A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of AAV2-hAQP1 Gene Therapy in Participants with Radiation-Induced Late Xerostomia
IRAS ID
1007453
Contact name
Maegan Little
Contact email
Sponsor organisation
MeiraGTx, LLC
Clinicaltrials.gov Identifier
Research summary
This main purpose of the study is to test the efficacy and safety of a new gene therapy, AAV2-hAQP1, for the treatment of late xerostomia caused by radiation therapy for head and neck cancer. People who receive radiation therapy to treat head and neck cancer can lose the ability to make enough saliva because of loss or damage to fluid producing cells.
This gene therapy will introduce new genetic material, so that the body makes more AQP1 protein. AQP1 is a water channel protein that allows water to move in and out of cells in the body. It may then be possible for the patients to make more saliva. The study will be performed in males and females aged over 18 years. It is planned that 120 adults will be enrolled and the study will be performed in the US, Canada and the UK, with 38 sites involved in recruiting patients. The planned study duration is 1 year. Over the study period, there will be 13 outpatient hospital visits.
Potential participants will complete a number of screening assessments over three visits to ensure they are eligible to take part in the study.
Participants who are eligible will receive either one of two concentrations of the active study therapy or a placebo, delivered to both parotid glands by an injection. Following treatment administration, participants will return to site ten times where they’ll have their vital signs checked, urine, saliva and blood samples will be taken, oral examination will be performed, and the moisture content of their mouths will be assessed using a special paper device. Participants are also required to complete several questionnaires and will be asked how they are feeling and if there have been any changes in their medications.REC name
North East - York Research Ethics Committee
REC reference
23/NE/0211
Date of REC Opinion
16 Jan 2024
REC opinion
Further Information Favourable Opinion