Dual implantation of NT-501 therapy for glaucoma treatment

A Randomized, Sham Controlled, Masked Phase II Study to Evaluate the Safety and Efficacy of Dual Intravitreal Implantation of NT-501 Encapsulated Cell Therapy for the Treatment of Glaucoma

This study is testing if two NT-501 implants can improve vision for people with glaucoma compared to one implant or a fake surgery.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Stanford University (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Palo Alto, California)
• Trial ID: NCT04577300 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the safety and efficacy of dual NT-501 encapsulated cell therapy in treating visual impairment related to glaucoma over a 24-month period. Participants will be randomly assigned to receive either two NT-501 implants, one implant, or a sham surgery. The study will involve up to 30 eyes, with safety examinations conducted shortly after the procedure and periodically throughout the study duration. The goal is to assess the potential benefits of this innovative therapy in improving visual outcomes for glaucoma patients.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with glaucoma who have stable intraocular pressure and preserved visual acuity.

Not a fit: Patients with advanced glaucoma leading to severe visual impairment or those with unstable eye pressure may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve visual function and quality of life for patients with glaucoma.

How similar studies have performed: While this approach is innovative, similar studies using encapsulated cell therapies have shown promise in other conditions, suggesting potential for success in this context.

Eligibility criteria

Inclusion Criteria:

1. Participant must be medically able to undergo the testing required in the schedule of events (SOE).
2. Participant's clinical diagnosis must be consistent with glaucoma characterized by the following features: Mean deviation (MD) of -3 to -20 dB on Humphrey Visual Field 24-2 testing and two visual field tests of adequate quality with a maximum VFI variability of ± 10%
3. Residual visual field preservation including best-corrected visual acuity (BCVA) better than 20/200 in either eye.
4. Participant's eye pressure must be clinically stable, with IOP \<21.
5. If a participant has two eyes meeting study criteria, the worse eye as determined by visual field index (VFI) or patient preference, will be deemed includable. If both eyes qualify and have the same VFI, a randomization procedure will assign one eye to the study.
6. Participant must understand and sign the informed consent. If the participant's vision is impaired to the point where he/she cannot read the informed consent document, the document will be read to the participant in its entirety.
7. Females of childbearing potential must agree to use an effective form of birth control.
8. Participant must be determined by the presurgical anesthesia or medical team to be fit for ophthalmic surgery for the NT-501 ECT implant insertion.

Exclusion Criteria:

1. Participant is unable to comply with study procedures or followup visits.
2. Participant has other optic nerve or retinal degenerative disease causing vision loss, irrespective of whether it is currently treated or untreated.
3. Participant is likely to be offered glaucoma surgery in the study eye within 6 months of screening.
4. Participant has cataract-associated vision loss to less than 20/40.
5. Participant has a history of ocular herpes zoster.
6. Participant has a requirement of acyclovir and/or related products during study duration. To be eligible for this study, the participant must discontinue use of these products prior to enrollment and must not continue with the products until after they have completed the study.
7. Participant has evidence of corneal opacification or lack of optical clarity.
8. Participant has uveitis or other ocular inflammatory disease.
9. Participant is receiving systemic steroids or other immunosuppressive medications.
10. Participant has diabetic macular edema and/or diabetic retinopathy.
11. Participant has myopic degeneration.
12. Participant is currently participating in or has within the last 3 months participated in any other clinical trial of a drug by ocular (if in the study eye) or systemic administration.
13. Participant is pregnant or lactating.
14. Participant is on chemotherapy.
15. Participant has a history of malignancy other than basal cell carcinoma, unless it was treated successfully 2 years prior to inclusion in the trial.
16. 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.
17. Any intraocular surgery of the study eye within 12 weeks prior to the screening visit
18. History of use of drugs with known retinal toxicity, at retinotoxic doses.
19. Patient has a history of multiple sclerosis.

Where this trial is running

Palo Alto, California

• Byers Eye Institute at Stanford University — Palo Alto, California, United States (Recruiting)

Study contacts

• Principal investigator: Jeffrey L Goldberg, MD, PhD — Stanford University
• Study coordinator: Mariana Nunez, MD

Conditions Glaucoma