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Enrolling by invitationPHASE2INTERVENTIONAL

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

This research is a follow-up study for adults who previously received a gene therapy treatment called AAV2-hAQP1. These individuals had severe dry mouth (xerostomia) as a result of radiation treatment for certain head and neck cancers, but not those involving the parotid glands. The main aim is to carefully observe the long-term safety and how well this gene therapy continues to work. By understanding its effects over several years, doctors hope to learn more about its potential to help people with this challenging side effect of cancer treatment. Your participation helps us understand if this treatment could offer a lasting solution for dry mouth.

At a glance

Status
Enrolling by invitation
Phase
PHASE2
Sponsor
MeiraGTx, LLC
Enrolment target
276
Start
02 Aug 2024
Estimated completion
01 Feb 2032

What is this study about?

Imagine your mouth feeling constantly dry, making it hard to eat, speak, or even swallow. This is a common and difficult side effect called xerostomia, which can happen to people after they've had radiotherapy for certain cancers in the head and neck area. This research study is a long-term follow-up for individuals who previously took part in an earlier study looking at a special treatment for this severe dry mouth.

The treatment being studied is a type of gene therapy called AAV2-hAQP1. It's designed to help the glands in your mouth that produce saliva work better. The 'gene therapy' part means it introduces a new gene into the cells to encourage them to produce more moisture. This study focuses on people whose dry mouth is caused by past radiotherapy, but specifically excludes cases where the parotid glands (large salivary glands near your ears) were directly affected.

The main goal of this particular follow-up study is to see how safe and effective this gene therapy is over a much longer period. Researchers want to understand if the benefits last and if there are any long-term effects. The information gathered from participants will be very important in helping doctors understand if this treatment could become a real option for many more people suffering from this often distressing side effect of cancer treatment.

Key takeaways

  • This is a follow-up study, not a new treatment study.
  • It's for people who previously received a gene therapy for dry mouth.
  • Aims to check long-term safety and how well the treatment continues to work.
  • Participation lasts for several years (4-5 years).
  • Your input helps future patients with severe dry mouth.

Who may be eligible?

This follow-up study is only for a very specific group of people. To be considered, you must have already taken part in a previous study related to this gene therapy, called Study MGT-AQP1-201.

Basically, if you previously received the AAV2-hAQP1 treatment as part of that earlier study, you would be eligible for this long-term follow-up. This is because the researchers want to continue monitoring the people who have already had the treatment.

However, you cannot take part if you previously decided to stop being part of Study MGT-AQP1-201. The study is open to both men and women aged 18 and over.

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.

  1. Did you take part in Study MGT-AQP1-201 and receive the study treatment?
  2. Are you 18 years old or older?
  3. Did you NOT withdraw from Study MGT-AQP1-201 previously?
Answer every question to see your result.

What does participation involve?

If you decide to take part in this follow-up study, it won't involve receiving any new treatment; it's all about checking in on your health after your previous gene therapy. The total time you'll be involved varies. If you received the treatment in Study MGT-AQP1-201, your participation will last about 48 months (four years). If you received it in a different previous study (Study MGT-AQP1-202), your participation will last at least 60 months (five years).

During this time, you will have scheduled visits with the study team. These visits will likely involve checking your general health, asking questions about your dry mouth, and possibly some tests to see how the gene therapy is still working. The aim is to carefully monitor your progress and any long-term effects of the treatment you received previously.

Potential risks and benefits

Taking part in this study may not directly benefit you, as it is a follow-up to a treatment you've already received. However, the information gathered from your participation is extremely valuable. It helps researchers understand the long-term safety and effectiveness of this gene therapy, potentially leading to new treatment options for many others suffering from dry mouth caused by radiotherapy. As with any study, there could be unforeseen risks or side effects from the previous treatment that might only become clear over time. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (8)

  • Banner MD Anderson Cancer Center
    Verified postcode
    Gilbert, United States
  • Miami Cancer Institute at Baptist Health South Florida
    Verified postcode
    Miami, United States
  • University of Iowa
    Verified postcode
    Iowa City, United States
  • University of Missouri
    Verified postcode
    Columbia, United States
  • Erie County Medical Center
    Verified postcode
    Buffalo, United States
  • Atrium Health
    Verified postcode
    Charlotte, United States
  • Alleghany General Hospital
    Verified postcode
    Pittsburgh, United States
  • Shirley and Jim Fielding Northeast Cancer Centre - Health Sciences North
    Verified postcode
    Greater Sudbury, Canada

Common questions

What is this study about?

This study is a long-term follow-up for people who previously received a special gene therapy for severe dry mouth caused by radiation treatment for certain cancers.

Why is this study important?

It helps doctors understand how safe and effective this gene therapy treatment is over many years, which could lead to better treatments for dry mouth in the future.

Will I get new treatment in this study?

No, this study is about monitoring your health and the effects of the treatment you received in a previous study. You won't get new treatment.

How long will I be involved?

Your involvement will last about 4 or 5 years, depending on which previous study you were part of.

Can I stop participating if I want to?

Yes, you are free to withdraw from the study at any time without it affecting your usual medical care.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "Long-term Follow-up of Gene Therapy for Radiation-Induced Xe…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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