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

NCT06544798 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: MGT-AQP1-202
• Study Type: interventional
• Target: 276
• Locations: 2 countries
• Sites: 8

Brief Summary

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.

Conditions

Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands

Outcome Measures

Primary Outcomes (1)

• Number of participants with study drug-related adverse events and serious adverse events

  From study start until Month 60 post-treatment

Study Arms (2)

Follow-up group

NO INTERVENTION

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.

Active treatment group

EXPERIMENTAL

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

Interventions

AAV2-hAQP1 GENETIC

Administration of a prespecified concentration of AAV2-hAQP1 via Stensen's duct to each parotid gland

Active treatment group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Received study drug in Study MGT-AQP1-201

You may not qualify if:

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

Sponsors & Collaborators

• MeiraGTx, LLC: lead

Study Sites (8)

Banner MD Anderson Cancer Center

Gilbert, Arizona, 85234, United States

Location

Miami Cancer Institute at Baptist Health South Florida

Miami, Florida, 33176, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Missouri

Columbia, Missouri, 65212, United States

Location

Erie County Medical Center

Buffalo, New York, 14215, United States

Location

Atrium Health

Charlotte, North Carolina, 28209, United States

Location

Alleghany General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North

Greater Sudbury, Ontario, P3E 5J1, Canada

Location

MeSH Terms

Conditions

Lymphoma, Follicular

Condition Hierarchy (Ancestors)

Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be assigned to the follow-up group or active treatment group based on the group to which they were randomized in Study MGT-AQP1-201.

Study Record Dates

• First Submitted: August 5, 2024
• First Posted: August 9, 2024
• Study Start: August 2, 2024
• Primary Completion (Estimated): February 1, 2032
• Study Completion (Estimated): February 1, 2032
• Last Updated: October 16, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (7); CA (1)