A follow-up study of AAV2-hAQP1 in participants with radiation induced late xerostomia

  • Research type

    Research Study

  • Full title

    Long-term Follow-up of AAV2-hAQP1 Gene Therapy in Participants with Radiation-Induced Late Xerostomia

  • IRAS ID

    1012478

  • Contact name

    Angela Mikroulis

  • Contact email

    angela.mikroulis@meiragtx.com

  • Sponsor organisation

    MeiraGTx, LLC

  • Clinicaltrials.gov Identifier

    NCT06544798

  • Research summary

    This follow-up study will follow participants that were part of the MGT-AQP1-201 study, a gene therapy trial for the treatment of late xerostomia caused by radiation therapy for head and neck cancer. The study will start with the rollover visit which will be performed at the same time as the Month 12 visit on the MGT-AQP1-201 study. If in the initial study, participants received the gene therapy treatment, they will continue on this long term follow-up study for an additional 48 months from their end of study visit on the MGT-AQP1-201 study. However, if a participant received placebo treatment in the initial trial, they will be offered the gene therapy treatment (as long as their are still eligible to continue) as part of this long term follow-up study. This will occur at the time of unblinding of the initial trial, which will happen after the last subject on the initial trial has performed their last visit on the study, anticipated to be completed in the first quarter of the year in 2027. Unblinding is the process in which the study team finds out who received the gene therapy treatment and who received the placebo treatment on the MGT-AQP1-201 study. Once a participant receives the gene therapy treatment as part of this long term follow-up trial, they will be followed up for 60 months.
    This long term follow-up study will continue to collect data to better understand how safe the drug is over the long term and if the study drug is working. over the long term. It is a regulatory requirement to follow-up with participants for 5 years after receiving a gene therapy drug as it requires a generally longer time period to better understand if the drug works well and how safe it is for patients.

  • REC name

    South Central - Oxford A Research Ethics Committee

  • REC reference

    25/SC/0270

  • Date of REC Opinion

    26 Sep 2025

  • REC opinion

    Further Information Favourable Opinion