Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma
Status:
Recruiting
Trial end date:
2022-06-01
Target enrollment:
Participant gender:
Summary
The primary objective of this protocol is to evaluate the response rate of bilateral disease
participants who have at least one eye with advanced intra-ocular retinoblastoma (stratum B)
using upfront therapy with chemotherapy delivered directly to the eye. The main biology
objective is to improve our understanding of the biology and tumorigenesis (how tumor
develops) of retinoblastoma when biology specimens are available. As clinicians, the primary
goal of the investigators for children with retinoblastoma is to provide optimal therapy
using multiple treatment approaches [chemotherapy (into the vein and directly into membrane
of eyeball), cryotherapy (freeze and destroy tumor), thermotherapy (laser or heat to destroy
tumor), radiation therapy, and surgical removal of eye if needed) in an attempt to preserve
the eye and vision whenever possible, while still curing the disease. Therefore, all children
with non-metastatic retinoblastoma at St. Jude will be offered enrollment on this study.
PRIMARY OBJECTIVE:
- To evaluate the response (complete + partial response) rate of bilateral disease
participants who have at least one eye with advanced intraocular retinoblastoma (Stratum
B) to two upfront courses of therapy consisting of subconjunctival carboplatin and
systemic topotecan.
SECONDARY OBJECTIVES:
- To evaluate the ocular survival of eyes and event-free survival of participants by
strata.
- To prospectively analyze intraocular disease tissue for participants with at least one
eye undergoing enucleation in order to identify the mechanism of RB1 bi-allelic
inactivation. Participants may undergo upfront enucleation (due to advanced disease at
diagnosis) or may receive enucleation due to progressive disease during protocol
therapy.