Retinoblastoma eye-sparing treatment for young children
Official title: Retinoblastoma Phase II Expanded Access Clinical Trial
Funded at Targeted Therapy Technologies, LLC · NIH-11193975 · Data updated 2026-11-12
A new, targeted eye treatment for young children with retinoblastoma aims to save eyes and reduce radiation exposure by delivering medicine directly into the eye.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Targeted Therapy Technologies, LLC NIH-funded |
| Lab location | 1 site (Somerset, United States) |
| Project start | September 15, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $650,000 |
| NIH project number | 5R01FD008443-02 |
| Project ID | NIH-11193975 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are infants and young children with retinoblastoma who have limited options for eye-salvage and are eligible for intraocular topotecan chemotherapy.
✕Less likely to help
Patients with disease characteristics not suitable for intraocular Chemoplaque or those with contraindications to the therapy may not benefit.
What this research studies
This grant focuses on a phase II expanded-access approach using Topotecan Chemoplaque delivered inside the eye to treat retinoblastoma in infants and young children. The study builds on prior data showing high local response rates and aims to progress to a dose of 1.2 mg for better sustained complete responses. It involves a single-arm regimen administered via chemoplaque implants, with careful patient selection based on age and tumor features to maximize eye salvage. Participants would continue to be monitored for long-term outcomes, including event-free and eye-preserving results, as part of ongoing care.
Why it matters
Potential benefit: If successful, this approach could increase eye preservation and reduce normal tissue exposure for young patients with retinoblastoma.
How similar studies have gone: Early data showed high sustained complete responses with the Chemoplaque approach in refractory RB cases, supporting further development.
Where this research is happening
-
Targeted Therapy Technologies, LLC
Somerset, United States Active
Researchers
- Principal investigator: Carvalho, Ricardo a — Targeted Therapy Technologies, LLC
- Study coordinator: Carvalho, Ricardo a
Related research
Other NIH-funded projects on the same topics.
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Understanding how premalignant retinoblastoma lesions form and progress in children
Researchers are studying how early retinal lesions in kids with RB1 loss become malignant, with the goal of finding ways to prevent retinoblastoma progression.
-
Aqueous humor liquid biopsy to guide prognosis and monitoring in children with retinoblastoma
A liquid test using a small amount of eye fluid could reveal important tumor details to help predict how retinoblastoma in young children will behave and how well treatment is working.
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Parent-of-Origin Effects in Retinoblastoma: Epigenetic Drivers of Tumor Risk and Progression
A study using long-read DNA sequencing to understand how the parent of origin of RB1 gene variants affects retinoblastoma risk and progression, with potential to guide personalized monitoring and treatment.
-
How early retinal changes lead to retinoblastoma
This project looks at how early abnormal cells in the retina form and later turn into retinoblastoma in children, using lab-grown retinal tissue.
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A tiny eye-fluid test to predict and track retinoblastoma in young children
This project uses a very small sample of eye fluid to look for tumor DNA that could help predict how retinoblastoma will behave and to follow treatment in children.
-
Handheld wide-view 3D eye scanner to find tiny retinoblastoma tumors
This project is building a new handheld 3D eye scanner intended to help find very small retinoblastoma tumors early in young children who need screening.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.