Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Stanford University
Must not be taking: Acyclovir, Steroids, Immunosuppressants, Chemotherapy
Disqualifiers: Retinal disease, Cataracts, Diabetes, others
No Placebo Group
Prior Safety Data
Approved in 1 jurisdiction
Trial Summary
What is the purpose of this trial?This trial tests tiny devices that release a protective protein into the eyes of patients with glaucoma-related vision problems. The protein helps protect nerve cells, potentially preventing further vision loss and improving sight.
Will I have to stop taking my current medications?
The trial requires that you stop taking acyclovir and related products before enrolling and throughout the study. If you are on systemic steroids, immunosuppressive medications, or chemotherapy, you cannot participate in the trial.
What data supports the effectiveness of the NT-501 treatment for glaucoma?
Research shows that ciliary neurotrophic factor (CNTF), a component of NT-501, has been effective in slowing retinal degeneration in animal models and improving visual acuity in some human trials for retinal diseases. Additionally, CNTF has shown potential in stimulating the regeneration of retinal cells in glaucoma models, suggesting it may help protect and enhance vision.
12345Is NT-501 Cell Therapy safe for humans?
The NT-501 Cell Therapy, which uses ciliary neurotrophic factor (CNTF) delivered through an encapsulated cell implant, has been shown to be safe in human trials for retinal conditions. In a Phase I trial, it was well-tolerated with minimal side effects, and a similar trial for glaucoma also focused on safety.
13456How is the NT-501 treatment for glaucoma different from other treatments?
The NT-501 treatment for glaucoma is unique because it uses an encapsulated cell implant to deliver ciliary neurotrophic factor (CNTF) directly into the eye, which may protect and enhance the function of retinal cells, unlike traditional treatments that primarily focus on lowering eye pressure.
12457Eligibility Criteria
This trial is for individuals with stable glaucoma, having an eye pressure (IOP) less than 21 and visual field loss within a specific range. Participants must have some remaining vision (20/200 or better), be able to undergo required tests, and agree to birth control if applicable. Exclusions include those with certain other eye conditions, recent participation in another drug trial, pregnancy, or systemic diseases affecting the eyes.Inclusion Criteria
Females of childbearing potential must agree to use an effective form of birth control
I am medically fit to undergo all required tests.
Participant's eye pressure must be clinically stable, with IOP <21
+10 more
Exclusion Criteria
I can follow the study's procedures and attend all visits.
You have had a past occurrence of eye herpes.
I am currently undergoing chemotherapy.
+16 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive NT-501 ECT implants or sham surgery, with follow-up examinations for safety and efficacy
24 months
Initial visit for implantation, followed by periodic visits for 24 months
Follow-up
Participants are monitored for safety and effectiveness after treatment, with an option for control arm participants to receive implants after 12 months
12 months
Periodic visits
Participant Groups
The study is testing NT-501 Encapsulated Cell Therapy (ECT), which aims to treat visual impairment due to glaucoma over a period of 24 months. Participants will receive either the NT-501 implants or a sham procedure as a comparison.
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Single ImplantationExperimental Treatment1 Intervention
One NT-501 device will be implanted in the study eye.
Group II: Dual ImplantationExperimental Treatment1 Intervention
Two NT-501 devices will be implanted in the study eye.
Group III: Sham ImplantationPlacebo Group1 Intervention
No NT-501 devices will be implanted in the study eye.
NT-501 is already approved in United States for the following indications:
🇺🇸 Approved in United States as NT-501 for:
- Glaucoma
- Retinitis pigmentosa
- Age-related macular degeneration
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Byers Eye Institute at Stanford UniversityPalo Alto, CA
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Who Is Running the Clinical Trial?
