OCS-01 for Macular Edema

(LEOPARD Trial)

Not currently recruiting at 7 trial locations

Overseen By Quan D Nguyen, MD, MSc

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Quan Dong Nguyen

Must be taking: Topical NSAID, Topical corticosteroids

Must not be taking: Immunosuppressives, Biologics

Disqualifiers: Diabetes, Glaucoma, Active infection, 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: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new steroid eye drop, OCS-01, for treating macular edema, a fluid buildup in the back of the eye that can cause vision loss. The trial targets individuals with macular edema due to uveitis (an eye disease causing inflammation) or following eye surgery. Participants will receive either a high or low dose of OCS-01 to assess its safety and effectiveness in clearing fluid and improving vision. Those who have had macular edema for less than a year, following uveitis or surgery, might be suitable for this study. Regular eye exams will monitor safety and effectiveness throughout the 24-week trial period. As a Phase 2 trial, this study measures how well OCS-01 works in an initial, smaller group, offering participants a chance to contribute to important research and potentially benefit from an innovative treatment.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify if you must stop all current medications. However, you cannot use other ophthalmic formulations during the study, except for intraocular pressure-lowering eye drops if needed. Also, if you have UME, your dose of immunosuppressives must be stable for 2 months before the trial, and oral prednisone must be stable for 1 month before the trial. Please consult with the trial team for specific guidance on your medications.

Is there any evidence suggesting that this trial's treatment is likely to be safe?

Research has shown that OCS-01, a type of steroid eye drop, has been well tolerated in past studies. Earlier trials used OCS-01 to treat eye conditions like pain and swelling after cataract surgery, and results indicated it was generally safe, with most patients not experiencing serious side effects.

Another study tested OCS-01 on patients with diabetic macular edema, a condition affecting the eye. These patients also tolerated the treatment well. While specific side effects weren't detailed, the overall findings suggest that OCS-01 is generally safe.

Currently, this treatment is in a mid-stage trial (Phase 2). While initial safety has been established, more detailed safety information is still being collected. Patients considering joining this trial can feel somewhat reassured by the earlier positive safety records but should understand that ongoing evaluation is part of the process. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for macular edema, like anti-VEGF injections and corticosteroids, focus on reducing fluid and swelling in the retina. But OCS-01 offers a different approach by delivering medication through eye drops, potentially making administration more comfortable and convenient for patients. Researchers are excited about OCS-01 because it targets the condition directly at the site of the edema with a novel formulation, which could lead to improved outcomes without the need for invasive procedures. Additionally, the flexibility in dosing—high or low—may allow for personalized treatment plans tailored to individual patient needs.

What evidence suggests that OCS-01 might be an effective treatment for macular edema?

Research has shown that OCS-01 eye drops may help treat macular edema, particularly in cases like diabetic macular edema (DME). Studies found that OCS-01 reduced the thickness at the center of the retina more effectively than a placebo, which is crucial for alleviating this condition. Additionally, patients experienced improved vision, as indicated by higher scores in best-corrected visual acuity (BCVA), a measure of clear vision. The treatment has also proven effective and well-tolerated for reducing inflammation and pain after cataract surgery. These findings suggest that OCS-01 could help reduce fluid buildup in the eye and improve vision. Participants in this trial will receive either a high dose or a low dose of OCS-01, with the low dose group also receiving placebo drops. ^{1 4 5 6 7}

Who Is on the Research Team?

Quan D Nguyen, MD, MSc

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

Adults over 18 with Uveitic macular edema (UME) or post-surgical macular edema (PSME), who have had the condition for less than 3 years (UME) or less than 1 year (PSME). Participants must have a certain level of vision impairment and be able to attend all study visits. Those with macular edema due to other causes, significant eye diseases, high myopia, history of steroid-induced increased eye pressure, active infections, pregnancy/breastfeeding, unstable uveitis or immunosuppressive treatment are excluded.

Inclusion Criteria

I tried a skin cream for pain or swelling without success for 1 to 3 months.

An ETDRS BCVA letter score ≤ 70 (Snellen 20/40) and ≥ 35 (Snellen 20/200) in the study eye at baseline (Visit 2)

My eye condition has lasted less than a year and involves fluid buildup.

Exclusion Criteria

Pregnancy/Breastfeeding

My dose of immunosuppressants has not increased in the last 2 months.

I can use eye drops for high eye pressure if needed during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Loading

Participants receive 01 drop of OCS-01, six times a day from baseline until week 4

4 weeks

Regular visits for eye exams

Treatment

Participants continue their assigned treatments until week 12, with primary endpoint assessments at week 12

8 weeks

Regular visits for eye exams

Follow-up

Participants receive treatment based on retreatment criteria from week 12 to week 24

12 weeks

Regular visits for eye exams

What Are the Treatments Tested in This Trial?

