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Long Term Follow up Study of AAV2hAQP1 for Radiation Induced Xerostomia

Sponsor
MeiraGTx UK II Ltd (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT05060341
Collaborator
(none)
30
Enrollment
5
Locations
83.6
Anticipated Duration (Months)
6
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is a longer-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

Condition or Disease Intervention/Treatment Phase

    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 24, 36, 48, and 60 months following study drug administration (for a total of 4 visits).

    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; 12-lead ECGs; ear, nose, and throat (ENT) screening for malignancy; imaging assessments (if indicated); salivary flow assessments; and questionnaire assessments.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    30 participants
    Observational Model:
    Cohort
    Time Perspective:
    Other
    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
    Actual Study Start Date :
    Nov 12, 2020
    Anticipated Primary Completion Date :
    Nov 1, 2026
    Anticipated Study Completion Date :
    Nov 1, 2027

    Outcome Measures

    Primary Outcome Measures

    1. • Incidence of AEs, treatment-emergent adverse events (TEAEs), and SAEs [4 years]

      Safety

    Secondary Outcome Measures

    1. Changes in saliva flow compared to baseline. [4 years]

      Changes in unstimulated and stimulated salivary output (mL/minute) of the treated parotid gland and/or whole saliva output as compared to baseline.

    2. Changes in the McMaster Global Rate of Change relative to Baseline [4 years]

      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.

    3. Changes in the Xerostomia Questionnaire relative to Baseline [4 years]

      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. Changes in the MD Anderson Symptom Inventory - Head and Neck [4 years]

      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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Inclusion Criteria:

    1. Are willing and able to provide informed consent

    2. Received AAV2hAQP1 administered via Stensen's duct to a single parotid gland 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Louisville Louisville Kentucky United States 40202
    2 Brigham and Women's Hospital Boston Massachusetts United States 02184
    3 Memorial Sloan Kettering Cancer Center New York New York United States 10065
    4 Atrium Health Charlotte North Carolina United States 28209
    5 Health Sciences North - Northeast Cancer Center Sudbury Ontario Canada

    Sponsors and Collaborators

    • MeiraGTx UK II Ltd

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    MeiraGTx UK II Ltd
    ClinicalTrials.gov Identifier:
    NCT05060341
    Other Study ID Numbers:
    • MGT-AQP1-102
    First Posted:
    Sep 29, 2021
    Last Update Posted:
    Sep 29, 2021
    Last Verified:
    Sep 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Xerostomia

    Study Results

    No Results Posted as of Sep 29, 2021