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

Radiation-induced Xerostomia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05060341
• Other study ID #: MGT-AQP1-102
• Status: Active, not recruiting
• Start date: November 12, 2020
• Est. completion date: March 2027

Study information

• Verified date: April 2024
• Source: MeiraGTx UK II Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 22
• Est. completion date: March 2027
• Est. primary completion date: March 2027
• Gender: All
• Age group: 18 Years to 81 Years

Eligibility

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

Related Conditions & MeSH terms

• Radiation-induced Xerostomia
• Xerostomia

Locations

Country	Name	City	State
Canada	Health Sciences North - Northeast Cancer Center	Sudbury	Ontario
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Atrium Health	Charlotte	North Carolina
United States	Leland Stanford Junior University	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
MeiraGTx, LLC

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	• Incidence of AEs, treatment-emergent adverse events (TEAEs), and SAEs	Safety	4 years
Secondary	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
Secondary	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
Secondary	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
Secondary	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