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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

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

RMATORPHAN_DRUGBREAKTHROUGH_THERAPY

Clinical trial landscape

AAV2hAQP1: 1 x 10^11 vg/gland · 1 trial · 3 indications

Phase 1 1
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 XerostomiaRadiation-Induced Parotid Gland Hypofunction
COMPLETED24 Analytics
PHASE1COMPLETED
A 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 Xerostomia
Radiation-Induced Parotid Gland HypofunctionUnlock trial analytics

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 to 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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Frequently asked questions about AAV2hAQP1: 1 x 10^11 vg/gland

What is AAV2hAQP1: 1 x 10^11 vg/gland used for?

AAV2hAQP1: 1 x 10^11 vg/gland is being studied for radiation-induced parotid gland hypofunction and xerostomia in patients who received radiotherapy for head and neck cancer. It is an investigational gene therapy designed to restore salivary gland function in this oncology setting.

Who is developing AAV2hAQP1: 1 x 10^11 vg/gland?

AAV2hAQP1: 1 x 10^11 vg/gland is being developed by MeiraGTx Holdings plc, which trades on the Nasdaq under the ticker MGTX. The company is the sponsor of the clinical program for this gene therapy candidate.

What phase is AAV2hAQP1: 1 x 10^11 vg/gland in?

AAV2hAQP1: 1 x 10^11 vg/gland is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The Phase 1 trial has been completed.

What clinical trials is AAV2hAQP1: 1 x 10^11 vg/gland in?

AAV2hAQP1: 1 x 10^11 vg/gland has been evaluated in one clinical trial, NCT04043104, a Phase 1 open-label, dose escalation study. The trial enrolled 24 participants across the United States and Canada and is now completed.

What FDA designations has AAV2hAQP1: 1 x 10^11 vg/gland received?

AAV2hAQP1: 1 x 10^11 vg/gland has received three FDA designations: Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Breakthrough Therapy. These designations support the development of the therapy for radiation-induced parotid gland hypofunction.