NCT01783535Active, not recruitingPhase 2
Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma
Source: ClinicalTrials.gov · Last updated 29 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 174 (actual)
- First submitted
- 31 Jan 2013
- Start date
- 19 Jun 2013
- Primary completion date
- 17 Mar 2023
- Lead sponsor
- St. Jude Children's Research Hospital
- Diseases
- Childhood cancers
- NCT number
- NCT01783535
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 174
- From 19 Jun 2013 to the primary completion date, 17 Mar 2023
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previously treated participants.
Other illness
- Presence of metastatic disease or gross (residual) orbital involvement
- Participants must not have an invasive infection at time of protocol entry.
Other
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
Who the study is looking for
- Newly diagnosed, untreated intraocular retinoblastoma. Participants previously diagnosed with unilateral retinoblastoma treated surgically, with focal therapy or needing chemotherapy who develop asynchronous involvement of the contralateral eye, or patients with unilateral retinoblastoma treated only with enucleation or focal therapy who develop asynchronous involvement of the contralateral eye, will be eligible for study.
- ECOG Performance Score must be ≤ 2 within two weeks prior to registration.
- Participants must have an adequate liver function, as defined by bilirubin ≤ to 3X upper limit of normal (ULN), and SGOT and SGPT ≤ to 3X ULN.
- Participants must have adequate renal function as defined by serum creatinine ≤ to 3X ULN for age.
- Legal guardians must sign an informed consent indicating that they are aware of this study, the possible benefits, and toxic side effects. Legal guardians will be given a signed copy of the consent form.
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.