Radiation-Induced Parotid Gland HypofunctionPhase 1NCT04043104
A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia
A Phase 1 study testing AAV2hAQP1: 1 x 10^11 vg/gland (single gland), AAV2hAQP1: 3 x 10^10 vg/gland (both glands) and AAV2hAQP1: 3 x 10^11 vg/gland (single gland) in people with Radiation-Induced Parotid Gland Hypofunction and Xerostomia Due to Radiotherapy, run by MeiraGTx UK II Ltd at 6 sites in 2 countries.
Trial summary
Open-label, non-randomized, dose escalation trial of AAV2hAQP1 administered via Stensen's duct to a single or both parotid glands in subjects with radiation-induced xerostomia The objectives are to evaluate the safety and identify either a maximum tolerated dose or a maximum feasible dose of a single dose of AAV2hAQP1… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Radiation-Induced Parotid Gland Hypofunction, Xerostomia Due to Radiotherapy, Head and Neck Cancer
- What is being tested?
- AAV2hAQP1: 1 x 10^11 vg/gland (single gland), AAV2hAQP1: 3 x 10^10 vg/gland (both glands) and AAV2hAQP1: 3 x 10^11 vg/gland (single gland)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 30 Jun 2019to 28 Mar 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subjects ≥18 years of age.
- History of radiation therapy for head and neck cancer.
- 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 the primary malignancy by an otolaryngology (ears, nose, and throat [ENT]) assessment. Additionally, all subjects must be disease-free of head and neck cancer for at least 5 years following the end of treatment at screening, with the exception of subjects with a history of HPV+ OPC (base of tongue, oropharynx, pharynx, soft palate, tonsil) who must be disease free for at least 2 years following the end of treatment. Disease status will be determined by negative clinical examinations and computed tomography (CT) scans of the neck and chest. If subjects have had a magnetic resonance imaging (MRI) of the neck or a positron emission tomography (PET) scan within 6 months of screening, then a CT scan is not required, except for HPV+ OPC subjects who must have scans at 2 years post treatment.
- Female subjects of childbearing potential (i.e., ovulating, pre-menopausal, and not surgically sterile) and all male subjects must use a medically accepted contraceptive regimen during their participation in the study and until all samples collected at 2 consecutive visits following AAV2hAQP1 administration are negative. Acceptable methods of contraception for male subjects include the following:
- Condoms with spermicide. Acceptable methods of contraception for female subjects include the following:
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- Intrauterine device for at least 12 weeks prior to Screening.
- Hormonal contraception (oral, implant, injection, ring, or patch) for at least 12 weeks prior to Screening.
- Diaphragm used in combination with spermicide.
Who cannot join
- Pregnant or lactating women or women planning to become pregnant.
- Any experimental therapy within 3 months before Day 1.
- Active infection that requires the use of intravenous antibiotics and does not resolve at least 1 week before Day 1.
- Uncontrolled ischemic heart disease (i.e., unstable angina, evidence of active ischemic heart disease on electrocardiogram [ECG]).
- History of systemic autoimmune diseases affecting the salivary glands.
- Use of systemic immunosuppressive medications (i.e., corticosteroids).
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- o Note: Topical, inhaled, or intranasal corticosteroids are allowed.
- Malignancy, other than head and neck cancer, within the past 3 years, with the exception of adequately treated basal cell or squamous cell carcinoma of the skin or in situ cervical carcinoma.
- Active infections including, Epstein-Barr virus (EBV), cytomegalovirus (CMV), hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV) infection.
- White blood cell count <3000/μL, absolute neutrophil count <1500/μL, hemoglobin <10.0 g/dL, platelet count <100,000/μL, or absolute lymphocyte count ≤500/μL.
- Alanine aminotransferase and/or aspartate aminotransferase >1.5 × the upper limit of normal (ULN), alkaline phosphatase >1.5 × ULN, or total bilirubin >1.5 × ULN with any elevation of liver enzymes.
- Estimated glomerular filtration rate <60 mL/min/1.73 m2 using the Modification of Diet in Renal Disease equation.
- Active use of tobacco products as determined by self-reporting.
- Allergy to iodine or shellfish, and thus unable to have sialographic evaluations.
- Allergy or hypersensitivity to glycopyrrolate.
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
Brigham and Women's Hospital
Boston, Massachusetts, 02184
Atrium Health
Charlotte, North Carolina, 28209
University of Louisville
Louisville, Kentucky, 40202
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Leland Stanford Junior University
Stanford, California, 94305
Canada1 site
Health Sciences North - Northeast Cancer Center
Greater Sudbury, Ontario
About this study
Open-label, non-randomized, dose escalation trial of AAV2hAQP1 administered via Stensen's duct to a single or both parotid glands in subjects with radiation-induced xerostomia The objectives are to evaluate the safety and identify either a maximum tolerated dose or a maximum feasible dose of a single dose of AAV2hAQP1 infused into one or both parotid glands:
To evaluate subject improvement of xerostomia symptoms, to evaluate the increase in parotid gland salivary output after treatment with AAV2hAQP1, to evaluate additional efficacy outcomes.
