OCS-01 for Macular Edema

(LEOPARD Trial)

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

28 Participants Needed

Trial Summary

What is the purpose of this trial?

The goal of the LEOPARD clinical trial is to investigate a new kind of steroid eye drops, OCS-01. Macular edema is a condition in which there is collection of fluid (edema) in the back of the eye (Macula) and it can lead to severe loss of vision. Among other causes, macular edema can happen because of a disease of the eye called Uveitis, and also after eye surgery. 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. In the LEOPARD study the investigators wish to see how safe is the study drug (OCS-01) and how well it works, in resolving the fluid collection in the eye in patients with Uveitis or in patients who have had eye surgery. Participants will undergo detailed eye exam, and record their eye and medical history to see what their disease status is and if they can be included in the study based on the study criteria. If included, they will take the study drug OCS-01 in different doses for 24 weeks. During the study period, they will have regular eye exams to ensure their safety and to assess the usefulness of the study drug.

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.

What data supports the idea that OCS-01 for Macular Edema is an effective treatment?

The available research shows that OCS-01, a topical dexamethasone ophthalmic suspension, is being evaluated for its effectiveness in treating diabetic macular edema. However, the research does not provide specific data or outcomes for OCS-01's effectiveness. In comparison, other treatments like the sustained-release dexamethasone implant (Ozurdex) have shown promise in treating macular edema from various causes, including diabetic retinopathy and retinal vein occlusion, by providing controlled drug release and potentially fewer side effects. This suggests that while OCS-01 is under investigation, other dexamethasone-based treatments have demonstrated effectiveness in clinical settings. ^{1 2 3 4 5}

What safety data is available for OCS-01 treatment?

The safety data for OCS-01, a novel topical dexamethasone formulation, has been evaluated in studies for inflammation and pain post-cataract surgery and diabetic macular edema. The study titled 'OCS-01 (Novel Topical Dexamethasone Formulation) in Inflammation and Pain Post Cataract Surgery' compared OCS-01 with a vehicle for safety and efficacy. Another study, 'Topical treatment of diabetic macular edema using dexamethasone ophthalmic suspension,' evaluated OCS-01 in diabetic macular edema. These studies provide insights into the safety profile of OCS-01 in different ophthalmic conditions. ^{2 5 6 7 8}

Is the drug OCS-01 a promising treatment for macular edema?

Yes, OCS-01 is a promising treatment for macular edema. It is a new form of the drug dexamethasone, which is used to reduce swelling in the eye. This drug can be applied as eye drops, making it easier to use compared to other treatments that require injections. This could help people with macular edema, a condition that can cause vision loss, by providing a more convenient and effective way to manage their condition. ^{1 2 3 5 6}

Research Team

Quan D Nguyen, MD, MSc

Principal Investigator

Stanford University

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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 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]

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]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Repeated treatment for macular edema in vein occlusion by intravitreal implant of dexamethasone. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Topical treatment of diabetic macular edema using dexamethasone ophthalmic suspension: A randomized, double-masked, vehicle-controlled study. [2023]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Clinical applications of the sustained-release dexamethasone implant for treatment of macular edema. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

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

5. United States pubmed.ncbi.nlm.nih.gov

Safety and efficacy of a dexamethasone anterior segment drug delivery system in patients after phacoemulsification. [2013]

6. United States 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. [2022]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Intravitreal Dexamethasone Implant in Patients with Diabetic Macular Edema. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Three-year, randomized, sham-controlled trial of dexamethasone intravitreal implant in patients with diabetic macular edema. [2022]