A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia (AQUAX2)

April 18, 2024 updated by: MeiraGTx, LLC

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

120

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 Contact

Study Locations

  • Canada
    • Ontario
      • Sudbury, Ontario, Canada, P3E 5J1
        • Recruiting
        • Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North
        • Contact:
        • Principal Investigator:
          • Saunders, Dr
  • United States
    • Arizona
      • Gilbert, Arizona, United States, 85234
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa
        • Contact:
        • Principal Investigator:
          • Henry Hoffman, Dr.
    • Maryland
      • Baltimore, Maryland, United States, 21231
        • Recruiting
        • Sidney Kimmel Comprehensive Cancer Center
        • Principal Investigator:
          • Quon, Dr
        • Contact:
          • Harry Quon, MD
      • Baltimore, Maryland, United States, 21231
        • Recruiting
        • Johns Hopkins Hospital
        • Contact:
        • Principal Investigator:
          • Quon, Dr
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Brigham and Women's Hospital
        • Contact:
        • Principal Investigator:
          • Herve Sroussi, Dr.
    • Missouri
      • Columbia, Missouri, United States, 65212
    • New York
      • Buffalo, New York, United States, 14215
        • Recruiting
        • Erie County Medical Center
        • Contact:
        • Principal Investigator:
          • Frustino, Dr
    • North Carolina
      • Charlotte, North Carolina, United States, 28209
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15212
        • Recruiting
        • Alleghany General Hospital
        • Contact:
        • Principal Investigator:
          • Swegal, Dr
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern Medical Center
        • Contact:
          • Thomas Schlieve, DDS, MD
          • Phone Number: 214-648-3034
        • Principal Investigator:
          • Schlieve, MD

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:

  • Completed beam radiation therapy for head and neck cancer at least 3 years prior to the first screening visit
  • No history of parotid gland cancer, recurrent cancer, or a second primary cancer
  • An unstimulated whole saliva flow rate (mL/min) >0 (i.e., at least one drop of saliva in the collection tube)
  • A stimulated whole saliva flow rate (mL/min) within a specified range after mechanical stimulation by chewing
  • Average screening XQ Total Score at or above a specified threshold
  • No evidence of head and neck cancer, defined as a negative otolaryngology exam and a negative computed tomography (CT) scan of the head, neck, and chest with contrast. If a participant has had a magnetic resonance imaging (MRI) study, CT scan, positron emission tomography (PET), or fluorodeoxyglucose-positron emission tomography (FDG-PET) scan of the head, neck, and chest within 6 months of study entry (and at least 3 years after the completion of radiotherapy), then that image may be used for eligibility determination and a CT scan at screening will not be required.
  • Either received treatment with one or more prescription sialagogues and elected to discontinue therapy or, in consultation with their physician, elected to not initiate such treatment
  • Participants taking a prescription sialagogue (specifically, pilocarpine or cevimeline) must stop that medication at least 2 weeks prior to Screening and be willing to refrain from taking such medications for the duration of the study
  • Participants who require medication for an underlying medical condition that is known to affect salivary output must be on stable doses of such medications for at least one month prior to the first screening visit

Exclusion Criteria:

  • Any malignancy within the preceding 3 years, except for treated basal cell or squamous cell carcinoma of the skin or in situ cervical carcinoma
  • History of systemic autoimmune disease affecting the salivary glands (e.g., Sjogren's disease)
  • Currently using systemic immunosuppressive medication(s) (e.g., corticosteroids or biologics) or treated with one within 4 weeks of the first screening visit. Note: Topical, inhaled, or intranasal corticosteroids are permitted.
  • Active viral infection with Epstein-Barr virus (EBV), defined as a positive anti-VCA IgM. In the event a potential participant has a positive anti-VCA IgM, they may be rescreened 2-4 months later at which time a positive Epstein-Barr Virus Nuclear Antigen (EBNA) will be considered as evidence of resolved EBV infection.
  • Evidence of active Hepatitis C virus (HCV) infection
  • Evidence of human immunodeficiency virus (HIV) infection
  • Diagnosis of myasthenia gravis
  • Personal or family history of acute or chronic angle-closure glaucoma (ACG), or at increased risk of developing ACG, or had prophylactic treatment to reduce the risk of developing ACG
  • Known allergy or hypersensitivity to glycopyrrolate
  • Current smokers or history of smoking within the preceding 3 years (includes vaping with tobacco additives)
  • Current alcohol misuse or a history of the same within the preceding 3 years (defined for men as an average intake of more than 14 drinks per week and for women as more than 7 drinks per week)
  • Poorly controlled diabetes (hemoglobin A1c >7%)

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: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AAV2-hAQP1 Group 1
Eligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Genetic: AAV2-hAQP1 Concentration 1
Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Experimental: AAV2-hAQP1 Group 2
Eligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Genetic: AAV2-hAQP1 Concentration 2
Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland
Placebo Comparator: Placebo group
Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland
Other: Placebo
Administration of diluent via Stensen's duct to each parotid gland

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score
Time Frame: 12 months
Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score. The XQ consists of 8 symptom-specific questions the participant rates from 0 (not present) to 10 (worst possible). The XQ Total Score is the sum of all individual item ratings and ranges from 0 to 80.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline to Month 12 in unstimulated whole saliva flow rate
Time Frame: 12 months
Change from Baseline to Month 12 in unstimulated whole saliva flow rate (mL/min)
12 months
Number of participants with treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Time Frame: from Baseline to Month 12
from Baseline to Month 12

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)

June 13, 2023

Primary Completion (Estimated)

July 1, 2025

Study Completion (Estimated)

July 1, 2025

Study Registration Dates

First Submitted

June 22, 2023

First Submitted That Met QC Criteria

June 22, 2023

First Posted (Actual)

July 3, 2023

Study Record Updates

Last Update Posted (Actual)

April 19, 2024

Last Update Submitted That Met QC Criteria

April 18, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MGT-AQP1-201

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.

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

  • Sanford Health
    National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators
    Recruiting
    Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)
    Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions
    United States, Australia

Clinical Trials on AAV2-hAQP1 Concentration 1

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Cambodia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe