NT-501 ECT Implant for Glaucoma

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Stanford University

Must not be taking: Acyclovir, Immunosuppressives, Chemotherapy, others

Disqualifiers: Retinal disease, Diabetic retinopathy, 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

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for glaucoma, a condition that damages the optic nerve and can lead to vision loss. The study tests an implant called NT-501 ECT, which releases a substance that may protect eye cells. Participants will either receive this implant or undergo a sham (fake) procedure to compare outcomes over 24 months. Individuals with glaucoma, stable eye pressure, and vision better than legal blindness might be suitable for this trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires that participants stop using acyclovir and related products before joining and throughout the study. If you are on systemic steroids or other immunosuppressive medications, you may not be eligible to participate.

What prior data suggests that the NT-501 ECT implant is safe for glaucoma patients?

Research has shown that the NT-501 ECT implant is safe. In previous studies, patients with glaucoma tolerated it well, with no serious side effects linked to the implant or the implantation procedure. This suggests the implant is likely safe. The device also improved the structure and function of patients' eyes, demonstrating its effectiveness. Overall, the NT-501 ECT implant appears safe based on current evidence. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for glaucoma?

Most treatments for glaucoma focus on reducing intraocular pressure through medications, laser treatments, or surgery. But the NT-501 ECT Implant works differently by delivering a sustained release of soluble ciliary neurotrophic factor (CNTF) directly into the eye. This method aims to protect and support the health of retinal cells, which is a novel approach compared to the typical pressure-lowering strategies. Researchers are excited because this could offer a new way to prevent vision loss in glaucoma patients by addressing the underlying health of the eye's cells, rather than just managing symptoms.

What evidence suggests that the NT-501 ECT implant is effective for glaucoma?

Research has shown that the NT-501 ECT implant, which participants in this trial may receive, could be a promising treatment for glaucoma. This implant releases a steady amount of ciliary neurotrophic factor (CNTF), a protein that can improve the eye's structure and function. Patients with the implant experienced positive changes in their vision, suggesting it might help slow or prevent vision loss. The implant has also proven safe, with most patients tolerating it well. Overall, early results are encouraging for NT-501 as a potential treatment for glaucoma. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for people with glaucoma who have specific visual field and retinal nerve fiber layer measurements, stable intraocular pressure, and good enough vision to participate. They must be able to undergo required tests, agree to birth control if applicable, and be fit for eye surgery. Exclusions include certain drug histories, other health conditions that could interfere with the study or pose risks.

Inclusion Criteria

I have glaucoma with stable eye pressure and some vision left.

Participant must be medically able to undergo the testing required in the schedule of events (SOE).

Participant must understand and sign the informed consent.

Exclusion Criteria

I am currently taking steroids or other medications that weaken my immune system.

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.

I have a disease affecting my optic nerve or retina that causes vision loss.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the NT-501 ECT implant or sham surgery and are followed for safety and efficacy

24 months

Regular visits for safety assessments, including 1 day, 1 week, and periodic visits thereafter

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Open-label extension (optional)

Participants in the control arm may receive the NT-501 ECT implant and be followed for an additional 12 months

12 months

What Are the Treatments Tested in This Trial?

Interventions

• NT-501 ECT implant

Trial Overview The trial is testing NT-501 encapsulated cell therapy (ECT) implant against a sham surgery in patients with glaucoma. Participants are randomly assigned to either receive the real implant or a fake procedure without explant needed. Safety checks follow one day and one week after surgery then periodically over two years.

How Is the Trial Designed?

Group I: NT-501 ECT Implant Experimental Treatment 1 Intervention

On eligible participants that are randomized to this group, the NT-501 Investigational product will be implanted in the study eye, and participants will be followed for 24 months. The investigational treatment, NT-501 ECT, provides intravitreal sustained release of soluble ciliary neurotrophic factor (CNTF) receptor after intraocular implantation.

Group II: Sham Placebo Group 1 Intervention

To maintain masking, participants randomized to this group receive sham surgery and will be followed for 12 months. Following analysis of the 6 month data, if the open label extension (OLE) is not triggered, patients in the control group will continue on the original study schedule timeline trough month 24. If OLE is triggered participants will be offered the NT-501 ECT investigational product and will followed for additional 12 months.

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10183667/

Phase I NT-501 Ciliary Neurotrophic Factor Implant Trial for ... The NT-501 CNTF implant was safe and well tolerated in eyes with POAG. Eyes with the implant demonstrated both structural and functional improvements.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02862938

Study of NT-501 Encapsulated Cell Therapy for Glaucoma ... Determine the efficacy of NT-501 ECT implant in patients with glaucoma as measured by change in visual field through 6 months as assessed by any one of the ...

3. iovs.arvojournals.org iovs.arvojournals.org/article.aspx?articleid=2800282

A Randomized, Sham Controlled, Masked Phase II Study for ... The NT-501 implant demonstrated an excellent safety profile in glaucoma patients, with promising structural enhancement indicating biological activity.

4. brightfocus.org brightfocus.org/grant/neuroregenerative-strategies-in-glaucoma/

Neuroregenerative Strategies in Glaucoma The NT-501 ECT contains cells designed to deliver a steady stream of a growth factor, called ciliary neurotrophic factor (CNTF), to test whether it can protect ...

5. clinicaltrials.stanford.edu clinicaltrials.stanford.edu/trials/d/NCT04577300.html

Dual Intravitreal Implantation of NT-501 Encapsulated Cell ... Purpose. To determine the safety and efficacy over 24 months of dual NT-501 CNTF encapsulated cell therapy (ECT) on visual impairment related to glaucoma.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5436351/

Delivery of therapeutics to posterior eye segment: cell ... There were no serious adverse events associated with the NT-501 device or the implantation procedures. ... As for glaucoma, early data from Phase 1 trial ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04577300

NCT04577300 | Dual Intravitreal Implantation of NT-501 ... To determine the safety and efficacy over 24 months of dual NT-501 CNTF encapsulated cell therapy (ECT) on visual impairment related to glaucoma. Detailed ...