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
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Radiation-induced Xerostomia
- Sponsor
- MeiraGTx, LLC
- Enrollment
- 22
- Locations
- 4
- Primary Endpoint
- • Incidence of AEs, treatment-emergent adverse events (TEAEs), and SAEs
- Status
- Active, not recruiting
- Last Updated
- 11 months ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Are willing and able to provide informed consent
- •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)
- •Are willing and able to adhere to the protocol and long-term follow-up
Exclusion Criteria
- •Subjects unwilling or unable to meet with the requirements of the study will be excluded
Outcomes
Primary Outcomes
• Incidence of AEs, treatment-emergent adverse events (TEAEs), and SAEs
Time Frame: 4 years
Safety
Secondary Outcomes
- Changes in saliva flow compared to baseline.(4 years)
- Changes in the MD Anderson Symptom Inventory - Head and Neck(4 years)
- Changes in the Xerostomia Questionnaire relative to Baseline(4 years)
- Changes in the McMaster Global Rate of Change relative to Baseline(4 years)