Ignite Creation Date:
2025-12-25 @ 3:21 AM
Ignite Modification Date:
2025-12-26 @ 2:00 AM
Study NCT ID:
NCT01064505
Status:
COMPLETED
Last Update Posted:
2013-05-13
First Post:
2010-02-04
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009896', 'term': 'Optic Atrophy'}, {'id': 'D018917', 'term': 'Optic Neuropathy, Ischemic'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D009902', 'term': 'Optic Neuritis'}, {'id': 'D029242', 'term': 'Optic Atrophy, Hereditary, Leber'}], 'ancestors': [{'id': 'D009901', 'term': 'Optic Nerve Diseases'}, {'id': 'D003389', 'term': 'Cranial Nerve Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D015418', 'term': 'Optic Atrophies, Hereditary'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D028361', 'term': 'Mitochondrial Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D034741', 'term': 'RNA, Small Interfering'}], 'ancestors': [{'id': 'D016372', 'term': 'RNA, Antisense'}, {'id': 'D016375', 'term': 'Antisense Elements (Genetics)'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D012313', 'term': 'RNA'}, {'id': 'D009696', 'term': 'Nucleic Acids'}, {'id': 'D058727', 'term': 'RNA, Small Untranslated'}, {'id': 'D022661', 'term': 'RNA, Untranslated'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 48}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-05', 'completionDateStruct': {'date': '2013-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-05-09', 'studyFirstSubmitDate': '2010-02-04', 'studyFirstSubmitQcDate': '2010-02-04', 'lastUpdatePostDateStruct': {'date': '2013-05-13', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2010-02-08', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To determine the safety, tolerability and the dose-limiting toxicities (DLTs) of QPI 1007 when administered as a single intravitreal (IVT) injection.', 'timeFrame': '12 Months Post-injection'}, {'measure': 'To assess the pharmacokinetics (PK) of QPI-1007 when administered as a single IVT injection.', 'timeFrame': '12 Months Post-Injection'}], 'secondaryOutcomes': [{'measure': 'To determine the presence of and to describe any anatomical changes in the optic nerve head and retina observed following the administration of a single IVT injection of QPI 1007.', 'timeFrame': '12 Months Post-Injection'}, {'measure': 'To assess any changes in visual acuity and visual field observed following the administration of a single IVT injection of QPI-1007.', 'timeFrame': '12 Months Post-Injection'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Ocular Neuroprotection', 'ocular neuroprotectant', 'NAION', 'NAAION', 'Non-arteritic ischemic optic neuropathy', 'Non-arteritic anterior ischemic optic neuropathy', 'chronic optic nerve atrophy', 'optic nerve atrophy', 'retinal degeneration', 'optic neuritis', 'end stage glaucoma', "Leber's hereditary optic neuropathy"], 'conditions': ['Optic Atrophy', 'Non-arteritic Anterior Ischemic Optic Neuropathy']}, 'descriptionModule': {'briefSummary': 'This is an open-label, dose escalation, safety, tolerability and pharmacokinetic study, where active study drug (QPI-1007) will be given to all patients who participate.\n\nThis study will determine whether QPI-1007 is safe when it is injected into the eye. The study will also reveal if there are any side effects of the drug and how long it takes for the body to clear the drug.', 'detailedDescription': 'Patients will be enrolled according to one of two sets of criteria designated as Stratum I and Stratum II.\n\n1. Stratum I will enroll Optic Nerve Atrophy patients who meet necessary criteria. Stratum I will consist of a maximum of 6 cohorts and each cohort will enroll 3 to 6 evaluable patients. Enrollment in Stratum I is now closed.\n2. Stratum II will enroll patients diagnosed with acute Non Arteritic Anterior Ischemic Optic Neuropathy (NAION) within 28 days of symptom onset who meet necessary criteria. Stratum II will consist of a maximum of 3 cohorts and each cohort will enroll up to 10 evaluable patients. Enrollment in Stratum II is still open.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '50 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Stratum I (Chronic Optic Nerve Atrophy) Inclusion Criteria:\n\n* "Legally blind" in the study eye as the result of an irreversible condition affecting the posterior segment of the eye.\n* Clear ocular media and able to undergo adequate pupil dilation.\n* Visual acuity and visual field in the non-study eye are better than or equal to the study eye\n* At least 21 years old.\n\nKey Stratum I (Chronic Optic Nerve Atrophy) Exclusion Criteria:\n\n* For the study eye only: history of any IVT injection or vitrectomy, vitreous hemorrhage, retinal detachment, or active inflammatory condition (e.g. conjunctivitis).\n* For either eye: history of uveitis.\n\nEnrollment is now closed in Stratum I.\n\nKey Stratum II (Acute NAION) Inclusion Criteria:\n\n* Positive diagnosis of NAION with symptom onset within 28 days prior to planned dosing with QPI-1007.\n* Visual acuity in the study eye is between 20/40 and light perception.\n* Clear ocular media and able to undergo adequate pupil dilation.\n* At least 50 years old.\n\nKey Stratum II (Acute NAION) Exclusion Criteria:\n\n* For the study eye only: Macular disease, retinopathy, or other eye disease limiting visual acuity; prior intraocular surgery (other than Lasik) and cataract surgery within 3 months prior to dosing; glaucoma laser surgery within 1 month prior to dosing; pain on or aggravated by eye movement; history of vitreous hemorrhage; history of retinal detachment; any active inflammatory condition (e.g. conjunctivitis); glaucoma or ocular hypertension; or intraocular pressure \\> 26 mmHg.