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| Name | Class |
|---|---|
| The Lowy Medical Research Institute Limited | OTHER |
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This is a multi-center, open-label, 6-month study to evaluate the safety of bilateral CNTF implants in participants with Macular Telangiectasia Type 2.
This was a multicenter, open-label study designed to evaluate the safety of bilateral NT-501 in subjects with MacTel. All subjects who received NT-501 in a single eye prior to or in the Phase 1/2 extension study (NTMT-01/02E) or in 1 of the 2 Phase 3 studies (NTMT-03-A or NTMT-03-B), and met all other eligibility criteria, qualified for participation in the current study; enrolled subjects underwent intraocular implantation of NT-501 in the fellow eye and were followed for 6 months. Note that, throughout this report, all references to "study eye" mean the eye that received NT-501 in the current study (ie, not the eye that was treated in the previous study).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NT-501 in Fellow Eye | Experimental | NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NT-501 CNTF implant | Combination Product | Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye |
|
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events | Assess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501 | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Serum Levels of CNTF and Immunogenicity for NT-501 | Incidence of positive serum levels of CNTF and immunogenicity for NT-501 with quantification of antibodies (Ab) to CNTF, neutralizing antibodies (NAb) to CNTF, Ab to NTC-201-6A cells, and Ab to mouse dihydrofolate reductase (mDHFR) at any time. | 6 months |
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General Inclusion Criteria
Ocular Inclusion Criteria
General Exclusion Criteria
Females of childbearing potential (those who are not surgically sterilized or post- menopausal, i.e., absence of menstruation for 12 months or longer) may not participate in the study if any of the following conditions exists:
Participant is too ill to likely complete the entire study, based on the investigator's assessment
Participant, in the opinion of the investigator, is not suitable for study participation
Participant with any screening laboratory finding (serum chemistry, hematology, urinalysis) that in the opinion of the investigator is not suitable for study participation
Participant has a history or current evidence of severe hypersensitivity to the NT-501 implant
Participant has a history or current evidence of a medical condition (systemic or ophthalmic disease, metabolic dysfunction, physical examination finding or clinical laboratory finding) that may in the opinion of the investigator preclude the safe administration of the NT-501 implant or adherence to the scheduled study visits, safe participation in the study or affect the results of the study (e.g., unstable or progressive cardiovascular, cerebral vascular, pulmonary, Parkinson's, liver or renal disease; depression, cancer, or dementia
Ocular Exclusion Criteria
Participant has a history or evidence of the following surgeries/procedures in the study eye, as assessed at Visit 1, including:
Participant has uncontrolled glaucoma; or ocular hypertension, i.e., IOP ≥ 25 mmHg in the study eye
Participant has evidence of intraretinal or subretinal neovascularization or central serous chorioretinopathy in the study eye
Participant has evidence of ocular disorder(s) in the study eye of a severity that could confound the interpretation of study results, compromise visual acuity or require medical or surgical intervention during the study period, including retinal vascular occlusion, severe nonproliferative or proliferative diabetic retinopathy, retinal detachment, macular hole, geographic atrophy, intraretinal or subretinal neovascularization, central serous chorio-retinopathy, pathological myopia
Participant has a vitreous hemorrhage in the study eye at Visit 1 (Screening)
Participant has a spherical equivalent of the refractive error in the study eye demonstrating more than 8 diopters of myopia
Participant has a history or evidence of penetrating ocular trauma in the study eye
Participant has an anticipated need for cataract extraction in the study eye within 6 months of Visit 1 (Screening) such as cortical opacity > standard 3, posterior subcapsular opacity > standard 2, or a nuclear opacity > standard 3 as measured on the Age-Related Eye Disease Study (AREDS) clinical lens grading system
Participant has uveitis, history of uveitis in either eye or history of ocular herpes virus in either eye
Participant has undergone major surgery within the last 6 months (systemic or ocular in either eye) or who are likely to require major surgery within 6 months of Visit 1 (Screening)
Participant has periocular or ocular/intraocular infection or inflammation in either eye (such as infectious conjunctivitis, keratitis, scleritis, endophthalmitis) within 3 months prior to Visit 1 (Screening)
Participant has ocular hypotension in either eye (<6 mmHg) that in the opinion of the Investigator would interfere with the NT-501 implant insertion
Participant has a history of scleritis, scleral thinning, periocular, ocular, or intraocular infection or inflammation, cicatrizing conjunctival diseases any other ocular conditions in the study eye that could interfere with or complicate the surgery associated with NT-501implant insertion
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| Name | Affiliation | Role |
|---|---|---|
| Thomas Aaberg, Jr, MD | Neurotech Pharmaceuticals, LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Jules Stein Eye Institute | Los Angeles | California | 90095 | United States | ||
| National Eye Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30292541 | Result | Chew EY, Clemons TE, Jaffe GJ, Johnson CA, Farsiu S, Lad EM, Guymer R, Rosenfeld P, Hubschman JP, Constable I, Wiley H, Singerman LJ, Gillies M, Comer G, Blodi B, Eliott D, Yan J, Bird A, Friedlander M; Macular Telangiectasia Type 2-Phase 2 CNTF Research Group. Effect of Ciliary Neurotrophic Factor on Retinal Neurodegeneration in Patients with Macular Telangiectasia Type 2: A Randomized Clinical Trial. Ophthalmology. 2019 Apr;126(4):540-549. doi: 10.1016/j.ophtha.2018.09.041. Epub 2018 Oct 4. |
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Eligible subjects must have had a positive diagnosis of MacTel and previously received NT-501 in one eye prior to or in the Phase 1/2 extension study (NTMT-01/02E) or in 1 of the 2 Phase 3 studies (NTMT-03-A or NTMT-03-B). Subjects who participated in the Phase 3 study must have completed the Month 24 visit in that study and subjects enrolled in either the Phase 1/2 extension study or the Phase 3 study must have discontinued prior to enrollment in the current study.