- Conditions
- Radiation-Induced Parotid Gland Hypofunction, Xerostomia Due to Radiotherapy, Head and Neck Cancer
- Being tested
- AAV2hAQP1: 1 x 10^11 vg/gland (single gland), AAV2hAQP1: 3 x 10^10 vg/gland (both glands), AAV2hAQP1: 3 x 10^11 vg/gland (single gland), AAV2hAQP1: 1 x 10^11 vg/gland (both glands), AAV2hAQP1: 1 x 10^12 vg/gland (single gland)
- Medical subject headings
- Head and Neck Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV2hAQP1: 1 x 10^11 vg/gland (single gland)Drug
Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10^11 vg/gland
AAV2hAQP1: 3 x 10^10 vg/gland (both glands)Drug
Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10^10 vg/gland
AAV2hAQP1: 3 x 10^11 vg/gland (single gland)Drug
Intra-parotid administration of AAV2hAQP1 of via Stensen's duct to a single parotid gland at a dose level of 3 x 10^11 vg/gland
AAV2hAQP1: 1 x 10^11 vg/gland (both glands)Drug
intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10^11 vg/gland
AAV2hAQP1: 1 x 10^12 vg/gland (single gland)Drug
Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10^12 vg/gland
AAV2hAQP1: 3 x 10^11 vg/gland (both glands)Drug
Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10^11 vg/gland
AAV2hAQP1: 3 x 10^12 vg/gland (single gland)Drug
Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 3 x 10^12 vg/gland
AAV2hAQP1: 1 x 10^12 vg/gland (both glands)Drug
Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10^12 vg/gland
| Study group | Role | What they receive |
|---|---|---|
| 1 x 10^11 vg/gland (single gland) | Experimental | Not described Receives: Drug: AAV2hAQP1: 1 x 10^11 vg/gland (single gland) |
| 3 x 10^10 vg/gland (both glands) | Experimental | Not described Receives: Drug: AAV2hAQP1: 3 x 10^10 vg/gland (both glands) |
| 3 x 10^11 vg/gland (single gland) | Experimental | Not described Receives: Drug: AAV2hAQP1: 3 x 10^11 vg/gland (single gland) |
| 1 x 10^11 vg/gland (both glands) | Experimental | Not described Receives: Drug: AAV2hAQP1: 1 x 10^11 vg/gland (both glands) |
| 1 x 10^12 vg/gland (single gland) | Experimental | Not described Receives: Drug: AAV2hAQP1: 1 x 10^12 vg/gland (single gland) |
| 3 x 10^11 vg/gland (both glands) | Experimental | Not described Receives: Drug: AAV2hAQP1: 3 x 10^11 vg/gland (both glands) |
| 3 x 10^12 vg/gland (single gland) | Experimental | Not described Receives: Drug: AAV2hAQP1: 3 x 10^12 vg/gland (single gland) |
| 1 x 10^12 vg/gland (both glands) | Experimental | Not described Receives: Drug: AAV2hAQP1: 1 x 10^12 vg/gland (both glands) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome is safety of AAV2hAQP1 administered to the parotid gland of adult subjects with radiation-induced xerostomia
Safety will be assessed by number of adverse events occurring with treatment
Measured: one day to one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2019
- Study start30 Jun 2019
- Primary completion28 Mar 2023
- Study completion28 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Radiation-Induced Parotid Gland HypofunctionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx UK II Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04043104
- Sponsor's protocol ID
- MGT016
- Status last verified
- Apr 2023
- First submitted
- 23 Jul 2019
- Last updated
- 24 Apr 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia
- ClinicalTrials.gov ID
- NCT04043104
- Acronym / sponsor ID
- MGT016
- Conditions
- Radiation-Induced Parotid Gland Hypofunction, Xerostomia Due to Radiotherapy, Head and Neck Cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- MeiraGTx UK II LtdIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2019 (actual)
- Primary completion
- 28 Mar 2023 (actual)
- Study completion
- 28 Mar 2023 (actual)
- First posted
- 2 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Open-label, non-randomized, dose escalation trial of AAV2hAQP1 administered via Stensen's duct to a single or both parotid glands in subjects with radiation-induced xerostomia The objectives are to evaluate the safety and identify either a maximum tolerated dose or a maximum feasible dose of a single dose of AAV2hAQP1 infused into one or both parotid glands:
To evaluate subject improvement of xerostomia symptoms, to evaluate the increase in parotid gland salivary output after treatment with AAV2hAQP1, to evaluate additional efficacy outcomes.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| 1 x 10^11 vg/gland (single gland) | Experimental | Drug: AAV2hAQP1: 1 x 10^11 vg/gland (single gland) |
| 3 x 10^10 vg/gland (both glands) | Experimental | Drug: AAV2hAQP1: 3 x 10^10 vg/gland (both glands) |
| 3 x 10^11 vg/gland (single gland) | Experimental | Drug: AAV2hAQP1: 3 x 10^11 vg/gland (single gland) |
| 1 x 10^11 vg/gland (both glands) | Experimental | Drug: AAV2hAQP1: 1 x 10^11 vg/gland (both glands) |
| 1 x 10^12 vg/gland (single gland) | Experimental | Drug: AAV2hAQP1: 1 x 10^12 vg/gland (single gland) |
| 3 x 10^11 vg/gland (both glands) | Experimental | Drug: AAV2hAQP1: 3 x 10^11 vg/gland (both glands) |
| 3 x 10^12 vg/gland (single gland) | Experimental | Drug: AAV2hAQP1: 3 x 10^12 vg/gland (single gland) |
| 1 x 10^12 vg/gland (both glands) | Experimental | Drug: AAV2hAQP1: 1 x 10^12 vg/gland (both glands) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female subjects ≥18 years of age.
