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. 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). * 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). * 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. Participants who received NT-501 in both eyes (ie, participated in study NTMT-02B) will be enrolled in either Arm 1 or Arm 2 based on the study in which the first NT-501 was implanted (ie, participants who received the first implant in a Phase 3 study will be enrolled in Arm 1 and those who received the first implant in the Phase 1 study or Phase 2 study will be enrolled in Arm 2). Only Arm 3 is an interventional study where participants will receive an NT-501 implant. A participant can receive NT-501 in either eye that meets eligibility criteria according to investigator assessment, and in accordance with participant choice.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
285
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
Jules Stein Eye Institute/UCLA
Los Angeles, California, United States
Stanford University School of Medicine
Palo Alto, California, United States
Bay Area Retina Associates
Walnut Creek, California, United States
Emory Eye Center
Atlanta, Georgia, United States
Incidence 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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Northwestern University
Chicago, Illinois, United States
Elman Retina Group, PA
Baltimore, Maryland, United States
National Eye Institute
Bethesda, Maryland, United States
Cumberland Valley Retina Consultants
Frederick, Maryland, United States
Massachusetts Eye and Ear Infirmary, Retina Service
Boston, Massachusetts, United States
...and 23 more locations