What side effects are associated with this type of intervention?

Common treatments for dry mouth include saliva substitutes, medications to stimulate saliva production, and gene therapy like AAV2-hAQP1. Saliva substitutes generally have minimal side effects but can sometimes cause irritation or an unpleasant taste. Medications such as pilocarpine and cevimeline may cause sweating, nausea, and increased urination. Gene therapy, like AAV2-hAQP1, aims to increase water transport and saliva production, but potential side effects could include immune reactions, local inflammation, and off-target effects. It is essential to discuss these options and their side effects with a healthcare provider.

What prior FDA approvals exist?

The provided context does not mention specific FDA-approved treatments for dry mouth that are directly similar to the AAV2-hAQP1 gene therapy. However, it does reference various treatments for related conditions, such as the use of anticholinergic agents like glycopyrrolate and scopolamine for drooling, and the use of topical agents like fluorometholone and cyclosporine for dry eye disease. These treatments aim to manage symptoms by either reducing saliva production or increasing tear production, respectively. For dry mouth specifically, common treatments include saliva substitutes, sialogogues like pilocarpine and cevimeline, and good oral hygiene practices.

What other types of research exist?

Promising alternatives to AAV2-hAQP1 for treating dry mouth include: 1) AdrAQP5, a recombinant adenovirus-mediated gene therapy that increases water permeability in salivary cells. 2) Autologous serum eye drops, which have shown efficacy in treating severe dry eye disease and may have potential applications for dry mouth. 3) ABCB5+ mesenchymal stem cells (allo-APZ2-EB), which have demonstrated efficacy in reducing symptoms in epidermolysis bullosa and could be explored for their regenerative properties in salivary glands.

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Gene Therapy

Gene Therapy for Radiation-Induced Dry Mouth (AQUAX2 Trial)

Phase 2

Recruiting

Research Sponsored by MeiraGTx, LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Summary

This trial will test a gene therapy injection into the salivary glands to help adults with severe dry mouth caused by radiation therapy produce more saliva. Gene therapy for dry mouth disorders has moved from theoretical to clinical proof with an initial trial using a specific method to express a protein in patients with radiation-induced dry mouth.

Who is the study for?

Adults who've had radiation therapy for head and neck cancer at least 3 years ago, have a certain level of dry mouth severity, and can produce some saliva. They shouldn't have recurring or new cancers, be on specific medications affecting saliva, or have conditions like uncontrolled diabetes, recent smoking history, alcohol misuse, autoimmune diseases affecting salivary glands or certain infections.

What is being tested?

The trial is testing two different doses of AAV2-hAQP1 gene therapy against a placebo to see if they can safely improve dry mouth caused by past radiation treatments. Participants will receive the treatment directly into their parotid gland (a major salivary gland).

What are the potential side effects?

While not explicitly listed in the provided information, potential side effects may include reactions at the injection site such as pain or swelling, immune responses to the viral vector used in gene therapy (like fever), and possible unintended effects on saliva production.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from Baseline to Month 12 in Xerostomia-specific Questionnaire (XQ) Total Score

Secondary study objectives

Change from Baseline to Month 12 in unstimulated whole saliva flow rate

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: AAV2-hAQP1 Group 2Experimental Treatment1 Intervention

Eligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Group II: AAV2-hAQP1 Group 1Experimental Treatment1 Intervention

Eligible participants will receive up to 3 mL of concentration 1 of AAV2-hAQP1 via Stensen's duct to each parotid gland

Group III: Placebo groupPlacebo Group1 Intervention

Eligible participants will receive up to 3 mL of diluent via Stensen's duct to each parotid gland

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Gene therapy treatments for dry mouth, such as AAV2-hAQP1, work by introducing genes that encode water channel proteins like aquaporin-1 into the salivary glands. These proteins facilitate the movement of water across cell membranes, thereby increasing saliva production and alleviating symptoms of dry mouth. This approach is particularly significant for patients with radiation-induced xerostomia, as it directly addresses the underlying issue of impaired water transport in salivary glands. Other treatments, such as artificial saliva and medications that stimulate saliva production, provide symptomatic relief but do not target the root cause. Gene therapy offers a promising, long-term solution by restoring the natural function of salivary glands.

Adenovirus-mediated expression of aquaporin-5 in epithelial cells.[Transcatheter delivery of recombinant adenovirus vector containing exogenous aquaporin gene in treatment of Sjögren's syndrome].Early responses to adenoviral-mediated transfer of the aquaporin-1 cDNA for radiation-induced salivary hypofunction.