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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 | Oncology |MeiraGTx Holdings plc|Last Updated: Feb 2, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment276
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05926765A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late XerostomiaPHASE2 RECRUITING 276Jun 13, 2023Dec 1, 2026Feb 2, 202633 United States, Canada +1
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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
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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
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Eligibility Criteria
Age Range18 Years — 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
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05926765primaryCompletionDate: changed
LOWMay 24, 2026NCT05926765studyFirstPostDate: changed