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AAV2hAQP1: 1 x 10^11 vg/gland

Phase 1

Radiation-Induced Parotid Gland Hypofunction | Small molecule | Oncology |MeiraGTx Holdings plc|Last Updated: Apr 24, 2023

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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04043104A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and XerostomiaPHASE1 COMPLETED 24Jun 30, 2019Mar 28, 2023Apr 24, 20236 United States, Canada
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Study Endpoints
Primary Endpoints
The primary outcome is safety of AAV2hAQP1 administered to the parotid gland of adult subjects with radiation-induced xerostomia
one day to one year

Safety will be assessed by number of adverse events occurring with treatment

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1 x 10^11 vg/gland (single gland)EXPERIMENTAL -
3 x 10^10 vg/gland (both glands)EXPERIMENTAL -
3 x 10^11 vg/gland (single gland)EXPERIMENTAL -
1 x 10^11 vg/gland (both glands)EXPERIMENTAL -
1 x 10^12 vg/gland (single gland)EXPERIMENTAL -
3 x 10^11 vg/gland (both glands)EXPERIMENTAL -
3 x 10^12 vg/gland (single gland)EXPERIMENTAL -
1 x 10^12 vg/gland (both glands)EXPERIMENTAL -
Interventions
NameTypeDescription
AAV2hAQP1: 1 x 10^11 vg/gland (single gland)DRUGIntra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10\^11 vg/gland
AAV2hAQP1: 3 x 10^10 vg/gland (both glands)DRUGIntra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10\^10 vg/gland
AAV2hAQP1: 3 x 10^11 vg/gland (single gland)DRUGIntra-parotid administration of AAV2hAQP1 of via Stensen's duct to a single parotid gland at a dose level of 3 x 10\^11 vg/gland
AAV2hAQP1: 1 x 10^11 vg/gland (both glands)DRUGintra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10\^11 vg/gland
AAV2hAQP1: 1 x 10^12 vg/gland (single gland)DRUGIntra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10\^12 vg/gland
AAV2hAQP1: 3 x 10^11 vg/gland (both glands)DRUGIntra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10\^11 vg/gland
AAV2hAQP1: 3 x 10^12 vg/gland (single gland)DRUGIntra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 3 x 10\^12 vg/gland
AAV2hAQP1: 1 x 10^12 vg/gland (both glands)DRUGIntra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10\^12 vg/gland
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: 1. Male or female subjects ≥18 years of age. 2. History of radiation therapy for head and neck cancer. 3. Abnormal parotid gland function as judged by both absence of unstimulated parotid salivary flow and a stimulated parotid salivary flow in the targeted parotid gland \>0 and ...

Countries:United StatesCanada
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