- History of radiation therapy for head and neck cancer.
- 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 the primary malignancy by an otolaryngology (ears, nose, and throat [ENT]) assessment. Additionally, all subjects must be disease-free of head and neck cancer for at least 5 years following the end of treatment at screening, with the exception of subjects with a history of HPV+ OPC (base of tongue, oropharynx, pharynx, soft palate, tonsil) who must be disease free for at least 2 years following the end of treatment. Disease status will be determined by negative clinical examinations and computed tomography (CT) scans of the neck and chest. If subjects have had a magnetic resonance imaging (MRI) of the neck or a positron emission tomography (PET) scan within 6 months of screening, then a CT scan is not required, except for HPV+ OPC subjects who must have scans at 2 years post treatment.
- Female subjects of childbearing potential (i.e., ovulating, pre-menopausal, and not surgically sterile) and all male subjects must use a medically accepted contraceptive regimen during their participation in the study and until all samples collected at 2 consecutive visits following AAV2hAQP1 administration are negative. Acceptable methods of contraception for male subjects include the following:
Key exclusion criteria (15 listed)
- Pregnant or lactating women or women planning to become pregnant.
- Any experimental therapy within 3 months before Day 1.
- Active infection that requires the use of intravenous antibiotics and does not resolve at least 1 week before Day 1.
- Uncontrolled ischemic heart disease (i.e., unstable angina, evidence of active ischemic heart disease on electrocardiogram [ECG]).
- History of systemic autoimmune diseases affecting the salivary glands.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV2hAQP1: 1 x 10^11 vg/gland (single gland)Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10^11 vg/gland | Drug | 1 x 10^11 vg/gland (single gland) |
| AAV2hAQP1: 3 x 10^10 vg/gland (both glands)Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10^10 vg/gland | Drug | 3 x 10^10 vg/gland (both glands) |
| AAV2hAQP1: 3 x 10^11 vg/gland (single gland)Intra-parotid administration of AAV2hAQP1 of via Stensen's duct to a single parotid gland at a dose level of 3 x 10^11 vg/gland | Drug | 3 x 10^11 vg/gland (single gland) |
| AAV2hAQP1: 1 x 10^11 vg/gland (both glands)intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10^11 vg/gland | Drug | 1 x 10^11 vg/gland (both glands) |
| AAV2hAQP1: 1 x 10^12 vg/gland (single gland)Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 1 x 10^12 vg/gland | Drug | 1 x 10^12 vg/gland (single gland) |
| AAV2hAQP1: 3 x 10^11 vg/gland (both glands)Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10^11 vg/gland | Drug | 3 x 10^11 vg/gland (both glands) |
| AAV2hAQP1: 3 x 10^12 vg/gland (single gland)Intra-parotid administration of AAV2hAQP1 via Stensen's duct to a single parotid gland at a dose level of 3 x 10^12 vg/gland | Drug | 3 x 10^12 vg/gland (single gland) |
| AAV2hAQP1: 1 x 10^12 vg/gland (both glands)Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10^12 vg/gland | Drug | 1 x 10^12 vg/gland (both glands) |
Primary outcome
The primary outcome is safety of AAV2hAQP1 administered to the parotid gland of adult subjects with radiation-induced xerostomia
Time frame: one day to one year
Safety will be assessed by number of adverse events occurring with treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2023.
Related trials for Radiation-Induced Parotid Gland Hypofunction
Active, not recruitingObservational study
Long-Term Follow-Up Study of AAV2hAQP1 for Radiation Induced Xerostomia
Radiation-induced Xerostomia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AAV2hAQP1: 1 x 10^11 vg/gland (single gland), AAV2hAQP1: 3 x 10^10 vg/gland (both glands) and AAV2hAQP1: 3 x 10^11 vg/gland (single gland) in people with Radiation-Induced Parotid Gland Hypofunction and Xerostomia Due to Radiotherapy, run by MeiraGTx UK II Ltd at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary outcome is safety of AAV2hAQP1 administered to the parotid gland of adult subjects with radiation-induced xerostomia (time frame: one day to one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2023.