Long-term Follow-up of Gene Therapy for Radiation-Induced Xerostomia

MeiraGTx

Status and phase: Invitation-only; Phase 2

Conditions: Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands

Treatments: Genetic: AAV2-hAQP1

Study type: Interventional

Funder types: Industry

Identifiers

NCT06544798

MGT-AQP1-202

Details and patient eligibility

About

This study will assess the long-term safety and efficacy of bilateral intra-parotid administration of AAV2-hAQP1 in adults with Grade 2 or Grade 3 radiation-induced late xerostomia.

Full description

Individual participation in this study will last approximately 48 months for participants who received AAV2-hAQP1 in Study MGT-AQP1-201 and at least 60 months for participants who receive AAV2-hAQP1 in Study MGT-AQP1-202.

Enrollment: 120 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Received study drug in Study MGT-AQP1-201

Exclusion criteria

• Withdrew consent to participate in Study MGT-AQP1-201.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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120 participants in 2 patient groups

Follow-up group

No Intervention group

Description:

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 group

Description:

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.

Treatment:

Genetic: AAV2-hAQP1