Ignite Creation Date:
2025-12-24 @ 7:56 PM
Ignite Modification Date:
2025-12-25 @ 5:31 PM
Study NCT ID:
NCT04043104
Status:
COMPLETED
Last Update Posted:
2023-04-24
First Post:
2019-07-23
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief 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
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006258', 'term': 'Head and Neck Neoplasms'}], 'ancestors': [{'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 24}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-06-30', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2023-04', 'completionDateStruct': {'date': '2023-03-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-04-21', 'studyFirstSubmitDate': '2019-07-23', 'studyFirstSubmitQcDate': '2019-08-01', 'lastUpdatePostDateStruct': {'date': '2023-04-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-08-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-03-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The primary outcome is safety of AAV2hAQP1 administered to the parotid gland of adult subjects with radiation-induced xerostomia', 'timeFrame': 'one day to one year', 'description': 'Safety will be assessed by number of adverse events occurring with treatment'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Radiation-Induced Parotid Gland Hypofunction', 'Xerostomia Due to Radiotherapy', 'Head and Neck Cancer']}, 'descriptionModule': {'briefSummary': "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:\n\nTo evaluate subject improvement of xerostomia symptoms, to evaluate the increase in parotid gland salivary output after treatment with AAV2hAQP1, to evaluate additional efficacy outcomes."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Male or female subjects ≥18 years of age.\n2. History of radiation therapy for head and neck cancer.\n3. 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.\n4. 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.\n5. 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:\n\n * Condoms with spermicide. Acceptable methods of contraception for female subjects include the following:\n * Intrauterine device for at least 12 weeks prior to Screening.\n * Hormonal contraception (oral, implant, injection, ring, or patch) for at least 12 weeks prior to Screening.\n * Diaphragm used in combination with spermicide.\n\nExclusion Criteria:\n\n1. Pregnant or lactating women or women planning to become pregnant.\n2. Any experimental therapy within 3 months before Day 1.\n3. Active infection that requires the use of intravenous antibiotics and does not resolve at least 1 week before Day 1.\n4. Uncontrolled ischemic heart disease (i.e., unstable angina, evidence of active ischemic heart disease on electrocardiogram \\[ECG\\]).\n5. History of systemic autoimmune diseases affecting the salivary glands.\n6. Use of systemic immunosuppressive medications (i.e., corticosteroids).\n\n o Note: Topical, inhaled, or intranasal corticosteroids are allowed.\n7. 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.\n8. Active infections including, Epstein-Barr virus (EBV), cytomegalovirus (CMV), hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV) infection.\n9. 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.\n10. 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.\n11. Estimated glomerular filtration rate \\<60 mL/min/1.73 m2 using the Modification of Diet in Renal Disease equation.\n12. Active use of tobacco products as determined by self-reporting.\n13. Allergy to iodine or shellfish, and thus unable to have sialographic evaluations.\n14. Allergy or hypersensitivity to glycopyrrolate.'}, 'identificationModule': {'nctId': 'NCT04043104', 'briefTitle': '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', 'organization': {'class': 'INDUSTRY', 'fullName': 'MeiraGTx UK II Ltd'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'MGT016'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1 x 10^11 vg/gland (single gland)', 'interventionNames': ['Drug: AAV2hAQP1: 1 x 10^11 vg/gland (single gland)']}, {'type': 'EXPERIMENTAL', 'label': '3 x 10^10 vg/gland (both glands)', 'interventionNames': ['Drug: AAV2hAQP1: 3 x 10^10 vg/gland (both glands)']}, {'type': 'EXPERIMENTAL', 'label': '3 x 10^11 vg/gland (single gland)', 'interventionNames': ['Drug: AAV2hAQP1: 3 x 10^11 vg/gland (single gland)']}, {'type': 'EXPERIMENTAL', 'label': '1 x 10^11 vg/gland (both glands)', 'interventionNames': ['Drug: AAV2hAQP1: 1 x 10^11 vg/gland (both glands)']}, {'type': 'EXPERIMENTAL', 'label': '1 x 10^12 vg/gland (single gland)', 'interventionNames': ['Drug: AAV2hAQP1: 1 x 10^12 vg/gland (single gland)']}, {'type': 'EXPERIMENTAL', 'label': '3 x 10^11 vg/gland (both glands)', 'interventionNames': ['Drug: AAV2hAQP1: 3 x 10^11 vg/gland (both glands)']}, {'type': 'EXPERIMENTAL', 'label': '3 x 10^12 vg/gland (single gland)', 'interventionNames': ['Drug: AAV2hAQP1: 3 x 10^12 vg/gland (single gland)']}, {'type': 'EXPERIMENTAL', 'label': '1 x 10^12 vg/gland (both glands)', 'interventionNames': ['Drug: AAV2hAQP1: 1 x 10^12 vg/gland (both glands)']}], 'interventions': [{'name': 'AAV2hAQP1: 1 x 10^11 vg/gland (single gland)', 'type': 'DRUG', 'description': "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", 'armGroupLabels': ['1 x 10^11 vg/gland (single gland)']}, {'name': 'AAV2hAQP1: 3 x 10^10 vg/gland (both glands)', 'type': 'DRUG', 'description': "Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10\\^10 vg/gland", 'armGroupLabels': ['3 x 10^10 vg/gland (both glands)']}, {'name': 'AAV2hAQP1: 3 x 10^11 vg/gland (single gland)', 'type': 'DRUG', 'description': "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", 'armGroupLabels': ['3 x 10^11 vg/gland (single gland)']}, {'name': 'AAV2hAQP1: 1 x 10^11 vg/gland (both glands)', 'type': 'DRUG', 'description': "intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10\\^11 vg/gland", 'armGroupLabels': ['1 x 10^11 vg/gland (both glands)']}, {'name': 'AAV2hAQP1: 1 x 10^12 vg/gland (single gland)', 'type': 'DRUG', 'description': "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", 'armGroupLabels': ['1 x 10^12 vg/gland (single gland)']}, {'name': 'AAV2hAQP1: 3 x 10^11 vg/gland (both glands)', 'type': 'DRUG', 'description': "Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 3 x 10\\^11 vg/gland", 'armGroupLabels': ['3 x 10^11 vg/gland (both glands)']}, {'name': 'AAV2hAQP1: 3 x 10^12 vg/gland (single gland)', 'type': 'DRUG', 'description': "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", 'armGroupLabels': ['3 x 10^12 vg/gland (single gland)']}, {'name': 'AAV2hAQP1: 1 x 10^12 vg/gland (both glands)', 'type': 'DRUG', 'description': "Intra-parotid administration of AAV2hAQP1 via Stensen's duct to both parotid glands at a dose level of 1 x 10\\^12 vg/gland", 'armGroupLabels': ['1 x 10^12 vg/gland (both glands)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Leland Stanford Junior University', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Louisville', 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '02184', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10065', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Memorial Sloan Kettering Cancer Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '28209', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Atrium Health', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'city': 'Greater Sudbury', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Health Sciences North - Northeast Cancer Center', 'geoPoint': {'lat': 46.49, 'lon': -80.99001}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'MeiraGTx UK II Ltd', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}