Of the 33 enrolled subjects, 32 (97.0%) received NT-501; the exception was a subject whose surgery was canceled due to an event of arterial fibrillation that occurred prior to surgery and resulted in subject discontinuation. No subject had NT-501 removed from either eye during the study, and all 32 subjects who received NT-501 in this study completed the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | NT-501 in Fellow Eye | NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study. NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 21, 2022 | Sep 20, 2024 |
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| Washington D.C. |
| District of Columbia |
| 20892 |
| United States |
| Bascom Palmer | Miami | Florida | 33136 | United States |
| Massachusetts Eye and Ear Infirmary, Retina Service | Boston | Massachusetts | 02114 | United States |
| University of Michigan, Kellogg Eye Center | Ann Arbor | Michigan | 48105 | United States |
| Retina Associates of Cleveland, Inc. | Beachwood | Ohio | 44122 | United States |
| Retina Consultants of Texas | Houston | Texas | 77030 | United States |
| Save Sight Institute | Sydney | New South Wales | Australia |
| Centre for Eye Research Australia | East Melbourne | Victoria | 3002 | Australia |
| Lions Eye Institute | Nedlands | Western Australia | 6009 | Australia |
| NT-501 Implanted in Study Eye |
|
| COMPLETED |
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| NOT COMPLETED |
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|
Baseline demographics and characteristics are based on total number of participants who completed NT-501 implant in study eye
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| ID | Title | Description |
|---|---|---|
| BG000 | NT-501 in Fellow Eye | NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study. NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Age, Customized | Age Groups of 50 to < 60, 60 to < 70, and greater than or equal to 70 | Count of Participants | Participants |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| BCVA (letters) for Study Eye | Baseline letter score using the standard Treatment Diabetic Retinopathy Study (ETDRS) chart | Mean | Standard Deviation | letters correctly read |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Frequency of Ocular and/or Non-ocular Treatment Emergent Adverse Events | Assess the incidence and severity of adverse events (AEs) following bilateral ocular implantation of NT-501 | Posted | Count of Participants | Participants | 6 months |
|
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Serum Levels of CNTF and Immunogenicity for NT-501 | Incidence of positive serum levels of CNTF and immunogenicity for NT-501 with quantification of antibodies (Ab) to CNTF, neutralizing antibodies (NAb) to CNTF, Ab to NTC-201-6A cells, and Ab to mouse dihydrofolate reductase (mDHFR) at any time. | A neutralizing antibody test to NT-501-produced CNTF was performed to verify or refute CNTF antibody detected in one participant. The neutralizing antibody test refuted that the antibody detected was to NT-501 CNTF. | Posted | Count of Participants | Participants | 6 months |
|
|
Through 6 months post implant
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | NT-501 in Fellow Eye | NT-501 was implanted on Day 0 in the fellow eye of subjects who received a single NT-501 in a previous study; subjects were followed for 6 months post-implantation of NT-501 in this study. NT-501 CNTF implant: Single implantation of CNTF-secreting NT-501 device into fellow (untreated) eye | 0 | 32 | 2 | 32 | 26 | 32 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Device Extrusion | Surgical and medical procedures | MedDRA 25.0 | Systematic Assessment |
| |
| Feces discolored | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eye pain | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Ocular discomfort | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Dry eye | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Anterior chamber cell | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Anterior chamber flare | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Cataract | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Cataract subcapsular | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Conjunctival hyperaemia | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Conjunctival oedema | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Conjunctivitis allergic | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Corneal pigmentation | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Delayed dark adaptation | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Epiretinal membrane | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Eye pruritus | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Foreign body sensation in eyes | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Halo vision | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Iris transillumination defect | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Iritis | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Photophobia | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Photopsia | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Punctate keratitis | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Retinal haemorrhage | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitreous detachment | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitreous haemorrhage | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Allergy to sutures | Immune system disorders | MedDRA 25.0 | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Corneal abrasion | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
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| Intraocular pressure decreased | Investigations | MedDRA 25.0 | Systematic Assessment |
| |
| Pyogenic granuloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 25.0 | Systematic Assessment |
| |
| Device extrusion | Product Issues | MedDRA 25.0 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| CMO | Neurotech Pharmaceuticals, LLC | 401-333-3880 | neurotech@neurotechusa.com |
| Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 14, 2022 | Mar 7, 2025 | SAP_002.pdf |
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| > or = to 70 years |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Title | Measurements |
|---|---|
|
| At least 1 nonocular TEAE |
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| At least 1 treatment related ocular TEAE |
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| At least 1 treatment related nonocular TEAE |
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| At least 1 SAE |
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| NT-501 removal from either eye |
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