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RecruitingNCT06971939Updated Sep 25, 2026

Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants

A Phase 4 interventional study of NT-501/revakinagene taroretcel-lwey in Macular Telangiectasia Type 2 (MacTel), sponsored by Neurotech Pharmaceuticals. Recruiting at 26 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-25.

Sponsored by Neurotech Pharmaceuticals · Phase 4, Interventional, and Treatment

From the registry’s dates

  • Started Nov 2025; still recruiting 11 months later.
Updated Sep 25, 2026Site recruiting status changedGo to Updates ↓
Phase
Phase 4
Study type
Interventional
Enrollment
234
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study 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 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.

Read the 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.

02

Conditions studied

  • Macular Telangiectasia Type 2 (MacTel)

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Keywords

  • Ciliary Neurotrophic Factor (CNTF)
  • Macular Telangiectasia Type 2 (MacTel)
03

In context

Telangiectasis

69 studies on the registry are indexed under Telangiectasis; 12 are open to participants now.

This study's planned enrollment of 234 is above the median of 30 across 59 interventional studies indexed under Telangiectasis.

Browse Telangiectasis studies →

Lead sponsor

Neurotech Pharmaceuticals is the lead sponsor of 13 studies on the registry; 2 are open to participants now.

Of its 8 completed or terminated interventional studies of FDA-regulated products, 7 (88%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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) and have retained at least 1 NT-501 at study entry.
    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) and have retained at least 1 NT-501 at study entry.

      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. Participant or their legally authorized representative must be able to provide written 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.
  3. Arm 3: female participants of must agree to use highly effective . 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.

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.
  3. Participant has undergone explantation of NT-501 prior to screening. Note: A participant who received bilateral implants and underwent explantation in only 1 eye prior to screening may be enrolled in the study.
  4. Participant received Encelto commercial implant in the fellow eye, if applicable, less than 3 months prior to study screening.

    Exclusion Criteria Applicable to Arm 3 Only:

  5. Participant has significant corneal or media opacities in the study eye to be implanted.
  6. Participant has evidence of pathologic myopia in the study eye to be implanted.
  7. 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.
  8. 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.
  9. 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).
  10. Received intravitreal therapy in the study eye to be implanted within 6 months prior to screening.
  11. 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.
  12. Evidence of central serous chorioretinopathy in the study eye to be implanted within 3 months prior to screening.
  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 that would compromise survival during the clinical study.
Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
234 participants (estimated)

Study arms

  • Other
    Arm 1 (Phase 3 Long-term Follow-up)

    For participants who received NT-501 (ENCELTO) in a previous Phase 3 study (NTMT-03-A or NTMT-03-B), or for participants who received NT-501 (ENCELTO) in both eyes (i.e. participated in study NTMT-02B) but received the first NT-501 (ENCELTO) implant in a Phase 3 study. No new treatment intervention will be administered. Arm 1 is focused on long term follow-up for participant who already has the ENCELTO implant at time of study screening and enrollment.

    Combination Product: NT-501/revakinagene taroretcel-lwey

  • Other
    Arm 2 (Phase 1 and Phase 2 Long-term Follow-up)

    For participants who received NT-501 (ENCELTO) in a previous Phase 1 study (NTMT-01), Phase 2 study (NTMT-02), in the sub study of Phase 1 and Phase 2 extension study (NTMT-01/02E), or for participants who received NT-501 (ENCELTO) in both eyes (i.e. participated in study NTMT-02B) but received the first NT-501 (ENCELTO) implant in a Phase 1 or Phase 2 study. No new treatment intervention will be administered. Arm 2 is focused on long term follow-up for participant who already has the ENCELTO implant at time of study screening and enrollment.

    Combination Product: NT-501/revakinagene taroretcel-lwey

  • Experimental
    Arm 3 (Phase 3 Sham to Implant Crossover)

    For participants who underwent Sham procedure in either Phase 3 study (NTMT-03-A or NTMT-03-B) and will receive NT-501 in this study.

    Combination Product: NT-501/revakinagene taroretcel-lwey

Interventions

  • Combination productNT-501/revakinagene taroretcel-lwey

    Drug page: Ciliary Neurotrophic Factor

    NT-501 is surgically implanted into the vitreous cavity to continuously release recombinant human ciliary neurotrophic factor (CNTF).

    Also known as: ENCELTO

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

07

Study locations

19 of 26 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Nov 2025 (actual). Primary completion is estimated for Jan 2032, 73 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Apr 29, 2025
First posted
May 14, 2025
StartActual
Nov 21, 2025
Primary completionEst.
Jan 15, 2032
CompletionEst.
Jan 15, 2032
Last update
Sep 25, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
6 sites changed recruiting status (6 now recruiting)
Show 6 sites
  • Stanford University School of Medicine · Palo Alto, United StatesNot yet recruiting→Recruiting
  • Colorado Retina · Lakewood, United StatesNot yet recruiting→Recruiting
  • Cumberland Valley Retina Consultants · Frederick, United StatesNot yet recruiting→Recruiting
  • Envision Ocular, LLC · Bloomfield, United StatesNot yet recruiting→Recruiting
  • Retina Northwest, P.C. · Portland, United StatesNot yet recruiting→Recruiting
  • University of Utah John A. Moran Center · Salt Lake City, United StatesNot yet recruiting→Recruiting
Sep 25, 2026
Show all 1 update
  1. Sep 25, 2026
    6 sites changed recruiting status (6 now recruiting)
    Show 6 sites
    • Stanford University School of Medicine · Palo Alto, United StatesNot yet recruiting→Recruiting
    • Colorado Retina · Lakewood, United StatesNot yet recruiting→Recruiting
    • Cumberland Valley Retina Consultants · Frederick, United StatesNot yet recruiting→Recruiting
    • Envision Ocular, LLC · Bloomfield, United StatesNot yet recruiting→Recruiting
    • Retina Northwest, P.C. · Portland, United StatesNot yet recruiting→Recruiting
    • University of Utah John A. Moran Center · Salt Lake City, United StatesNot yet recruiting→Recruiting
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT06971939
Lead sponsor
Neurotech Pharmaceuticals
Responsible party
Sponsor
First posted
May 14, 2025
Start date
Nov 21, 2025
Primary completion
Jan 15, 2032 (estimated)
Completion
Jan 15, 2032 (estimated)
Last update
Sep 25, 2026

Study contacts

Patricia Davis
Contact
p.davis@neurotechusa.com
401-333-3880
Thomas Aaberg, Jr, MD
study chair · Neurotech Pharmaceuticals

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Ciliary Neurotrophic Factor

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