CBT-004 for Pinguecula

Overseen By Van Dinh, MBA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Cloudbreak Therapeutics, LLC

Must not be taking: Topical ophthalmic medications

Disqualifiers: Uncontrolled systemic disease, Active ocular disease, Ocular surgery, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new eye treatment called CBT-004 for pinguecula, which are yellowish bumps on the eye that can become red and irritating. Researchers seek to determine the effectiveness of different strengths of CBT-004 in reducing these symptoms compared to a placebo (a fake treatment). Individuals experiencing bothersome pinguecula with noticeable redness may be suitable candidates for this study. Participants will use eye drops in one eye for four weeks, followed by an observation period of another four weeks. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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

You will need to stop using any topical eye medications in the study eye at least 2 weeks before the trial starts (4 weeks for Restasis® or Xiidra®). Artificial tears are allowed until 7 days before the trial and should not be used during the treatment phase.

Is there any evidence suggesting that CBT-004 is likely to be safe for humans?

Research has shown that CBT-004 is safe based on previous studies. This treatment was tested on individuals with vascularized pinguecula, yielding positive results. The studies found that patients tolerated CBT-004 well, with no major safety issues.

Additionally, the research indicated that CBT-004 helped reduce eye redness and other symptoms without causing serious side effects. These findings suggest the treatment is safe for individuals. While encouraging, it is important to discuss potential risks and benefits with a healthcare provider before joining a clinical trial. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for pinguecula, which typically involve lubricating eye drops or anti-inflammatory medications, CBT-004 is being developed as a potential new therapy with a unique formulation. Researchers are excited about CBT-004 because it employs a novel compound designed specifically to target the underlying causes of pinguecula, rather than just alleviating symptoms. This investigational treatment is being tested in two different concentrations, 0.05% and 0.075%, which might offer tailored options for patients with varying severity of the condition. By directly addressing the root of the problem, CBT-004 has the potential to provide more effective and lasting relief for those suffering from pinguecula.

What evidence suggests that CBT-004 might be an effective treatment for pinguecula?

Research has shown that CBT-004 yields promising results for treating vascularized pinguecula, which causes redness in the white part of the eye. In earlier studies, patients using CBT-004 experienced a noticeable decrease in eye redness by Day 28. The treatment also alleviated symptoms reported by patients. These improvements suggest that CBT-004 could effectively reduce eye redness and discomfort for those with this condition. Studies have consistently shown positive results, making CBT-004 a potential option for people affected by this eye issue. Participants in this trial will receive either 0.05% CBT-004, 0.075% CBT-004, or a placebo comparator to evaluate the treatment's effectiveness. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with a pinguecula, which is an eye condition that causes a yellowish bump on the white of the eye. Participants must have noticeable blood vessel growth and experience discomfort from their condition. They need to be able to follow the study's procedures and give written consent.

Inclusion Criteria

I have a score of 2 or more on the eye discomfort questionnaire.

I have a pinguecula with significant blood vessel growth.

I can give written consent and commit to all study assessments for the entire duration of the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive one drop of the assigned study medication in the study eye TID for 4 weeks

4 weeks

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CBT-004

Trial Overview The trial tests CBT-004, a new treatment for pinguecula. It compares CBT-004 with a placebo (Vehicle) in different groups without anyone knowing who gets what (double-masked). The treatment involves putting one drop into the affected eye three times daily for four weeks.

How Is the Trial Designed?

Group I: 0.075% CBT-004 Experimental Treatment 1 Intervention

Group II: 0.05% CBT-004 Experimental Treatment 1 Intervention

Group III: CBT-004 Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Cloudbreak Therapeutics, LLC

Lead Sponsor

Trials

6

Recruited

1,000+

Published Research Related to This Trial

The novel paclitaxel microemulsion demonstrated similar pharmacokinetics and antitumor efficacy compared to the standard paclitaxel injection, while significantly reducing allergic reactions associated with Cremophor EL.

In animal studies, the microemulsion did not cause harmful effects like hemolysis or erythrocyte agglutination, indicating a safer profile for patients compared to the conventional injection.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A novel paclitaxel microemulsion containing a reduced amount of Cremophor EL: pharmacokinetics, biodistribution, and in vivo antitumor efficacy and safety. Wang, Y., Wu, KC., Zhao, BX., et al.[2021]

The CF-PTX formulation, which is free from Cremophor EL, showed significantly higher intracellular uptake of paclitaxel (85.4%) compared to the conventional formulation (19.8%), indicating improved delivery of the drug into cancer cells.

