New treatment for retinoblastoma in young children

Retinoblastoma Phase II Expanded Access Clinical Trial

['FUNDING_R01'] · TARGETED THERAPY TECHNOLOGIES, LLC · NIH-11105727

This study is testing a new way to treat retinoblastoma, a rare eye cancer in babies and young kids, by using a special device to deliver a chemotherapy drug right to the eye, which could help keep the cancer away without needing more intense treatments like surgery or radiation.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorTARGETED THERAPY TECHNOLOGIES, LLC (nih funded)
Locations1 site (SOMERSET, UNITED STATES)
Trial IDNIH-11105727 on ClinicalTrials.gov

What this research studies

This research focuses on a novel treatment for retinoblastoma, a rare eye cancer affecting infants and young children. The approach involves using a specially designed Chemoplaque to deliver the chemotherapy drug Topotecan directly to the eye, minimizing systemic exposure and potential side effects. The trial aims to evaluate the effectiveness of this treatment in achieving sustained cancer remission without the need for more invasive standard care options, such as eye removal or radiation therapy.

Who could benefit from this research

Good fit: Ideal candidates for this research are infants and young children diagnosed with retinoblastoma who have not responded to standard treatments.

Not a fit: Patients with retinoblastoma who are not eligible for the trial due to age or those who have already undergone extensive treatment may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could provide a safer and more effective treatment option for children with retinoblastoma, potentially preserving their vision and reducing the risk of long-term complications.

How similar studies have performed: Previous research has shown promising results with similar localized chemotherapy delivery methods, indicating potential for success in this novel approach.

Where this research is happening

SOMERSET, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.