A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia
This study is investigating a new gene therapy, AAV2-hAQP1, for people who experience severe dry mouth, known as xerostomia, because of radiotherapy treatment for head and neck cancers. This dry mouth can be a long-lasting side effect and significantly impact daily life. The research aims to find out if this gene therapy, given as an injection into the salivary glands, is both safe and effective at helping the body produce more saliva. Researchers will compare different strengths of the gene therapy with a dummy treatment (placebo) to see if it can improve symptoms for those living with radiation-induced dry mouth.
At a glance
What is this study about?
This research study is about helping people who suffer from a very dry mouth, a condition called xerostomia. This often happens as a side effect after having radiotherapy for certain head and neck cancers. For many, this dryness can be quite severe (what doctors call Grade 2 or Grade 3) and can last a long time, making everyday things like eating, speaking, and sleeping difficult.
The study is testing a new gene therapy treatment. Think of gene therapy as a way to send new instructions to parts of your body. In this case, the therapy, called AAV2-hAQP1, aims to target the glands in your mouth that produce saliva. The idea is that it might help these glands work better and produce more moisture, reducing the discomfort of a dry mouth.
Researchers want to find out if this treatment is safe to use and if it actually helps people produce more saliva and feel better. They will give different groups of participants either a dose of the new therapy or a 'dummy' treatment (a placebo) to see if there's a real difference. This is a Phase 2 study, which means it’s still in the earlier stages of testing – they’re checking to see if the treatment works and is safe in a larger group of people than in initial tests.
Key takeaways
- Exploring a new gene therapy for long-term dry mouth after cancer radiotherapy.
- Aimed at adults who had head and neck cancer treatment over 3 years ago.
- Will involve injections into the salivary glands.
- Compares different strengths of the therapy against a dummy treatment (placebo).
- Participation requires stopping certain existing dry mouth medications.
- Focuses on both safety and whether the treatment helps produce more saliva.
Who may be eligible?
To join this study, you need to be an adult (18 or older) who had radiotherapy for head and neck cancer at least three years ago. Your dry mouth must be a lasting problem from that treatment, and you shouldn't have new or returning cancer in your head or neck area, or cancer of the salivary glands themselves. Your scans will need to show that your cancer is still gone.
Doctors will also check your saliva flow. Even if it's low, you must be able to produce at least a tiny bit of saliva naturally. They will also do a test where you chew to stimulate saliva and measure that. You'll also complete a questionnaire about your dry mouth symptoms, and your scores will need to be above a certain level.
It's important that you haven't been relying on certain medications (like pilocarpine or cevimeline) for your dry mouth just before joining, and you'll need to be willing to stop them for the study. If you take other medicines that affect saliva, you need to have been on a steady dose for at least a month.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Did you have radiotherapy for head and neck cancer more than 3 years ago?
- Is your dry mouth a lasting problem from that radiotherapy?
- Are you free from new, returning, or secondary cancers in your head and neck area (excluding certain skin cancers)?
- Are you willing and able to stop certain dry mouth medications (like pilocarpine or cevimeline) for the study duration?
- Can you produce at least a tiny bit of saliva naturally?
What does participation involve?
If you join this study, you might receive one of several different strengths of the new gene therapy or a placebo (a dummy treatment). The treatment will be given as injections into the salivary glands on both sides of your face. You will have regular visits to the clinic so doctors can monitor your health, check your saliva production, and ask about your dry mouth symptoms.
These visits will involve various assessments, including physical exams, blood tests, and questionnaires about how you're feeling. You'll need to stop taking certain dry mouth medications like pilocarpine or cevimeline before the study and throughout its duration. The study will last for a specific period, involving initial treatment and then follow-up to see how you respond.
Potential risks and benefits
Locations (33)
- Banner MD Anderson Cancer CenterVerified postcodeGilbert, United States· Recruiting
- City of HopeVerified postcodeDuarte, United States· Recruiting
- Miami Cancer Institute at Baptist Health South FloridaVerified postcodeMiami, United States· Recruiting
- University of IowaVerified postcodeIowa City, United States· Recruiting
- Johns Hopkins HospitalVerified postcodeBaltimore, United States· Recruiting
- Tufts University School of Dental MedicineVerified postcodeBoston, United States· Recruiting
- Brigham and Women's HospitalVerified postcodeBoston, United States· Recruiting
- Henry Ford HealthVerified postcodeDetroit, United States· Recruiting
- University of MissouriVerified postcodeColumbia, United States· Recruiting
- Washington University - St. LouisVerified postcodeSt Louis, United States· Recruiting
- Erie County Medical CenterVerified postcodeBuffalo, United States· Recruiting
- UNC-Chapel HillVerified postcodeChapel Hill, United States· Recruiting
Common questions
What is xerostomia?
Xerostomia is the medical term for having a very dry mouth, which can make eating, speaking, and swallowing difficult.
What is gene therapy?
Gene therapy is a type of treatment that aims to correct or change the way cells in your body work, often to make them function better.
What does a 'placebo' mean?
A placebo is a dummy treatment that looks just like the real treatment but contains no active medicine. It helps researchers fairly compare how well the new treatment works.
Will I know if I'm getting the real treatment or the placebo?
No, this is usually a 'blinded' study, meaning neither you nor your doctors will know whether you are receiving the active treatment or the placebo until after the study is complete, to keep the results fair.
Can I continue my regular dry mouth medication?
No, if you're taking specific prescription medications for dry mouth (like pilocarpine or cevimeline), you will need to stop them before and during the study.
How to find out more
MeiraGTx Clinical Project Manager
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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