Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants
Start Date
11/21/2025
Completion Date
1/15/2032
Summary
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.
Detailed Description
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.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
NT-501/revakinagene taroretcel-lwey
Conditions
Locations
Scripps Health
La Jolla, California 92037
United States
Jules Stein Eye Institute/UCLA
Los Angeles, California 90085
United States
Stanford University School of Medicine
Palo Alto, California 94303
United States
Bay Area Retina Associates
Walnut Creek, California 94598
United States
Colorado Retina
Lakewood, Colorado 80228
United States
Bascom Palmer
Miami, Florida 33136
United States
Emory Eye Center
Atlanta, Georgia 30322
United States
Northwestern University
Chicago, Illinois 60611
United States
National Eye Institute
Bethesda, Maryland 20892
United States
Cumberland Valley Retina Consultants
Frederick, Maryland 21702
United States
Massachusetts Eye and Ear Infirmary, Retina Service
Boston, Massachusetts 02114
United States
Foundation for Vision Research
Grand Rapids, Michigan 49525
United States
Retina Consultants of Minnesota
Minneapolis, Minnesota 55435
United States
Envision Ocular, LLC
Bloomfield, New Jersey 07003
United States
Cincinnati Eye Institute
Cincinnati, Ohio 45242
United States
Retina Associates of Cleveland, Inc.
Cleveland, Ohio 44126
United States
Retina Northwest, P.C.
Portland, Oregon 97221
United States
Scheie Eye Institute
Philadelphia, Pennsylvania 19104
United States
Southeastern Retina Associates, P.C.
Knoxville, Tennessee 37922
United States
Texas Retina Associates
Dallas, Texas 75231
United States
Retina Center of Texas
Dallas, Texas 75251
United States
Retina Consultants of Texas
Houston, Texas 77030
United States
University of Utah John A. Moran Center
Salt Lake City, Utah 84132
United States
Cerulea
East Melbourne,
Australia
Lion's Eye Institute
Perth,
Australia
Sydney Eye Hospital
Sydney,
Australia