQP1-201
Acronym: AQUAx2
Status last verified: Jul 2026
First submitted: 22 Jun 2023
Last updated: 24 Jul 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Study at a glance

From the registry record, last updated by the sponsor on 24 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT05926765
Acronym / sponsor ID: AQUAx2 / MGT-AQP1-201
Conditions: Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands
Phase: Phase 2
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 290 participants (actual)
Lead sponsor: MeiraGTx, LLCIndustry (pharma, biotech, devices)
Study start: 13 Jun 2023 (actual)
Primary completion: Jun 2027 (estimated)
Study completion: Jun 2027 (estimated)
First posted: 3 Jul 2023 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, investigator, outcomes assessor)
Number of arms: 6

Arm	Type	Receives
Cohort 1: AAV2-hAQP1 Group 1	Experimental	Genetic: AAV2-hAQP1 Concentration 1Eligible 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	Experimental	Genetic: AAV2-hAQP1 Concentration 2Eligible 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 comparator	Other: PlaceboEligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
Cohort 2: AAV2-hAQP1 Group 3	Experimental	Genetic: AAV2-hAQP1 Concentration 3Eligible 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	Experimental	Genetic: AAV2-hAQP1 Concentration 4Eligible 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 comparator	Other: PlaceboEligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (9 listed)

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

Key exclusion criteria (12 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
AAV2-hAQP1 Concentration 1Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland	Genetic	Cohort 1: AAV2-hAQP1 Group 1
AAV2-hAQP1 Concentration 2Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland	Genetic	Cohort 1: AAV2-hAQP1 Group 2
PlaceboAdministration of diluent via Stensen's duct to each parotid gland	Other	Cohort 1: Placebo group, Cohort 2 Placebo group
AAV2-hAQP1 Concentration 3Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland	Genetic	Cohort 2: AAV2-hAQP1 Group 3
AAV2-hAQP1 Concentration 4Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland	Genetic	Cohort 2: AAV2-hAQP1 Group 4

Primary outcome

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

2 secondary outcomes

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

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2026.
Individual participant dataSharing planned: No

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

33 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 24 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 33 sites in United States, United Kingdom, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05926765.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2026.