The purpose of this global, multicenter, open-label, Phase 4 clinical extension study is to evaluate the long-term safety and efficacy of revakinagene taroretcel-lwey (Encelto™; hereinafter referred to as NT-501), in participants with macular telangiectasia type 2 (MacTel) who previously received the intraocular implant in a Phase 1, Phase 2, or Phase 3 clinical study and have retained at least 1 NT-501 at study entry. In addition, this study will evaluate the safety and efficacy of NT501 in participants who previously underwent the sham procedure in a Phase 3 MacTel clinical study and elect to have NT-501 implanted intraocularly in this Phase 4 study.
This extension study consists of 3 arms: * Arm 1 (Phase 3 Treated Participants' Long-term Follow-up): Arm 1 is to assess the long-term safety and efficacy of NT-501 in participants who received NT-501 in a previous MacTel Phase 3 clinical study (NTMT-03-A or NTMT-03-B) and have retained at least 1 NT-501at study entry. * Arm 2 (Phase 1 and Phase 2 Long-term Follow-up): Similar to Arm 1, Arm 2 is to assess the long-term safety of NT-501 in participants who received NT-501 in a previous MacTel Phase 1 or Phase 2 clinical study (NTMT-01, NTMT-02, or NTMT-01/02E) and have retained at least 1 NT-501 at study entry. * Arm 3 (Phase 3 Sham to Implant Crossover): Arm 3 will evaluate the safety and efficacy of NT-501 in participants who previously underwent the sham procedure in a Phase 3 MacTel clinical study (NTMT-03-A or NTMT-03-B) and elect to have NT-501 implanted intraocularly in this extension study. Arms 1 and 2 are noninterventional and observational studies. For participants who previously received a single NT-501 implant, the implanted eye is designated the study eye. For participants who received an NT-501 in both eyes (ie, the first implant received in NTMT-01, NTMT-02, NTMT-01/02E, NTMT-03A, or NTMT-03B, and the second implant received in NTMT-02B), both eyes will be designated as study eyes. Only participants in Arm 3 will receive active intervention during this Phase 4 study. A participant can receive NT-501 in either eye that meets eligibility criteria according to investigator assessment, and in accordance with participant choice. However, if only 1 eye is eligible for implantation based on all inclusion/exclusion criteria, then that eye will be selected for NT-501 implantation. The eye selected for NT-501 implantation will be designated as the study eye. Regardless of study eye designation, both eyes of each participant in all 3 study arms will be evaluated during the study.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
234
NT-501 is surgically implanted into the vitreous cavity to continuously release recombinant human ciliary neurotrophic factor (CNTF).
Scripps Health
La Jolla, California, United States
NOT_YET_RECRUITINGJules Stein Eye Institute/UCLA
Los Angeles, California, United States
NOT_YET_RECRUITINGStanford University School of Medicine
Palo Alto, California, United States
NOT_YET_RECRUITINGBay Area Retina Associates
Walnut Creek, California, United States
RECRUITINGIncidence of ocular and nonocular adverse events (AEs) and serious adverse events (SAEs)
Incidence of ocular and nonocular adverse events (AEs) and serious adverse events (SAEs)
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Percentage of participants with a loss of 15 letters or more in best corrected visual acuity (BCVA) compared to presurgery baseline BCVA
Percentage of participants with a loss of 15 letters or more in best corrected visual acuity (BCVA) compared to presurgery baseline BCVA using the Early Treatment Diabetic Retinopathy Study (ETDRS) distance chart
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Change from presurgery baseline in ellipsoid zone (EZ) area loss
Change from presurgery baseline in ellipsoid zone (EZ) area loss as measured by en face spectral domain optical coherence tomography (SD-OCT) for both eyes
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Change from presurgery baseline in BCVA
Change from presurgery baseline in BCVA using the ETDRS distance chart for both eyes
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Change from presurgery baseline in aggregate retinal sensitivity
Change from presurgery baseline in aggregate retinal sensitivity as measured by microperimetry within the EZ line break area for both eyes
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Change from presurgery baseline in monocular reading speed
Change from presurgery baseline in monocular reading speed for both eyes
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
Change from presurgery baseline in the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25)
Change from presurgery baseline in NEI-VFQ-25 near activities subscale score. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively.
Time frame: Baseline and annually for up to 5 yrs for Arms 1 and 2 and baseline, perioperative, and annually for up to 5 yrs for Arm 3 participants
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Colorado Retina
Lakewood, Colorado, United States
NOT_YET_RECRUITINGBascom Palmer
Miami, Florida, United States
NOT_YET_RECRUITINGEmory Eye Center
Atlanta, Georgia, United States
RECRUITINGNorthwestern University
Chicago, Illinois, United States
RECRUITINGNational Eye Institute
Bethesda, Maryland, United States
NOT_YET_RECRUITINGCumberland Valley Retina Consultants
Frederick, Maryland, United States
NOT_YET_RECRUITING...and 16 more locations