Long-Term Follow-Up Study of AAV2hAQP1 for Radiation Induced Xerostomia

Long-Term Follow-Up Study of Participants Enrolled in the MGT016 Phase 1, Open-Label, Dose-Escalation Study of AAV2hAQP1 for Radiation-Induced Parotid Gland Hypofunction and Xerostomia

This study is a long-term follow-up study for patients who have been administered AAV2hAQP1 in the Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity in Subjects with Radiation Induced Parotid Gland Hypofunction and Xerostomia

Study Overview

• Status: Active, not recruiting
• Conditions: Radiation-induced Xerostomia

Detailed Description

This clinical trial is designed to assess the long-term safety and activity of intra-parotid administration of AAV2hAQP1 in subjects with radiation-induced parotid salivary hypofunction and xerostomia.

After completion of their Month 12 Follow-Up visit in Study MGT016, subjects will be invited to enroll in this 4-year follow-up study during which they will be assessed for up to 60 months following study drug administration. This follow-up trial is a non-interventional study designed to collect data on longer-term safety and activity at 18, 24, 36, 48, and 60 months following study drug administration.

Over the course of the study, subjects will undergo physical and oral examinations; vital sign measurements; weight measurements; AE assessments; concurrent medication assessments; sample blood collection for hematology and chemistry; urinalysis; ear, nose, and throat (ENT) screening for malignancy; imaging assessments (if indicated); salivary flow assessments; and questionnaire assessments.

• Study Type: Observational
• Enrollment (Actual): 22

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Sudbury, Ontario, Canada
      • Health Sciences North - Northeast Cancer Center
• United States
  • California
    • Stanford, California, United States, 94305
      • Leland Stanford Junior University
  • Massachusetts
    • Boston, Massachusetts, United States, 02184
      • Brigham and Women's Hospital
  • North Carolina
    • Charlotte, North Carolina, United States, 28209
      • Atrium Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 81 years (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Subjects with radiation induced parotid gland hypofunction and xerostomia who have particpated in the MGT016 AAV2hAQP1 study.

Description

Inclusion Criteria:

1. Are willing and able to provide informed consent
2. Received AAV2hAQP1 administered via Stensen's duct to a single or both parotid glands in the prior open-label, Phase 1, dose-escalation study (Study MGT016)
3. Are willing and able to adhere to the protocol and long-term follow-up

Exclusion Criteria:

1. Subjects unwilling or unable to meet with the requirements of the study will be excluded

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
• Incidence of AEs, treatment-emergent adverse events (TEAEs), and SAEs	Safety	4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in saliva flow compared to baseline.	Changes in unstimulated and stimulated salivary output (mL/minute) of the treated parotid gland and/or whole saliva output as compared to baseline.	4 years
Changes in the McMaster Global Rate of Change relative to Baseline	This first part of this questionnaire asks the patient to evaluate their Dry Mouth relative to before treatment as better, worse, or the same. If better or worse, the patient is asked to rate the magnitude of change, on a scale of 1-7, with a higher score being a better outcome. This first part of this questionnaire asks the patient to evaluate how limited they are in daily activities due to dry mouth, relative to before treatment as better, worse, or the same. If better or worse, the patient is asked to rate the magnitude of change, on a scale of 1-7, with a higher score being a better outcome.	4 years
Changes in the Xerostomia Questionnaire relative to Baseline	The XQ is a series of 8 questions designed to assess the degree of xerostomia symptoms. Each question is a scale of 1-10, with high values indicating worse xerostomia.	4 years
Changes in the MD Anderson Symptom Inventory - Head and Neck	The MDASI-HN is a questionnaire consisting of 28 questions designed to assess the degree of xerostomia symptoms. Each question is a scale of 1-10, with high values indicating worse xerostomia.	4 years

Collaborators and Investigators

• Sponsor: MeiraGTx, LLC

Study record dates

Study Major Dates

• Study Start (Actual): November 12, 2020
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: March 9, 2021
• First Submitted That Met QC Criteria: September 24, 2021
• First Posted (Actual): September 29, 2021

Study Record Updates

• Last Update Posted (Actual): May 22, 2025
• Last Update Submitted That Met QC Criteria: May 19, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mouth Diseases; Stomatognathic Diseases; Salivary Gland Diseases; Xerostomia
• Other Study ID Numbers: MGT-AQP1-102

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: No
• Studies a U.S. FDA-regulated device product: No