RGN-259 for Neurotrophic Keratopathy

Recruiting at 34 trial locations

Overseen By Ted Um

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: ReGenTree, LLC

Must not be taking: Lacrimation drugs, Immunosuppressives

Disqualifiers: Ocular surgery, Autoimmune disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 37

Duration: 4 weeks

70 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new eye drop treatment called RGN-259 for Neurotrophic Keratopathy (NK), a condition affecting the cornea that can cause vision problems. Researchers aim to determine if this treatment is safe and effective compared to a placebo (a substance with no active medicine). Participants will apply the eye drops five times a day for 28 days. Individuals with a persistent corneal defect for over a week, despite other treatments, might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop taking my current medications to join the trial?

The trial protocol does not clearly specify if you need to stop taking your current medications. However, you cannot use drugs that affect tear production or the trigeminal nerve, or topical immunosuppressive therapy within 30 days before the study, and you should not anticipate using them during the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that RGN-259, an eye drop containing Thymosin Beta 4 (Tβ4), is generally safe and well-tolerated. In earlier studies, participants using RGN-259 did not experience significant side effects. These studies found that the treatment helped heal damage to the eye's surface, particularly in cases where corneal wounds were not healing. In special cases where doctors used the treatment for severe conditions outside of a clinical trial, no noticeable side effects occurred, and positive healing effects were observed.

This suggests that the treatment is safe for humans, with no major safety concerns reported so far. ^{1 2 3 4 5}

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

Unlike the standard treatments for neurotrophic keratopathy, which often include artificial tears or surgery, RGN-259 is unique because it contains Thymosin Beta 4 (Tβ4), a novel peptide that promotes wound healing and tissue regeneration. This preservative-free, sterile ophthalmic solution is applied directly to the eyes, potentially offering a more effective and convenient treatment option. Researchers are excited about RGN-259 because it targets the underlying biological processes of the condition, rather than just alleviating symptoms, which could lead to faster and more comprehensive healing.

What evidence suggests that RGN-259 might be an effective treatment for Neurotrophic Keratopathy?

Research has shown that RGN-259, an eye drop containing thymosin beta-4, may help treat neurotrophic keratopathy (NK), a condition affecting the eye's surface. In one study, 6 out of 10 people using RGN-259 experienced complete healing after four weeks, compared to only 1 out of 8 people using a placebo. Another study found a 35% decrease in eye discomfort and a 59% decrease in eye surface damage for those treated with thymosin beta-4. These findings suggest that RGN-259 could aid in healing the eye and reducing NK symptoms. However, not all studies have consistently met their primary objectives. Participants in this trial will receive either the 0.1% RGN-259 Ophthalmic Solution or a Placebo Ophthalmic Solution as part of the study's treatment arms. ^{1 3 4 5 6}

Are You a Good Fit for This Trial?

Adults with stage 2 or 3 neurotrophic keratopathy (NK) in one eye, confirmed not to be just superficial damage. They must have reduced corneal sensitivity and a specific level of vision impairment. Participants need to agree to contraception use and can't have lid abnormalities, active infections/inflammation unrelated to NK, recent ocular surgery, or conditions that might affect the study's outcome.

Inclusion Criteria

Have provided written informed consent

I agree to use effective birth control.

Have evidence of decreased corneal sensitivity ≤40 mm using the Cochet-Bonnet aesthesiometer at Visit 1

Exclusion Criteria

Have any clinically significant slit-lamp findings in the study eye that in the opinion of the investigator may interfere with the study parameters

I do not have an autoimmune or chronic inflammatory disease that could affect the study treatment.

I plan to use serum tears in my study eye.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 0.1% RGN-259 Ophthalmic Solution or placebo five times per day for 28 days

4 weeks

Visits on Day 1, 8, 15, 22, 29

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

Visits on Day 36 and 43

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• RGN-259

Trial Overview The trial is testing RGN-259 ophthalmic solution against a placebo for treating NK. The goal is to see if RGN-259 is safe and works better than a placebo in healing the eye condition.

How Is the Trial Designed?

Group I: 0.1% RGN-259 Opthalmic Solution Experimental Treatment 1 Intervention

It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into study eye(s), five times per day for 28 days

Group II: Placebo Ophthalmic Solution (Vehicle for RGN-259 Ophthalmic Solution) Placebo Group 1 Intervention

It is composed of the same excipients as RGN-259 but does not contain Tβ4

Who Is Running the Clinical Trial?

ReGenTree, LLC

Lead Sponsor

Trials

7

Recruited

1,800+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9820614/

0.1% RGN-259 (Thymosin ß4) Ophthalmic Solution Promotes ... RGN-259-treated subjects had significant improvements at multiple time points in ocular discomfort, foreign body sensation, and dryness which ...

2. regenerx.com regenerx.com/2023-01-03-Publication-of-RGN-259-Phase-3-Clinical-Trial-Results-in-Patients-with-Neurotrophic-Keratopathy

Publication of RGN-259 Phase 3 Clinical Trial Results in ... Complete healing occurred after 4 weeks in 6 of 10 RGN-259-treated subjects and 1 of 8 placebo-treated subjects, indicating a strong efficacy ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05555589

Study Details | NCT05555589 | Assessment of the Safety ... Neurotrophic keratopathy (NK) is a degenerative corneal disease that occurs as a result of partial or total impairment of trigeminal innervation.

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0083672916300255

Thymosin Beta 4: A Potential Novel Therapy for ... The 12 eyes treated with Tβ4 had a 35% reduction of ocular discomfort compared to vehicle control (6 eyes) (p = 0.0141), and a 59% reduction of total corneal ...

5. ophthalmologytimes.com ophthalmologytimes.com/view/hlb-therapeutics-misses-primary-endpoint-in-phase-3-seer-3-trial-of-rgn-259

HLB Therapeutics misses primary end point in phase 3 ... RGN-259, a thymosin beta-4–based eye drop, failed to meet the primary endpoint in a European phase 3 trial for neurotrophic keratitis. The ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01387347

Safety and Efficacy of Thymosin Beta 4 Ophthalmic ... In compassionate-use cases, Tβ4 has demonstrated efficacy in repairing non-healing neurotrophic corneal ulcers and other corneal epithelial wounds. In ...