Clinical trial NCT05060341: Long-Term Follow-Up Study of AAV2hAQP1 for Radiation Induced Xerostomia
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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Radiation-induced Xerostomia
Lead sponsor: MeiraGTx UK II Ltd
Sponsor class: INDUSTRY
First posted: 2021-09-29
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 81
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects with radiation induced parotid gland hypofunction and xerostomia who have particpated in the MGT016 AAV2hAQP1 study.

Brief 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

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.

Eligibility criteria: 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

Primary outcomes:
measure: • Incidence of AEs, treatment-emergent adverse events (TEAEs), and SAEs
timeFrame: 4 years
Secondary outcomes:
measure: Changes in saliva flow compared to baseline.
timeFrame: 4 years
measure: Changes in the McMaster Global Rate of Change relative to Baseline
timeFrame: 4 years
measure: Changes in the Xerostomia Questionnaire relative to Baseline
timeFrame: 4 years
measure: Changes in the MD Anderson Symptom Inventory - Head and Neck
timeFrame: 4 years
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Leland Stanford Junior University
state: California
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Charlotte
country: United States
facility: Atrium Health
state: North Carolina
city: Sudbury
country: Canada
facility: Health Sciences North - Northeast Cancer Center
state: Ontario
Study officials:
References: