Ignite Creation Date:
2025-12-24 @ 1:01 PM
Ignite Modification Date:
2025-12-29 @ 12:45 PM
Study NCT ID:
NCT05930561
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-22
First Post:
2023-06-26
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
4D-150 in Patients With Diabetic Macular Edema
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003930', 'term': 'Diabetic Retinopathy'}], 'ancestors': [{'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D003925', 'term': 'Diabetic Angiopathies'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D048909', 'term': 'Diabetes Complications'}, {'id': 'D003920', 'term': 'Diabetes Mellitus'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C533178', 'term': 'aflibercept'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'No masking in Dose Confirmation (Part 1). In Dose Expansion (Part 2), participants and outcomes assessors will be masked to the treatment arm for the duration of the trial.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'In Dose Confirmation (Part 1), subjects (n=12-18) will be sequentially assigned to one of two open-label dose levels of 4D-150 (n=6-9 per dose level). In Dose Expansion (Part 2), subjects (n=54) will be randomized to receive one of two dose levels of 4D-150 (n=18 per dose level) based on results from Dose Confirmation, or aflibercept control (n=18). Subjects will be masked to treatment arm in Dose Expansion (Part 2).'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 72}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-08-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2029-02-28', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-17', 'studyFirstSubmitDate': '2023-06-26', 'studyFirstSubmitQcDate': '2023-06-26', 'lastUpdatePostDateStruct': {'date': '2025-09-22', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-07-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-02-28', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Incidence and severity of treatment-emergent adverse events and serious adverse events, including clinically significant changes in safety parameters', 'timeFrame': '104 weeks'}, {'measure': 'Development of anti-drug antibodies to capsid protein (4D-R100) and the transgene product (aflibercept) and change in antibody titers in subjects receiving 4D-150', 'timeFrame': '104 weeks'}, {'measure': 'Change in levels of aflibercept protein in aqueous humor and serum overtime', 'timeFrame': '104 weeks'}], 'primaryOutcomes': [{'measure': 'Incidence and severity of TEAEs and SAEs, including clinically significant changes in safety parameters (Part 1)', 'timeFrame': '60 months'}, {'measure': 'Annualized number of aflibercept injections in the study eye (Part 2)', 'timeFrame': '52 weeks'}], 'secondaryOutcomes': [{'measure': 'Mean cumulative number of aflibercept injections over time', 'timeFrame': '52 weeks'}, {'measure': 'Change from baseline in BCVA as assessed using the ETDRS Visual Acuity Chart at Weeks 52 and 104', 'timeFrame': '104 weeks'}, {'measure': 'Change from baseline in CST measured by spectral domain optical coherence tomography (SD-OCT) at Weeks 104', 'timeFrame': '104 weeks'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Retinal gene therapy', 'Intravitreal gene therapy', 'DME', 'SPECTRA'], 'conditions': ['Diabetic Macular Edema', 'Diabetic Retinopathy']}, 'descriptionModule': {'briefSummary': 'Phase 2 randomized, active-controlled, double-masked, dose-ranging trial in adults with Diabetic Macular Edema (DME).', 'detailedDescription': 'This Phase 2 trial is a prospective, multicenter, randomized, active-controlled, double-masked dose-ranging trial to evaluate the safety and efficacy of 4D-150 in adults with DME. The trial will be conducted in two parts: Dose Confirmation and Dose Expansion.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* ≥18 years of age\n* Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea; diagnosis of DME must be within 2 years of Screening\n* CST ≥ 350 μm (Spectralis SD-OCT) in the study eye at Screening, confirmed by the independent reading center\n* Demonstrate clinical response to on-study aflibercept injection in the study eye.\n* Decreased visual acuity attributable primarily to DME\n* BCVA in the study eye between 25 and 83 ETDRS letters, inclusive (\\~20/320 and 20/25, respectively) at Screening\n* Study eye amenable to IVT injection\n* Sufficient clear ocular media, pupil dilation and fixation in the study eye to permit adequate imaging; ability to perform tests of visual and retinal function and structure; and ability to comply with other protocol-specified procedures\n* Provide written informed consent\n\nExclusion Criteria:\n\n* Macular edema in the study eye considered to be secondary to a cause other than DME\n* Systemic anti-VEGF treatment (e.g. sunitinib, bevacizumab, pazopanib) within 6 months, or anticipated need for systemic anti-VEGF therapy during study participation\n* Systemic corticosteroids (oral, intravenous, intramuscular, intra-articular) or other immunosuppressive medications within 3 months\n* Received an investigational drug, agent, device, or therapy (ocular or non-ocular) in the 3 months (or at least 5 half-lives, whichever is longer) prior to 4D-150 administration (Day 1)\n* Prior gene therapy (ocular or non-ocular) and/or ocular stem cell therapy in either eye\n* Any concurrent ocular condition in the study eye that is likely to require surgical intervention (e.g. cataract surgery) during the 2 year (104 week) study duration\n\nNote: Other inclusion/exclusion criteria apply.'}, 'identificationModule': {'nctId': 'NCT05930561', 'briefTitle': '4D-150 in Patients With Diabetic Macular Edema', 'organization': {'class': 'INDUSTRY', 'fullName': '4D Molecular Therapeutics'}, 'officialTitle': 'A Phase 2 Randomized, Active-Controlled, Double-masked Trial of Intravitreal 4D-150 Gene Therapy in Adults With Diabetic Macular Edema (SPECTRA)', 'orgStudyIdInfo': {'id': '4D-150-C002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '4D-150 Part 1 Dose Confirmation Dose Level 1', 'description': '4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.', 'interventionNames': ['Biological: 4D-150 IVT']}, {'type': 'EXPERIMENTAL', 'label': '4D-150 Part 1 Dose Confirmation Dose Level 2', 'description': '4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.', 'interventionNames': ['Biological: 4D-150 IVT']}, {'type': 'EXPERIMENTAL', 'label': '4D-150 Part 2 Dose Expansion Dose Level 1', 'description': '4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.', 'interventionNames': ['Biological: 4D-150 IVT']}, {'type': 'EXPERIMENTAL', 'label': '4D-150 Part 2 Dose Expansion Dose Level 2', 'description': '4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.', 'interventionNames': ['Biological: 4D-150 IVT']}, {'type': 'ACTIVE_COMPARATOR', 'label': '4D-150 Part 2 Dose Expansion Control', 'description': 'Aflibercept at a fixed regimen will be administered.', 'interventionNames': ['Biological: Aflibercept IVT']}], 'interventions': [{'name': '4D-150 IVT', 'type': 'BIOLOGICAL', 'description': '4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept.', 'armGroupLabels': ['4D-150 Part 1 Dose Confirmation Dose Level 1', '4D-150 Part 1 Dose Confirmation Dose Level 2', '4D-150 Part 2 Dose Expansion Dose Level 1', '4D-150 Part 2 Dose Expansion Dose Level 2']}, {'name': 'Aflibercept IVT', 'type': 'BIOLOGICAL', 'otherNames': ['Eylea'], 'description': 'Commercially available Active Comparator', 'armGroupLabels': ['4D-150 Part 2 Dose Expansion Control']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21740', 'city': 'Hagerstown', 'state': 'Maryland', 'country': 'United States', 'facility': 'Cumberland Valley Retina Consultants', 'geoPoint': {'lat': 39.64176, 'lon': -77.71999}}, {'zip': '97401', 'city': 'Eugene', 'state': 'Oregon', 'country': 'United States', 'facility': 'Verum Research, LLC', 'geoPoint': {'lat': 44.05207, 'lon': -123.08675}}, {'zip': '16507', 'city': 'Erie', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Erie Retina Research', 'geoPoint': {'lat': 42.12922, 'lon': -80.08506}}, {'zip': '78750', 'city': 'Austin', 'state': 'Texas', 'country': 'United States', 'facility': 'Austin Clinical Research', 'geoPoint': {'lat': 30.26715, 'lon': -97.74306}}, {'zip': '24502', 'city': 'Lynchburg', 'state': 'Virginia', 'country': 'United States', 'facility': 'Piedmont Eye Center', 'geoPoint': {'lat': 37.41375, 'lon': -79.14225}}, {'zip': 'X00612', 'city': 'Arecibo', 'country': 'Puerto Rico', 'facility': 'Emanuelli Research and Development Center', 'geoPoint': {'lat': 18.47245, 'lon': -66.71573}}], 'overallOfficials': [{'name': 'Julie Clark, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': '4D Molecular Therapeutics'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': '4D Molecular Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}