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Long-term safety and effects of the NT-501 eye implant for Macular Telangiectasia Type 2, including participants who previously had a sham procedure

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)

Phase 4 Interventional Neurotech Pharmaceuticals · NCT06971939

This study will try the NT-501 intraocular implant in people with MacTel Type 2 who previously received the implant or a sham procedure to see if it stays safe and helps preserve vision over the long term.

Quick facts

PhasePhase 4
Study typeInterventional
Enrollment285 (estimated)
Ages18 Years and up
SexAll
SponsorNeurotech Pharmaceuticals Industry-sponsored
Drugs / interventionschemotherapy
Locations33 sites (La Jolla, California and 32 other locations)
Trial IDNCT06971939 on ClinicalTrials.gov

What this trial studies

This multicenter Phase 4 extension follows participants who took part in earlier NT-501 MacTel studies to monitor long-term safety and clinical outcomes. The protocol has three arms: two observational arms that follow participants previously implanted with NT-501 in Phase 1/2 or Phase 3 studies, and one interventional crossover arm in which prior sham participants may receive the implant. Participants undergo regular ophthalmic examinations, retinal imaging, and visual function tests to track structural and functional changes, along with standard safety monitoring. The trial focuses on sustained device tolerability, adverse events, and measures of retinal health and vision over extended follow-up.

Who should consider this trial

Good fit: Adults who previously completed a MacTel NT-501 clinical study and either received the NT-501 implant (for observational arms) or previously underwent a sham procedure and elect to receive the implant (crossover arm) are eligible.

Not a fit: People who never participated in prior NT-501 MacTel studies or whose eyes do not meet the investigator's implant eligibility are unlikely to qualify and may not benefit from this extension.

Why it matters

Potential benefit: If successful, the implant could slow disease progression and help preserve central retinal structure and visual function in people with MacTel Type 2.

How similar studies have performed: Earlier Phase 1–3 NT-501 studies demonstrated acceptable safety and some structural retinal changes, but clear, durable visual benefit remains limited and is being followed further in this extension.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria

To be eligible to participate in this study, an individual participant must meet all the following criteria:

1. Male or female adult participants who previously completed an NT-501 MacTel clinical study and:

   1. Arm 1: received NT-501 intraocular implant in either Phase 3 study (NTMT-03-A or NTMT-03-B)
   2. Arm 2: received NT-501 intraocular implant in the Phase 1 study (NTMT-01), Phase 2 study (NTMT-02), or in the substudy of the Phase 1 and Phase 2 extension study (NTMT-01/02E)

      Note: 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).
   3. Arm 3: underwent the sham procedure in either Phase 3 study (NTMT-03-A or NTMT-03-B)
2. Participants must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality retinal imaging.
3. Participant or their legally authorized representative must be able to provide written informed consent to participate in the study, in accordance with the International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, and local regulations, before initiating any clinical study related procedures.
4. Arm 3: female participants of childbearing potential must agree to use highly effective contraception. Highly effective forms of contraception (generally a first-year typical use pregnancy risk of 1% or less) include the following: contraceptive implants; an intrauterine device; permanent contraceptive procedures (vasectomy, bilateral salpingectomy, bilateral tubal occlusion, or partial salpingectomy); abstinence. A combination of male condom with either cap, diaphragm, or sponge with spermicide (ie, double barrier methods) is also considered an acceptable, but not highly effective, method of birth control.

Note: Participants enrolled at investigator sites in France or Germany must agree to use 2 forms of contraception listed above during study participation.

Exclusion Criteria

To participate in this study, the potential participant must not meet any of the following criteria. (Note that the ocular exclusion criteria are relevant to the study eye\[s\], unless indicated for both eyes):

Exclusion Criteria Applicable to Arms 1, 2, and 3:

1. Participant is medically unable to comply with clinical study procedures or visits.
2. Participant has, in the opinion of the investigator, any physical or mental condition that would increase the risk of participation in the study or may interfere with the study procedures, evaluations, and outcome assessments.

   Exclusion Criteria Applicable to Arm 3 Only:
3. Participant has significant corneal or media opacities in the study eye to be implanted.
4. Participant has evidence of pathologic myopia in the study eye to be implanted.
5. Participant has any of the following lens opacities in the study eye to be implanted: 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. Note: the participant can be reconsidered for enrollment 90 days after undergoing cataract surgery.
6. Participant has undergone lens removal in the study eye to be implanted in the previous 3 months or YAG laser within 4 weeks of the screening/baseline visit.
7. Participant has evidence of ocular disease in the study eye to be implanted other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures, or outcome of the study (eg. glaucoma, severe nonproliferative or proliferative diabetic retinopathy, uveitis).
8. Received intravitreal steroid therapy in the study eye to be implanted within the last year.
9. Received intravitreal anti-vascular endothelial growth factor therapy in the study eye to be implanted within the last 6 months.
10. Evidence of active exudative intraretinal neovascularization or subretinal neovascularization in the study eye to be implanted, as evidenced by hemorrhage, hard exudate, subretinal fluid, or intraretinal fluid.
11. Evidence of central serous chorioretinopathy in the study eye to be implanted.
12. Participant underwent vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty in the study eye to be implanted.
13. A history of ocular herpes virus in the study eye to be implanted.
14. Participant is on chemotherapy.
15. Participant is pregnant or breastfeeding.
16. Participation in any other clinical study of an intervention (drug or device) within 6 months prior to the screening/baseline visit.
17. A history of malignancy that would compromise survival during the clinical study.

Where this trial is running

La Jolla, California and 32 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Macular Telangiectasia Type 2Ciliary Neurotrophic Factor
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.