Long-term Follow-up of Gene Therapy for Radiation-Induced Xerostomia

October 14, 2025 updated by: MeiraGTx, LLC

Long-term Follow-up of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia

This study will assess the long-term safety and efficacy of bilateral intra-parotid administration of AAV2-hAQP1 in adults with Grade 2 or Grade 3 radiation-induced late xerostomia.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Detailed Description

Individual participation in this study will last approximately 48 months for participants who received AAV2-hAQP1 in Study MGT-AQP1-201 and at least 60 months for participants who receive AAV2-hAQP1 in Study MGT-AQP1-202.

Study Type

Interventional

Enrollment (Estimated)

276

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Greater Sudbury, Ontario, Canada, P3E 5J1
        • Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North
  • United States
    • Arizona
      • Gilbert, Arizona, United States, 85234
        • Banner MD Anderson Cancer Center
    • Florida
      • Miami, Florida, United States, 33176
        • Miami Cancer Institute at Baptist Health South Florida
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Missouri
      • Columbia, Missouri, United States, 65212
        • University of Missouri
    • New York
      • Buffalo, New York, United States, 14215
        • Erie County Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28209
        • Atrium Health
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15212
        • Alleghany General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Received study drug in Study MGT-AQP1-201

Exclusion Criteria:

  • Withdrew consent to participate in Study MGT-AQP1-201.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Follow-up group
Participants who were randomized to AAV2-hAQP1 treatment in Study MGT-AQP1-201 will continue in this long-term follow-up schedule to complete an additional 48 months of follow-up after their end-of-study visit in Study MGT-AQP1-201. All study participants are to be followed for a period of 60 months after vector administration.
Experimental: Active treatment group
Participants who were randomized to placebo treatment in Study MGT-AQP1-201, will be offered to transition from the long-term follow-up schedule to an active treatment schedule at the time of unblinding. Study drug administration should be completed after unblinding and after confirmation of the participant's continued eligibility for treatment. Upon completion of the 12-month primary treatment period, participants will continue in the study according to a follow-up schedule, to complete a total of 60 months of follow-up after AAV2-hAQP1 administration.
Genetic: AAV2-hAQP1
Administration of a prespecified concentration of AAV2-hAQP1 via Stensen's duct to each parotid gland

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants with study drug-related adverse events and serious adverse events
Time Frame: From study start until Month 60 post-treatment
From study start until Month 60 post-treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

MeiraGTx, LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 2, 2024

Primary Completion (Estimated)

February 1, 2032

Study Completion (Estimated)

February 1, 2032

Study Registration Dates

First Submitted

August 5, 2024

First Submitted That Met QC Criteria

August 5, 2024

First Posted (Actual)

August 9, 2024

Study Record Updates

Last Update Posted (Estimated)

October 16, 2025

Last Update Submitted That Met QC Criteria

October 14, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MGT-AQP1-202

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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