NCT04156347

Topotecan Episcleral Plaque for Treatment of Retinoblastoma

A Phase I Study of Sequestered Transscleral, Controlled-Release Topotecan Delivered From an Episcleral Reservoir in Retinoblastoma Eyes

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Targeted Therapy Technologies, LLC · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This single-arm, non-randomized, dose escalation phase I clinical trial will assess primarily the safety and secondarily the efficacy of episcleral topotecan in patients with active de novo or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy.

Detailed description

Retinoblastoma is the most common pediatric malignant intraocular tumor and originates from the retina. Treatment of eyes with advanced intraocular retinoblastoma remains a challenge. The historic standard of care for patients with unilateral disease is enucleation and for those with bilateral disease, a variety of modalities have been tried. These include radiation therapy, systemic chemotherapy, periocular administration of chemotherapy, selective intra-arterial chemotherapy, and intravitreal chemotherapy. Unfortunately, all of these modalities are associated with significant morbidity and investigators are looking for new ways to treat these patients either with novel directed drug delivery methods or with new less toxic agents. This study will evaluate the safety and efficacy of topotecan delivered directly to the eye using a novel sustained-release topotecan episcleral delivery system (also referred to as a Chemoplaque) in patients with active de novo or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy. The study intervention involves the insertion and removal of the Chemoplaque, examinations under anaesthesia (EUAs), visits to clinic to monitor for adverse events throughout, and post plaque removal toxicity evaluation. EUAs, clinic visits and laboratory tests are standard of care for retinoblastoma patients.

Conditions

• Retinoblastoma

Interventions

Type	Name	Description
DRUG	Episcleral Topotecan	Transscleral Topotecan

Timeline

• Start date: 2021-06-16
• Primary completion: 2024-10-01
• Completion: 2024-10-14
• First posted: 2019-11-07
• Last updated: 2023-10-31

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study