Interventions

• OCS-01

Trial Overview The LEOPARD trial is testing OCS-01 steroid eye drops' safety and effectiveness in treating fluid buildup in the back of the eye caused by UVE or following surgery. Patients will use different doses for 24 weeks while undergoing regular detailed eye exams to monitor their response and any side effects.

How Is the Trial Designed?

Group I: Low dose PSME - 3 drops of OCS-01 and 3 drops of Placebo Experimental Treatment 1 Intervention

From baseline until week 4 all participants will receive 01 drop of OCS-01, six times a day. At week 4, the participants randomized to low dose group will receive 01 drop of OCS-01 three times a day and 01 drop of placebo three times a day, (total 6 drops each day) until the primary end point at week 12. Starting week 12 the treatment will be administered based on the retreatment criteria until the end of study at week 24.

Group II: Low Dose UME 3 drops OCS-01 and 3 drops Placebo Experimental Treatment 1 Intervention

From baseline until week 4 all participants will receive 01 drop of OCS-01, six times a day. At week 4, the participants randomized to low dose group will receive 01 drop of OCS-01 three times a day and 01 drop of placebo three times a day,( total 6 drops each day) until the primary end point at week 12. Starting week 12 the treatment will be administered based on the retreatment criteria until the end of study at week 24.

Group III: High dose PSME - 6 drops of OCS-01 Experimental Treatment 1 Intervention

From baseline until week 4 all participants will receive 01 drop of OCS-01, six times a day. At week 4, participants randomized to the high dose group will continue to receive 01 drop of OCS-01 six times a day until the primary end point at week 12. Starting week 12 the treatment will be administered based on the retreatment criteria until the end of study at week 24.

Group IV: High Dose UME - 6 drops OCS-01 Experimental Treatment 1 Intervention

From baseline until week 4 all participants will receive 01 drop of OCS-01, six times a day. At week 4, participants randomized to the high dose group will continue to receive 01 drop of OCS-01 six times a day until the primary end point at week 12. Starting week 12 the treatment will be administered based on the retreatment criteria until the end of study at week 24.

Who Is Running the Clinical Trial?

Quan Dong Nguyen

Lead Sponsor

Trials

3

Recruited

70+

Oculis

Industry Sponsor

Trials

9

Recruited

1,700+

Global Ophthalmic Research Center (GORC)

Collaborator

Trials

2

Recruited

40+

Published Research Related to This Trial

In a Phase II study involving 153 adult patients after cataract surgery, topical dexamethasone 1.5% (OCS-01) significantly reduced inflammation, with 51% of patients on once-daily treatment and 66.7% on BID treatment showing no anterior chamber cells by Day 15, compared to only 19.6% in the vehicle group.

Both OCS-01 treatment regimens also effectively alleviated pain, with 72.5% of patients on once-daily and 62.7% on BID reporting no pain by Day 4, while the vehicle group had only 45.1% reporting no pain, indicating that OCS-01 is both effective and well tolerated.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

OCS-01 (Novel Topical Dexamethasone Formulation) in Inflammation and Pain Post Cataract Surgery: A Randomized, Double-Masked, Vehicle-Controlled Study. Korenfeld, M., Gira, J., Jong, K., et al.[2022]

In a study involving 1048 patients with diabetic macular edema, both the 0.7 mg and 0.35 mg dexamethasone intravitreal implants significantly improved best-corrected visual acuity (BCVA) compared to a sham treatment, with 22.2% and 18.4% of patients achieving a ≥15-letter improvement, respectively.

The safety profile of the DEX implants was acceptable, with manageable increases in intraocular pressure and a higher incidence of cataract-related adverse events in treated patients, but only a small percentage required surgical intervention.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Three-year, randomized, sham-controlled trial of dexamethasone intravitreal implant in patients with diabetic macular edema. Boyer, DS., Yoon, YH., Belfort, R., et al.[2022]

The Surodex dexamethasone drug delivery system was found to be as effective as traditional dexamethasone 0.1% eyedrops in managing inflammation after cataract surgery, based on a study involving 19 patients.

Both treatment methods demonstrated a similar safety profile, with no severe adverse events reported, indicating that Surodex is a viable alternative to eyedrops for post-surgical inflammation control.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of a dexamethasone anterior segment drug delivery system in patients after phacoemulsification. Wadood, AC., Armbrecht, AM., Aspinall, PA., et al.[2013]

Topical dexamethasone ophthalmic suspension OCS-01 significantly reduced central macular thickness in patients with diabetic macular edema (DME) compared to a vehicle, with a mean decrease of -53.6 μm versus -16.8 μm after 12 weeks (p = 0.0115).