Stanford UniversityLead Sponsor
References
Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants. [2022]Neurotrophic factors are agents with a promising ability to retard progression of neurodegenerative diseases and are effective in slowing photoreceptor degeneration in animal models of retinitis pigmentosa. Here we report a human clinical trial of a neurotrophic factor for retinal neurodegeneration. In this Phase I safety trial, human ciliary neurotrophic factor (CNTF) was delivered by cells transfected with the human CNTF gene and sequestered within capsules that were surgically implanted into the vitreous of the eye. The outer membrane of the encapsulated cell implant is semipermeable to allow CNTF to reach the retina. Ten participants received CNTF implants in one eye. When the implants were removed after 6 months, they contained viable cells with minimal cell loss and gave CNTF output at levels previously shown to be therapeutic for retinal degeneration in rcd1 dogs. Although the trial was not powered to form a judgment as to clinical efficacy, of seven eyes for which visual acuity could be tracked by conventional reading charts, three eyes reached and maintained improved acuities of 10-15 letters, equivalent to two- to three-line improvement on standard Snellen acuity charts. A surgically related choroidal detachment in one eye resulted in a transient acuity decrease that resolved with conservative management. This Phase I trial indicated that CNTF is safe for the human retina even with severely compromised photoreceptors. The approach to delivering therapeutic proteins to degenerating retinas using encapsulated cell implants may have application beyond disease caused by genetic mutations.
Expression of ciliary neurotrophic factor after induction of ocular hypertension in the retina of rats. [2007]Glaucoma is mainly characterized by the loss of retinal ganglion cells. Ciliary neurotrophic factor (CNTF) is believed to stimulate the regeneration of axons of retinal ganglion cells. The objective of our study was to detect the expression of CNTF in the retina of a rat glaucoma model with increased intraocular pressure (IOP).
Topical instillation of ciliary neurotrophic factor inhibits retinal degeneration in streptozotocin-induced diabetic rats. [2019]Intraocular injections of ciliary neurotrophic factor (CNTF) or intraocular adenovirus-mediated CNTF gene transfer have been reported to inhibit retinal alterations in inherited retinal degeneration in mice strains. To investigate whether or not CNTF administered by eye drops prevents retinal degeneration in streptozotocin (STZ)-induced diabetic rats, recombinant CNTF was administered to eyes of diabetic rats (n=20) twice daily for 1 month after the onset of diabetes. The b-wave amplitude of electroretinogram in CNTF-administered diabetic rats was significantly larger than that of diabetic rats, and approached that of the controls. Atrophy of the inner plexiform layer, and cavity formation in the pigment epithelium, which were observed in diabetic rats, were prevented in CNTF-administered diabetic rats. These results indicate that CNTF administration by eye drops prevents retinal degeneration in STZ-induced diabetic rats.
Ciliary neurotrophic factor for macular telangiectasia type 2: results from a phase 1 safety trial. [2022]To evaluate the safety and tolerability of intraocular delivery of ciliary neurotrophic factor (CNTF) using an encapsulated cell implant for the treatment of macular telangiectasia type 2.
Phase I NT-501 Ciliary Neurotrophic Factor Implant Trial for Primary Open-Angle Glaucoma: Safety, Neuroprotection, and Neuroenhancement. [2023]To assess the safety and efficacy of a ciliary neurotrophic factor (CNTF) intraocular implant on neuroprotection and neuroenhancement in glaucoma.
Ciliary neurotrophic factor (CNTF) delivery to retina: an overview of current research advancements. [2019]The intraocular administration of the ciliary neurotrophic factor (CNTF) has been found to attenuate the photoreceptor degeneration and preserve retinal functions in the animal research models of the inherited or induced retinal disease. Studies with the aim of CNTF transfer to the posterior segment inside the eye have been directed to determine the best method for its administration. An ideal delivery method would overcome the eye drug elimination mechanisms or barriers and provide the sustained release of the CNTF into retina in the safest fashion with the minimum harm to the quality of life. This review focuses on the present state of CNTF delivery to retina, also provides an overview of available technologies and their challenges.
[Protective effects of transplantation of hCNTF-transfected cell line on axotomy-induced degeneration of retinal ganglion cells]. [2005]To investigate the protective effects of transplantation of cell line stably expressing and secreting human ciliary neurotrophic factor (hCNTF) on the degeneration of retinal ganglion cells (RGCs) after optic nerve transection.