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Also known as AAV2-hAQP1 Concentration 1
AAV2-hAQP1 · 3 trials · 4 indications
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.
| Arm | Type | Description |
|---|---|---|
| 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. |
| Cohort 1: AAV2-hAQP1 Group 1 | EXPERIMENTAL | Eligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| Cohort 1: AAV2-hAQP1 Group 2 | EXPERIMENTAL | Eligible participants will receive up to 3 mL of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| Cohort 1: Placebo group | PLACEBO_COMPARATOR | Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland |
| Cohort 2: AAV2-hAQP1 Group 3 | EXPERIMENTAL | Eligible participants will receive up to 3 mL of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| Cohort 2: AAV2-hAQP1 Group 4 | EXPERIMENTAL | Eligible participants will receive up to 3 mL of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| Cohort 2 Placebo group | PLACEBO_COMPARATOR | Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland |
| single arm dose escalation | EXPERIMENTAL | single arm dose escalation |
| Name | Type | Description |
|---|---|---|
| AAV2-hAQP1 | GENETIC | Administration of a prespecified concentration of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| AAV2-hAQP1 Concentration 1 | GENETIC | Administration of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| AAV2-hAQP1 Concentration 2 | GENETIC | Administration of concentration 2 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| Placebo | OTHER | Administration of diluent via Stensen's duct to each parotid gland |
| AAV2-hAQP1 Concentration 3 | GENETIC | Administration of concentration 3 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| AAV2-hAQP1 Concentration 4 | GENETIC | Administration of concentration 4 of AAV2-hAQP1 via Stensen's duct to each parotid gland |
| AAV2hAQP1 | BIOLOGICAL | Infusion of gene therapy |
Inclusion Criteria: * Received study drug in Study MGT-AQP1-201 Exclusion Criteria: * Withdrew consent to participate in Study MGT-AQP1-201.
AAV2-hAQP1 is an investigational gene therapy being developed for grade 2 and 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract, excluding the parotid glands. It is also studied in squamous cell head and neck cancer with radiation-induced xerostomia and salivary hypofunction.
AAV2-hAQP1 targets aquaporin-1, or AQP1. It is an adeno-associated viral vector that delivers the human AQP1 gene to salivary glands, with the aim of restoring water transport and improving saliva production in patients with radiation-induced xerostomia.
AAV2-hAQP1 is being developed by MeiraGTx Holdings plc, which trades on the Nasdaq under the ticker MGTX. The company is the sponsor of the clinical program evaluating this gene therapy in radiation-induced xerostomia.
AAV2-hAQP1 is in Phase 2 clinical development. It has not been approved by the FDA and remains investigational. The program has received RMAT, orphan drug, and breakthrough therapy designations from the FDA.
AAV2-hAQP1 has been studied in three trials: a completed Phase 1 trial, NCT02446249, and two Phase 2 trials, NCT05926765 and NCT06544798. The Phase 2 trials are randomized, double-blind, and placebo-controlled, enrolling adults in the United States, Canada, and the United Kingdom.
Yes, AAV2-hAQP1 is also known as AAV2hAQP1 and AAV2-hAQP1 Concentration 1. These names refer to the same investigational gene therapy developed by MeiraGTx Holdings plc for radiation-induced xerostomia.