Recent Updates
Recently added Catalysts

AAV2-hAQP1 Concentration 1

Phase 2

Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands | Gene therapy | ENT |MeiraGTx Holdings plc|Last Updated: Jul 24, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment290

FDA Designations

RMATORPHAN_DRUGBREAKTHROUGH_THERAPY

Clinical trial landscape

AAV2-hAQP1 Concentration 1 · 1 trial · 1 indication

Phase 2 1
NCT05926765A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late XerostomiaGrade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands
ACTIVE NOT_RECRUITING290 Analytics
PHASE2ACTIVE NOT_RECRUITING
A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia
Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid GlandsUnlock trial analytics

Study Endpoints

Primary Endpoints

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

Secondary Endpoints

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
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1: AAV2-hAQP1 Group 1EXPERIMENTALEligible 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 2EXPERIMENTALEligible participants will receive up to 3 mL of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Cohort 1: Placebo groupPLACEBO_COMPARATOREligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
Cohort 2: AAV2-hAQP1 Group 3EXPERIMENTALEligible 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 4EXPERIMENTALEligible participants will receive up to 3 mL of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Cohort 2 Placebo groupPLACEBO_COMPARATOREligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland

Interventions

NameTypeDescription
AAV2-hAQP1 Concentration 1GENETICAdministration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
AAV2-hAQP1 Concentration 2GENETICAdministration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
PlaceboOTHERAdministration of diluent via Stensen's duct to each parotid gland
AAV2-hAQP1 Concentration 3GENETICAdministration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland
AAV2-hAQP1 Concentration 4GENETICAdministration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites33

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 ...

Countries:United StatesCanadaUnited Kingdom
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

MEDIUMAug 1, 2026NCT05926765Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 1, 2026NCT05926765Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 1, 2026NCT05926765Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about AAV2-hAQP1 Concentration 1

What is AAV2-hAQP1 Concentration 1 used for?

AAV2-hAQP1 Concentration 1 is an investigational gene therapy being developed for Grade 2 and 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract, excluding the parotid glands. It is intended to treat dry mouth resulting from radiation therapy in these patients.

How does AAV2-hAQP1 Concentration 1 work?

AAV2-hAQP1 Concentration 1 is a gene therapy that uses an adeno-associated virus vector to deliver the human aquaporin-1 (hAQP1) gene. This gene encodes a water channel protein that is intended to restore salivary gland function and increase saliva production in patients with radiation-induced xerostomia.

Who is developing AAV2-hAQP1 Concentration 1?

AAV2-hAQP1 Concentration 1 is being developed by MeiraGTx Holdings plc, a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for radiation-induced late xerostomia.

What phase is AAV2-hAQP1 Concentration 1 in?

AAV2-hAQP1 Concentration 1 is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug has received FDA designations including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Breakthrough Therapy.

What clinical trials is AAV2-hAQP1 Concentration 1 in?

AAV2-hAQP1 Concentration 1 is being studied in a Phase 2 clinical trial with the identifier NCT05926765. This randomized, double-blind, placebo-controlled trial is active but not recruiting, with an estimated enrollment of 290 participants. The study is being conducted in the United States, Canada, and the United Kingdom.

Is AAV2-hAQP1 Concentration 1 the same as AAV2-hAQP1?

AAV2-hAQP1 Concentration 1 is a specific concentration or formulation of the gene therapy AAV2-hAQP1. The drug is referred to as AAV2-hAQP1 in clinical trial documentation, and the Concentration 1 designation indicates a particular dose or preparation being evaluated in the study.