Recruiting

NT-501

Sponsor:

Neurotech Pharmaceuticals

Code:

NCT06971939

Conditions

Macular Telangiectasia Type 2 (MacTel)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

NT-501/revakinagene taroretcel-lwey

Study Details

Brief 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.

Conditions

Macular Telangiectasia Type 2 (MacTel)

Study ID

NCT06971939

Start date

Nov 21, 2025

Status verified date

Jul, 2026

Completion date

Jan 15, 2032

Anticipated

Primary completion date

Jan 15, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 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.
3. 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.

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 steroid 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 malignancy that would compromise survival during the clinical study.

Study Design

Enrollment

234 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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.

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.

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.

Interventions

NT-501/revakinagene taroretcel-lwey

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

Primary outcome measure

  • 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 [ 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 ]

Central Contacts and Locations

Central contacts

Locations

Bay Area Retina Associates

Recruiting

Walnut Creek, California, United States, 94598

Contacts

Principal Investigator:

Roger Goldberg, MD

Emory Eye Center

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Donna Leef

dleef@emory.edu

Principal Investigator:

Jiong Yan, MD

Northwestern University

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Principal Investigator:

Ivy Zhu, MD

Foundation for Vision Research

Recruiting

Grand Rapids, Michigan, United States, 49525

Contacts

Holly Vincent

holly@fvr.org

Principal Investigator:

Liliya Sutherland, MD

Cincinnati Eye Institute

Recruiting

Cincinnati, Ohio, United States, 45242

Contacts

Principal Investigator:

Lucas Lindsell, MD

Retina Associates of Cleveland, Inc.

Recruiting

Cleveland, Ohio, United States, 44126

Contacts

Principal Investigator:

Joseph Coney, MD

Southeastern Retina Associates, P.C.

Recruiting

Knoxville, Tennessee, United States, 37922

Contacts

Principal Investigator:

Stephen Perkins, MD

Texas Retina Associates

Recruiting

Dallas, Texas, United States, 75231

Contacts

Principal Investigator:

Ashkan Abbey, MD

Retina Center of Texas

Recruiting

Dallas, Texas, United States, 75251

Contacts

Principal Investigator:

Jawad Qureshi, MD

Retina Consultants of Texas

Recruiting

Houston, Texas, United States, 77030

Contacts

Principal Investigator:

Charles Wykoff, MD

More Information

Sponsor

Neurotech Pharmaceuticals

Last update posted

Aug 13, 2026

Last verified

Jul, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Neurotech Pharmaceuticals on 2026-08-13.