NIH R01 · 2024
Retinoblastoma Phase II Expanded Access Clinical Trial
Abstract Retinoblastoma (RB) is a micro-orphan disease intraocular cancer in infants and children (typically diagnosed between birth and 5 years of age). Approximately 250 new cases are diagnosed annually in the United States. Standard of care for retinoblastoma includes removing the affected eye (safest) or attempted salvage of the eye starting with systemic or intra-arterial chemotherapy. Since external beam radiotherapy incurs life-long high risk of second cancers in RB H1 patients carrying the RB1 cancer predisposition gene, it is reserved for last effort to save a last eye. There is no FDA approved intervention for retinoblastoma. Topotecan Chemoplaque (IND #112785) as a single (one)…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.