NCT01783535

Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma

Active, enrolled Phase 2 Last updated 11 April 2024

What this trial tests

Phase 2 trial testing vincristine in Retinoblastoma in 174 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

19 June 2013

Primary endpoint
17 March 2023

1 January 2028

Quick facts

Lead sponsor	St. Jude Children's Research Hospital
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	174
Start date	19 June 2013
Primary completion	17 March 2023
Estimated completion	1 January 2028
Sites	1 location across United States

Drugs / interventions tested

• vincristine (vincristine) — full drug profile →
• topotecan (topotecan) — full drug profile →
• filgrastim
• PEG-filgrastim
• carboplatin (Carboplatin) — full drug profile →
• focal therapy
• etoposide
• cyclophosphamide (cyclophosphamide) — full drug profile →
• MESNA (MESNA) — full drug profile →
• doxorubicin
• enucleation
• external beam radiation or proton beam radiation

Conditions studied

• Retinoblastoma — all drugs for Retinoblastoma →

Sponsor

St. Jude Children's Research Hospital

Who can join

Eligibility, any sex, with Retinoblastoma. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Response rate (complete or partial response)
  Time frame: After two upfront courses of chemotherapy (approximately two months after patient enrollment)
  Stratum B patients, those Stratum B patients who had no significant subretinal seeding and received vincristine and topotecan are not evaluable for this primary objective.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Trial Watch: Chemotherapy with immunogenic cell death inducers.
   Vacchelli E, Aranda F, Eggermont A, Galon J, et al · · 2014 · cited 123× · PMID 24800173 · DOI 10.4161/onci.27878
2. Ocular Salvage and Vision Preservation Using a Topotecan-Based Regimen for Advanced Intraocular Retinoblastoma.
   Brennan RC, Qaddoumi I, Mao S, Wu J, et al · · 2017 · cited 33× · PMID 28034080 · DOI 10.1200/jco.2016.69.2996
3. Lessons from Retinoblastoma: Implications for Cancer, Development, Evolution, and Regenerative Medicine.
   Dyer MA. · · 2016 · cited 33× · PMID 27567287 · DOI 10.1016/j.molmed.2016.07.010
4. Nanotechnology for Pediatric Retinoblastoma Therapy.
   Russo E, Spallarossa A, Tasso B, Villa C, et al · · 2022 · cited 18× · PMID 36145308 · DOI 10.3390/ph15091087
5. Retinoblastoma: An update on genetic origin, classification, conventional to next-generation treatment strategies.
   Pareek A, Kumar D, Pareek A, Gupta MM, et al · · 2024 · cited 10× · PMID 38975183 · DOI 10.1016/j.heliyon.2024.e32844
6. Targeting the Platelet-Derived Growth Factor-beta Stimulatory Circuitry to Control Retinoblastoma Seeds.
   Goldsmith ZK, Coppess W, Irvine AS, Yuan K, et al · · 2018 · cited 7× · PMID 30193324 · DOI 10.1167/iovs.18-24359