NT-501 for MacTel

Not yet recruiting at 35 trial locations

Overseen By Patricia Davis

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Neurotech Pharmaceuticals

Must not be taking: Intravitreal steroids, Anti-VEGF

Disqualifiers: Ocular disease, Chemotherapy, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety and effectiveness of NT-501, an implant for individuals with macular telangiectasia type 2 (MacTel), a condition affecting the retina. Participants who received the NT-501 implant in earlier trials will continue to be monitored, while those who underwent a sham procedure will now receive the implant. Ideal candidates for this trial are those who participated in earlier NT-501 MacTel studies and have stable eye fixation, allowing for clear retinal imaging. As a Phase 4 trial, this research focuses on an FDA-approved treatment, aiming to understand its benefits for a broader patient population.

Do I have to stop taking my current medications for the NT-501 for MacTel trial?

The trial information does not specify if you need to stop taking your current medications. However, if you have received certain eye treatments like intravitreal steroid or anti-VEGF therapy recently, you may not be eligible to participate.

What is the safety track record for NT-501?

Research has shown that NT-501, also known as Encelto™, has undergone safety testing in earlier studies. Larger studies found that most participants generally tolerated the treatment well. The main goal was to check for serious side effects and any major vision loss, such as losing 15 or more letters on an eye chart.

Results indicated that serious side effects were rare and similar to those in people who did not receive the treatment, suggesting a relatively low risk of severe problems. NT-501 has been tested multiple times, so its safety is well-understood.

Since this study is in Phase 4, the treatment has already passed earlier safety checks and is now being studied for long-term effects. This adds extra confidence in its safety for people with macular telangiectasia type 2 (MacTel). However, discussing any concerns or questions with the study team and your doctor before joining a trial is always important. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

NT-501 is unique because it offers a new approach to treating Macular Telangiectasia (MacTel) by using a tiny implant that releases a protein to support retinal health. Unlike other treatments that typically involve injections into the eye, NT-501 provides a more sustained delivery method, potentially reducing the frequency of treatments. Researchers are excited about NT-501 because it targets the disease at a cellular level, aiming to preserve vision by slowing the progression of retinal degeneration. This innovative delivery system could improve patient adherence and overall outcomes.

What is the effectiveness track record for NT-501 in treating MacTel?

Studies have shown that NT-501 effectively treats macular telangiectasia type 2 (MacTel), a condition affecting the eyes. Research indicates that NT-501 protects the eye's light-sensing cells, called photoreceptors, from damage. This treatment has slowed the disease's progression over 24 months. In previous trials, patients who received NT-501 achieved better results compared to those who received a placebo. In this trial, participants in Arm 3, who previously underwent a sham procedure, will receive NT-501. NT-501 is already approved for treating adults with MacTel, highlighting its proven effectiveness. ^{1 2 6 7 8}

Who Is on the Research Team?

Thomas Aaberg, Jr, MD

Principal Investigator

Neurotech Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for people with a condition called MacTel Type 2 who have already been part of earlier phases (1, 2, or 3) of NT-501 studies. It's also open to those who had a sham procedure in Phase 3 and now choose to get the real NT-501 implant.

Inclusion Criteria

I have completed an NT-501 MacTel study before.

I can focus steadily and have clear enough vision for quality retinal images.

I can give my consent or have someone who can legally do it for me.

Exclusion Criteria

Participant is living outside the US

I have a history of cancer.

I haven't had cancer in the last 5 years, except for skin cancer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Implantation

Participants in Arm 3 who previously underwent the sham procedure will receive the NT-501 implant

Perioperative period

1 visit (in-person)

Long-term Follow-up

Participants are monitored for safety and efficacy of NT-501 implant

5 years

Annual visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• NT-501

Trial Overview The study tests the long-term safety and effectiveness of an eye implant called NT-501 for treating MacTel Type 2. Participants from previous trials will continue to be observed, and new participants from the sham group will receive the actual treatment.

How Is the Trial Designed?

Group I: Arm 3 (Phase 3 Sham to Implant Crossover) Experimental Treatment 1 Intervention

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.

Group II: Arm 2 (Phase 1 and Phase 2 Long-term Follow-up) Experimental Treatment 1 Intervention

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.

Group III: Arm 1 (Phase 3 Long-term Follow-up) Experimental Treatment 1 Intervention

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 2 is focused on long term follow-up for participant who already has the ENCELTO implant at time of study screening and enrollment.

Who Is Running the Clinical Trial?

Neurotech Pharmaceuticals

Lead Sponsor

Trials

12

Recruited

680+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06971939

Phase 4 Study: Long-term Safety and Efficacy of NT-501 in ... 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 ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40693847/

Cell-Based Ciliary Neurotrophic Factor Therapy for ... Conclusions: NT-501 for MacTel resulted in statistically significantly reduced EZA loss compared with sham procedures. (Funded by Neurotech ...

3. encelto.com encelto.com/ecp/

ENCELTO for MacTel type 2 ENCELTO is an allogeneic encapsulated cell-based gene therapy indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel).

4. scripps.edu scripps.edu/news-and-events/press-room/2025/20250801-friedlander-encelto.html

Study confirms long-term benefit of implant for blinding eye ... While both trials showed that ENCELTO slowed the loss of light-sensing retinal cells, the effect was stronger in one trial, likely due to ...

5. neurotechpharmaceuticals.com neurotechpharmaceuticals.com/wp-content/uploads/Neurotech_Press-Release_BLA_Approval_FINAL.pdf

Neurotech's ENCELTOTM (revakinagene taroretcel-lwey) ... the implant, ENCELTO significantly slowed the loss of macular photoreceptors in MacTel patients over. 24 months. ENCELTO is expected to be ...

6. fda.gov fda.gov/media/185972/download?attachment

ENCELTO The primary safety evaluation is based on data from the two phase 3 studies with additional, supportive safety analyses conducted on data from ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03316300

NCT03316300 | A Study to Determine the Safety and ... This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia ...

8. evidence.nejm.org evidence.nejm.org/doi/abs/10.1056/EVIDoa2400481

Cell-Based Ciliary Neurotrophic Factor Therapy for Macular ... Safety end points included the proportion of participants experiencing one or more treatment-emergent serious adverse event(s) and loss of 15 or more letters in ...