\n* For either eye: History of optic neuritis; or history of uveitis.\n* Received any treatment for NAION prior to dosing.\n* Any other abnormality which in the opinion of the investigator is suggestive of a disease other than NAION in the study eye only.\n* Clinical evidence of temporal arteritis.\n* History of collagen vascular disease or other inflammatory disease, or history of multiple sclerosis.\n\nEnrollment in Stratum II is still open.'}, 'identificationModule': {'nctId': 'NCT01064505', 'briefTitle': 'Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Quark Pharmaceuticals'}, 'officialTitle': 'A Phase I Open-Label, Dose Escalation Trial of QPI-1007 Delivered by a Single Intravitreal Injection to Patients With Optic Nerve Atrophy (Stratum I) and Acute Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION) (Stratum II)', 'orgStudyIdInfo': {'id': 'QRK.007'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'QPI-1007', 'interventionNames': ['Drug: QPI-1007 at various doses']}], 'interventions': [{'name': 'QPI-1007 at various doses', 'type': 'DRUG', 'otherNames': ['QPI-1007', 'siRNA', 'small interfering RNA', 'short interfering RNA'], 'description': 'Single Intravitreal Injection', 'armGroupLabels': ['QPI-1007']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85014', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Retinal Consultants of Arizona', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90211', 'city': 'Beverly Hills', 'state': 'California', 'country': 'United States', 'facility': 'Retina-Vitreous Associates Medical Group', 'geoPoint': {'lat': 34.07362, 'lon': -118.40036}}, {'zip': '90049', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Jules Stein Eye Institute, University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'Rocky Mountain Multiple Sclerosis Center at Anschutz Medical Campus, University of Colorado', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'Bascom Palmer at the University of Miami', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '34239', 'city': 'Sarasota', 'state': 'Florida', 'country': 'United States', 'facility': 'Sarasota Retina Institute', 'geoPoint': {'lat': 27.33643, 'lon': -82.53065}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'University of Illinois at Chicago, Eye and Ear Infirmary', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '66208', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'Kansas University Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '40536', 'city': 'Lexington', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Kentucky, Department of Ophthalmology', 'geoPoint': {'lat': 37.98869, 'lon': -84.47772}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota, Department of Ophthalmology', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '65212', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'Mason Eye Institute, University of Missouri', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}, {'zip': '10003', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York Eye and Ear Infirmary', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'Flaum Eye Institute, University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '28803', 'city': 'Asheville', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Western Carolina Retinal Associates', 'geoPoint': {'lat': 35.60095, 'lon': -82.55402}}, {'zip': '28210', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Charlotte Eye, Ear, Nose and Throat Associates, PA', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Eye Center, Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cole Eye Institute, Cleveland Clinic Foundation', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania, Department of Ophthalmology', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15212', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Allegheny Ophthalmic and Orbital Associates', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Alkek Eye Center Baylor College of Medicine', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'John A. Moran Eye Center at the University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '23298', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': '84101', 'city': 'Beersheba', 'country': 'Israel', 'facility': 'Soroka University Medical Center, Dept of Ophthalmology', 'geoPoint': {'lat': 31.25181, 'lon': 34.7913}}, {'zip': '33394', 'city': 'Haifa', 'country': 'Israel', 'facility': 'Bnai Zion Medical Center', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'zip': '49100', 'city': 'Petah Tikva', 'country': 'Israel', 'facility': 'Rabin Medical Center, Belinson Campus, Dept of Ophthalmology', 'geoPoint': {'lat': 32.08707, 'lon': 34.88747}}, {'zip': '76100', 'city': 'Rehovot', 'country': 'Israel', 'facility': 'Kaplan Medical Center, Department of Ophthalmology', 'geoPoint': {'lat': 31.89421, 'lon': 34.81199}}, {'zip': '64239', 'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'The Tel-Aviv Sourasky Medical Center, Dept of Ophthalmology', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'zip': '52621', 'city': 'Tel Litwinsky', 'country': 'Israel', 'facility': 'The Chaim Sheba Medical Center, Dept of Ophthalmology', 'geoPoint': {'lat': 32.05096, 'lon': 34.84588}}], 'overallOfficials': [{'name': 'Rabia Ozden, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Quark Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Quark Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}