NT-501 Extension Study in MacTel
Research type
Research Study
Full title
A Phase 4, Multicenter, Open-label Study to Evaluate Long-term Safety and Efficacy of Revakinagene Taroretcel-Lwey (NT-501) Previously Implanted During a Phase 1, Phase 2, or Phase 3 Clinical Study and to Evaluate NT-501 Implanted in Participants who Underwent the Sham Procedure in a Phase 3 Clinical Study of Macular Telangiectasia Type 2 (MacTel)
IRAS ID
1012848
Contact name
Tea Borojevic
Contact email
Sponsor organisation
Neurotech Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
This study will look at the long-term safety and effectiveness of NT-501 (Revakinagene Taroretcel-Lwey), a small implant placed inside the eye to treat Macular Telangiectasia Type 2 (MacTel), a rare progressive eye disease that affects central vision in both eyes.
NT-501 contains special cells that release a protein (CNTF) believed to protect the retina and slow vision loss. Previous clinical studies (Phases 1–3) showed that NT-501 may help slow the disease and preserve vision. NT-501 is now approved for use in the U.S.
This Phase 4 study will continue monitoring participants who previously received NT-501 in earlier studies and will also offer the implant to those who had a “sham” (placebo) procedure in a prior Phase 3 trial. The study will take place at around 47 sites in the U.S., Australia, France, Germany, and the UK. Two sites in the UK will participate. Up to 285 adult participants will be enrolled across all participating countries.
Participants are divided into three groups:
Group 1: People who had NT-501 during the Phase 3 study
Group 2: People who had NT-501 during earlier Phase 1 or 2 studies
Group 3: People who had sham surgery in Phase 3 and are now offered the real NT-501 implant
Groups 1 and 2 will not receive new treatment but will attend regular follow-up visits. Group 3 will have NT-501 surgically implanted at the start of the study and will be followed for at least two years.
At visits, participants will undergo eye tests such as vision checks, retinal scans, reading tests, and questionnaires about how vision affects daily life. The study will also monitor for any side effects or health changes.
The goal is to better understand the long-term benefits and safety of NT-501 and provide access to treatment for those who had the sham procedure in earlier studies.REC name
South Central - Oxford A Research Ethics Committee
REC reference
26/SC/0103
Date of REC Opinion
22 May 2026
REC opinion
Further Information Unfavourable Opinion