In an in vivo study using mice with Ehrlich ascites carcinoma, CF-PTX reduced tumor volume by 72.5%, outperforming the marketed formulation's 58.6% reduction, and also demonstrated a longer mean residence time and elimination half-life, suggesting enhanced efficacy and sustained action.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anti-cancer, pharmacokinetic and biodistribution studies of cremophor el free alternative paclitaxel formulation. Jain, SK., Utreja, P., Tiwary, AK., et al.[2019]

The study found that the excipient Cremophor® EL (CrEL) in the conventional paclitaxel formulation (Taxol®) causes red blood cell (RBC) lysis and other harmful effects, which could lead to complications for patients, especially those with blood disorders.

In contrast, the nanoparticle albumin-bound paclitaxel (Abraxane®) did not cause these adverse effects on RBCs, highlighting that the choice of excipients can significantly impact the safety and efficacy of cancer treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Taxol(®)-induced phosphatidylserine exposure and microvesicle formation in red blood cells is mediated by its vehicle Cremophor(®) EL. Vader, P., Fens, MH., Sachini, N., et al.[2015]

Polyethoxylated surfactants, specifically Cremophor-EL and Tween-80, have been shown to activate the complement system in human serum and plasma, which may lead to side effects in patients.

The activation of the complement system by these surfactants is linked to therapeutic side effects like acute hypersensitivity and systemic immunostimulation, suggesting that their use in intravenous nanomedicines could pose immunological risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Complement activation by polyethoxylated pharmaceutical surfactants: Cremophor-EL, Tween-80 and Tween-20. Weiszhár, Z., Czúcz, J., Révész, C., et al.[2022]

Radiographic contrast media (RCM) such as bilignost, triombrast, and iodamide-380 activate the complement system in rats, indicating a potential immune response triggered by these substances.

The degree of complement activation varies with the type and dose of RCM, with triombrast showing the highest effect, followed by iodamide and bilignost, suggesting that different RCMs may have varying impacts on immune function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Effect of x-ray contrast substances on the complement system of rats]. Sergeev, PV., Kul'berg, AIa., Shimanovskiĭ, NL., et al.[2015]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04884256

Safety and Efficacy Study of CBT-004 in Patients With ... Active ocular disease other than pinguecula that may confound the study data, including but not limited to severe dry eye disease, pterygium, uncontrolled ...

2. touchophthalmology.com touchophthalmology.com/insight/cloudbreak-pharma-announced-positive-topline-results-from-its-phase-2-trial-cbt%E2%80%91004-p-designed-to-treat-vascularized-pinguecula-the-trial-demonstrated-that-c/

Positive phase II data announced for CBT‑004 in ... The trial demonstrated that CBT‑004 met its primary endpoint, showing a statistically significant reduction in conjunctival redness at Day 28 ...

3. ophthalmologytimes.com ophthalmologytimes.com/view/cloudbreak-pharma-reports-positive-phase-2-results-for-cbt-004-in-vascularized-pinguecula

Cloudbreak Pharma reports positive phase 2 results for ... CBT-004 demonstrated significant improvements in conjunctival hyperemia and patient-reported symptoms in a phase 2 trial for vascularized ...

4. cloudbreakpharma.com cloudbreakpharma.com/clinical-trial/

Clinical Trial The objective of the study is to evaluate the safety, tolerability and efficacy of a CBT-004 ophthalmic emulsion when instilled twice daily (BID) for 28 days in ...

5. businesswire.com businesswire.com/news/home/20250717640231/en/Cloudbreak-Pharma-Inc.-Announces-Positive-Phase-2-Results-for-CBT-004-in-Patients-with-Vascularized-Pinguecula

Cloudbreak Pharma Inc. Announces Positive Phase 2 ... Novel preservative-free CBT-004 eye drop formulation demonstrates statistically significant improvements in conjunctival hyperemia and ...

6. cloudbreakpharma.com cloudbreakpharma.com/cloudbreak-pharma-inc-s-cbt-004-indicated-for-the-treatment-of-vascularised-pinguecula-has-completed-phase-ii-clinical-trials-in-the-united-states/

Cloudbreak Pharma Inc.'s CBT-004 indicated for the ... Preliminary data analysis demonstrateed excellent ocular safety profile. The primary efficacy endpoint of this study was conjunctival hyperemia, which met the ...

7. healio.com healio.com/news/ophthalmology/20250722/phase-2-trial-preservativefree-eye-drop-promising-for-vascularized-pinguecula-hyperemia

Eye drop promising for vascularized pinguecula Rapid onset of improvement was observed as early as day 7, with benefits from the highest concentration of CBT-004 persisting through day 28.