While the improvement in visual acuity (measured by ETDRS letter score) was not statistically significant overall, patients with lower baseline vision (≤65 letters) showed a notable improvement of +3.8 letters with OCS-01 compared to +0.9 letters with the vehicle, suggesting targeted efficacy in this subgroup.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topical treatment of diabetic macular edema using dexamethasone ophthalmic suspension: A randomized, double-masked, vehicle-controlled study. Stefansson, E., Loftsson, T., Larsen, M., et al.[2023]

In a study of 78 patients with diabetic macular edema, a single intravitreal implant of dexamethasone (Ozurdex®) led to a significant reduction in central retinal thickness by an average of 145.2 µm and an improvement in best-corrected visual acuity by 6.7 letters after 6 months.

The treatment was generally safe, with only 11.7% of patients experiencing increased intraocular pressure, all of which were effectively managed with topical treatment, and no serious complications like glaucoma or endophthalmitis reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Intravitreal Dexamethasone Implant in Patients with Diabetic Macular Edema. Guigou, S., Pommier, S., Meyer, F., et al.[2022]

A 75-year-old woman with macular edema due to branch vein occlusion showed significant improvement in visual acuity and macular thickness after receiving 4 consecutive injections of the Ozurdex implant over 17 months.

The treatment demonstrated quick efficacy and good tolerance, with no signs of reduced effectiveness (tachyphylaxis) after multiple injections, suggesting that Ozurdex can be safely administered beyond the previously studied limit of 3 injections.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Repeated treatment for macular edema in vein occlusion by intravitreal implant of dexamethasone. Matonti, F., Hoffart, L., Baeteman, C., et al.[2021]

The sustained-release dexamethasone intravitreal implant (Ozurdex(®)) offers a new treatment option for macular edema, which is a major cause of vision loss in conditions like retinal vein occlusion and diabetic retinopathy.

Clinical studies suggest that Ozurdex(®) provides controlled drug release over a long period, potentially leading to better efficacy and a lower rate of adverse events compared to traditional treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical applications of the sustained-release dexamethasone implant for treatment of macular edema. Herrero-Vanrell, R., Cardillo, JA., Kuppermann, BD.[2021]

The use of slow-release dexamethasone implants (Ozurdex®) significantly improved visual acuity and reduced central macular thickness in patients with macular edema due to retinal vein occlusion, with notable improvements observed within 2 months after the first implant.

Despite the initial success, 91% of patients experienced a recurrence of macular edema within 5 months, necessitating a second implant, and some patients experienced manageable increases in intraocular pressure, indicating the need for monitoring and potential treatment for elevated pressure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of macular edema associated with retinal vein occlusion using sustained-release dexamethasone implants in a clinical setting. Merkoudis, N., Granstam, E.[2017]

Citations

1. investors.oculis.com investors.oculis.com/news-releases/news-release-details/oculis-present-clinical-trial-results-diabetic-macular-edema-and

Oculis to Present Clinical Trial Results in Diabetic Macular ... The results highlight the potential of OCS-01 to benefit various DME patient profiles, including phakic and pseudophakic patients, as well as ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35848336/

Topical treatment of diabetic macular edema using ... Conclusion: Topical OCS-01 was significantly more effective than vehicle in improving central macular thickness in patients with DME. Visual ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05608837

Multicenter Study on the Efficacy and Safety of OCS-01 in ... Treatment of macular edema remains a challenge as the condition may persist for several months and may lead to irreversible changes in the eye and poor vision.

4. retinalphysician.com retinalphysician.com/issues/2025/september/euretina3/

Initial DIAMOND Results Support OCS-01 Efficacy Data on patients with DME, reported at Euretina, show BCVA gains and improvement in retinal thickness at 12 weeks. Retinal Physician September 4 ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0149291822003782

OCS-01 (Novel Topical Dexamethasone Formulation) in ... OCS-01 once daily and BID were more effective than vehicle and well tolerated in the treatment of inflammation and pain after cataract surgery.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05608837

Multicenter Study on the Efficacy and Safety of OCS-01 in ... Treatment of macular edema remains a challenge as the condition may persist for several months and may lead to irreversible changes in the eye and poor vision.

7. oculis.com oculis.com/our-areas-of-focus/post-ocular-surgery/

OCS-01: Eye Pain After Cataract Surgery: Post-Ocular Relief Furthermore, OCS-01 was well tolerated with a favorable safety profile. The data is consistent with the Phase 2 SKYGGN trial for the same indication. If ...