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AAV2-hAQP1 · 1 trial · 1 indication
| 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. |
| Name | Type | Description |
|---|---|---|
| AAV2-hAQP1 | GENETIC | Administration of a prespecified concentration of AAV2-hAQP1 via Stensen's duct to each parotid gland |
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 squamous cell head and neck cancer and for grade 2 and 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract, excluding the parotid glands. It is designed to address irradiation-induced parotid salivary hypofunction.
AAV2-hAQP1 is an adeno-associated viral vector encoding human aquaporin-1. It is designed to deliver the gene for aquaporin-1 to salivary gland tissue to restore salivary function in patients with radiation-induced xerostomia.
AAV2-hAQP1 is being developed by MeiraGTx Holdings plc, a biopharmaceutical company traded on the stock exchange under the ticker symbol MGTX.
AAV2-hAQP1 is in Phase 2 clinical development. It has received FDA designations including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Breakthrough Therapy. It is investigational and not yet approved for commercial use.
AAV2-hAQP1 has been studied in two clinical trials. NCT02446249 was a Phase 1 safety study in 17 participants with irradiation-induced parotid salivary hypofunction, completed in the United States. NCT06544798 is a Phase 2 long-term follow-up study enrolling 276 participants with grade 2 and 3 late xerostomia in the United States and Canada.
Yes, AAV2-hAQP1 is also referred to as AAV2hAQP1 in clinical trial documentation. Both names refer to the same adeno-associated viral vector encoding human aquaporin-1.