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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT05926765 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Grade 2 and 3 Radiation-Induced Late Xerostomia
Disease Ontology Term DOID:10854
Compound Name AAV-hAQP1
Sponsor MeiraGTx, LLC
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 120
Results Posted Not Available

Therapy Information


Target Gene/Variant AQP1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Parotid
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 0.4E12 vg/gland
Dose 2 1.2E12 vg/gland
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase2
Submit Date 2023-06-22
Completion Date 2025-07
Last Update 2024-10-08

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 22
Locations Canada,United States,United Kingdom

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates FDA in support of Phase II study as pivotal for BLA submission

Resources/Links