Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human A… (NCT02446249) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction
United States17 participantsStarted 2015-05-04
Plain-language summary
Background:
\- Radiation can cause the parotid salivary glands to make less saliva (dry mouth). This can cause problems like infections and tooth decay. Researchers hope a new drug can help people with dry mouth caused by radiation.
Objectives:
\- To examine the safety of AAV2hAQP1 gene therapy. To see if the drug increases saliva in people whose parotid glands have had radiation.
Eligibility:
\- People at least 18 years of age with a history of radiation therapy for head and neck cancer.
Design:
Participants will be screened in 2 visits with:
* medical history
* physical exam
* scans of the head, neck, and chest
* intravenous administration of glycopyrrolate to stop saliva
* saliva collections
* sialogram which is a procedure in which a substance is injected in the parotid gland and X-rays are taken.
* non-drug infusion
* a small piece of skin being taken
3-5-day hospital stay: Participants will receive the gene infusion. The AAV2hAQP1 will be in a solution in a syringe. It will be slowly pushed into the parotid gland through the parotid duct, an opening in the mouth near the second upper molar tooth.
10 outpatient visits over 3 years. These may include:
* repeats of selected screening tests, including saliva collection
* blood and urine tests
* oral and dental examinations
* head and neck exams, including the use of a thin scope to see the back of the throat
* questionnaires
* a small piece of parotid tissue being taken by either a small scope through the parotid duct or by a small needle guided by ultrasound
* scans of the head and neck. For some, contrast will be injected in a vein
* completion of a diary about how the participant feels between visits
* swabs of teeth and gums to assess the microbiome of the mouth
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. At least 18 years of age
. History of external beam radiation therapy for head and neck cancer, with a mean dose equal to or greater than 15 Gy to a parotid gland.
. Abnormal parotid gland function as judged by both absence of unstimulated parotid salivary flow and a stimulated parotid salivary flow in the targeted parotid gland \>0 and \<0.3 mL/min/gland after 2% citrate stimulation.
. No evidence of recurrence of primary malignancy by otolaryngology (ENT) assessment. Additionally, all patients must have been disease-free of head and neck cancer for at least 5 years, a status to be determined at pre-dose screening using negative clinical exams and PET and or CT imaging of the neck and chest. The anatomic subset of HPV positive oropharyngeal cancer may be enrolled after 2 years post completion of therapy.
. Willingness to practice the required birth control method ("barrier" contraception, condoms, diaphragm) until AAV2hAQP1 is no longer detectable in their serum or saliva.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Women who cannot bear children (post-menopausal or due to a surgical intervention) also will be required to practice barrier birth control methods until AAV2hAQP1 is no longer detectable in their serum or saliva.
. Ability to stay at the NIH hospital for the period of time necessary to complete each on-site phase of the protocol (3-5 days).
. No history of allergies to any medications or agents to be used in this protocol.
Exclusion criteria
. Pregnant or lactating women. Women of childbearing potential are required to have a negative serum pregnancy test at screening and a negative urine pregnancy test within 48 hours prior to gene infusion.
. Any experimental therapy within 3 months.
. Any active respiratory tract infection in the 3 weeks prior to day 1 of the protocol
. Active infection that requires the use of intravenous antibiotics and does not resolve at least 1 week before Day 1.
. Uncontrolled ischemic heart disease: unstable angina, evidence of active ischemic heart disease on ECG, congestive heart failure (left ventricular ejection fraction \< 45% on MUGA or echo) or cardiomyopathy.
. Asthma or chronic obstructive pulmonary disease requiring regular inhaled or systemic corticosteroids.
. Individuals with a history of autoimmune diseases affecting salivary glands, including Sjogren's syndrome, lupus, scleroderma, type I diabetes, sarcoidosis, amyloidosis, and chronic graft versus host disease. Organ specific autoimmune conditions may be included if clinically stable.
. Use of systemic immunosuppressive medications (,i.e., corticosteroids). Topical corticosteroids are allowed.