Chemoplaque for Retinoblastoma

Recruiting in Palo Alto (17 mi)

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Targeted Therapy Technologies, LLC

Must not be taking: Chemotherapy, Investigational agents

Disqualifiers: Optic nerve invasion, Metastatic retinoblastoma, others

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. This study will investigate the utility of a chemoplaque(s) to salvage eyes involved with retinoblastoma. The goal of the study is to further determine/assess the safety and efficacy and optimal chemotherapy dose for retinoblastoma.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have received chemotherapy or other retinoblastoma treatments within 3 weeks before starting the study treatment.

What data supports the effectiveness of the treatment Chemoplaque for retinoblastoma?

Research shows that combining different chemotherapy drugs, like carboplatin and melphalan, can help treat retinoblastoma by reducing the need for more invasive treatments like eye removal or radiation. This suggests that Chemoplaque, which may include similar components, could be effective in managing retinoblastoma.

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What safety data exists for Chemoplaque (carboplatin) in humans?

Studies have looked at the safety of carboplatin, a component of Chemoplaque, for treating retinoblastoma. These studies have evaluated its use in the eye and found it can be safe, but there is a risk of retinal toxicity (damage to the retina, the light-sensitive layer at the back of the eye) at higher doses.

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Eligibility Criteria

This trial is for children under 8 years old with advanced or recurrent retinoblastoma, potentially facing eye removal. They must have some vision potential in the affected eye and no signs of cancer spread outside the eye. Kids need to be generally healthy with good blood counts and kidney function.

Inclusion Criteria

My white blood cell count is high enough for treatment.

My kidney function, measured by creatinine clearance or GFR, is sufficient.

I am under 8 years old.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Chemoplaque is applied to the eye for sustained delivery of chemotherapy over 8 weeks

8 weeks

4 visits (in-person) on Day 0, Day 28, Day 56, and Day 84

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person) on Day 84

Participant Groups

The study tests a treatment called Chemoplaque (Episcleral Topotecan) for saving eyes from being removed due to retinoblastoma. It aims to find out how safe it is, how well it works, and what's the best dose for treating this type of eye cancer.

Group I: Chemoplaque Experimental Treatment 1 Intervention

Chemoplaque (also referred to as Episcleral Topotecan) treatment will involve single application of the Episcleral Topotecan 1.2 mg dose that delivers sustained delivery for 8 weeks.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

NewYork Presbyterian Morgan Stanley Children's Hospital New York, NY

Who Is Running the Clinical Trial?

Targeted Therapy Technologies, LLC Lead Sponsor

Columbia-New York-Presbyterian Morgan Stanley Children's Hospital Collaborator

Morgan Stanley Children's Hospital Collaborator

References

1. Germany pubmed.ncbi.nlm.nih.gov

Addition of intravitreal carboplatin with melphalan for management of vitreous seeding in retinoblastoma. [2023] To evaluate the efficacy and toxicity of intravitreal carboplatin plus melphalan for the treatment of vitreous seeds in eyes with retinoblastoma (RB).

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

Systemic neoadjuvant chemotherapy for Group B intraocular retinoblastoma (ARET0331): A report from the Children's Oncology Group. [2018] To evaluate a chemoreduction regimen using systemic vincristine and carboplatin (VC) and local ophthalmic therapies to avoid external-beam radiotherapy (EBRT) or enucleation in patients with Group B intraocular retinoblastoma.

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

Ocular Survival Following Intravitreal Melphalan as Adjuvant Treatment for Vitreous Retinoblastoma Seeding. [2023] To evaluate the efficacy of intravitreal chemotherapy for vitreous seeding in patients with retinoblastoma (Rb).

4. England pubmed.ncbi.nlm.nih.gov

Chemoreduction improves eye retention in patients with retinoblastoma: a report from the German Retinoblastoma Reference Centre. [2015] Retinoblastoma is the most common intraocular childhood tumour. Although mortality is low in Western countries, long-term sequelae, including secondary tumours, compromised vision or loss of one or both eyes are common. Chemoreduction combined with focal treatment is currently the leading conservative treatment for retinoblastoma, with success rates of 50-75% reported. We assessed a new chemoreduction protocol using intravenous cyclophosphamide with reduced dose of carboplatin on eye retention in patients with retinoblastoma.

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

Chemoreduction for group E retinoblastoma: comparison of chemoreduction alone versus chemoreduction plus low-dose external radiotherapy in 76 eyes. [2015] To evaluate chemoreduction (CRD) for group E retinoblastoma.

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

Intravitreal Carboplatin as Salvage Treatment for Progressive Vitreous Disease in Retinoblastoma: A Phase I Clinical Trial. [2023] To determine the safety and toxicity profile of intravitreal carboplatin as salvage treatment for retinoblastoma with vitreous disease.

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

A phase I/II study of subconjunctival carboplatin for intraocular retinoblastoma. [2022] To evaluate the efficacy and toxicity of subconjunctival carboplatin for intraocular retinoblastoma.

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

Local carboplatin therapy in transgenic murine retinoblastoma. [2013] To determine the efficacy and toxicity associated with intraocular delivery of carboplatin in the treatment of murine transgenic hereditary retinoblastoma.

9. Czech Republic pubmed.ncbi.nlm.nih.gov

Retinal toxicity after repeated intravitreal carboplatin injection into rabbit eyes. [2015] The aim of this study was to assess retinal toxicity in a rabbit model after carboplatin delivered as repeated transcorneal intravitreal injection, in order to determine the highest possible safe dose for use in human retinoblastoma "seeding" tumor chemotherapy.