A Phase 3 interventional study of Combination Chemotherapy plus Intrathecal Topotecan and Higher Dose Combination Chemotherapy plus Intrathecal Topotecan in Unilateral Retinoblastoma, sponsored by Hospital JP Garrahan. Completed at 1 site in Argentina. Open to participants aged 0 Years to 6 Years. Per ClinicalTrials.gov, last updated 2025-04-13.
Sponsored by Hospital JP Garrahan · Phase 3, Interventional, and Treatment
This protocol provides guidelines for the management of non-metastatic unilateral retinoblastoma and introduces an innovative adjuvant therapy for higher risk patients based upon the results of the Grupo de America Latina de Oncologia Pediatrica (GALOP) I study. Conservative therapy will be not protocolized.
Patients with non-metastatic retinoblastoma undergoing enucleation will be staged using the American Joint Committee on Cancer (AJCC), version 8, Tumor, Node, Metastasis (TNM)- H system and the International Retinoblastoma Staging System (IRSS). IRSS stage I patients recognized as higher risk will be assigned for adjuvant therapy. Those with standard risk will not receive adjuvant therapy after enucleation. Higher risk patients are defined as those with pathological retrolaminar optic nerve invasion and-or any degree of scleral invasion (pT3b, pT3c, pT3d). Based on the results of the GALOP I protocol, they will receive a reduced dose adjuvant chemotherapy regimen with 3 cycles of alternating cyclophosphamide, vincristine and idarubicin alternating with another 3 cycles of carboplatin and etoposide. Six doses of intrathecal topotecan will be given. Patients presenting with severe buphthalmia (cT3c-cT3e) will receive neo-adjuvant therapy with the same intensive regimen but including a higher dose of carboplatin as per GALOP I protocol plus intrathecal topotecan and secondary enucleation followed by adjuvant chemotherapy for a total of 8 cycles. Stage II patients (pT4) will receive the same adjuvant regimen plus orbital radiotherapy (45 cGy).
124 studies on the registry are indexed under Retinoblastoma; 23 are open to participants now.
This study's enrollment of 84 is above the median of 30 across 81 interventional studies indexed under Retinoblastoma.
Browse Retinoblastoma studies →Hospital JP Garrahan is the lead sponsor of 4 studies on the registry; 2 are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients with IRSS stage I, pT1, pT2 and pT3 stage will not receive adjuvant therapy
Other: No Adjuvant Therapy
Patients with IRSS stage I, pT3b, pT3c, pT3d will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan
Combination Product: Combination Chemotherapy plus Intrathecal Topotecan
Patients with Stage II (pT4) will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan and orbital radiotherapy
Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy
Patients with buphthalmus (cT3c, cT3e) will receive 2 cycles of neo-adjuvant chemotherapy plus 6 doses of intrathecal topotecan followed by secondary enucleation and 6 cycles of adjuvant chemotherapy.
Combination Product: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan
Drug page: Topotecan
Adjuvant chemotherapy with a reduced dose (compared to GALOP I protocol) of carboplatin
GALOP I-based systemic adjuvant therapy plus intrathecal topotecan
GALOP I-based systemic adjuvant therapy plus intrathecal topotecan and orbital radiotherapy (45 Gy) up to chiasm
Patients will not receive any adjuvant therapy after enucleation of the affected eye.
Rate of extraocular relapses
Number of participants experiencing extraocular relapse
Time frame: 3 years
Evaluation of number of patients experiencing acute, chronic and fatal toxicities
Number of participants experiencing treatment-related adverse effects and mortality as measured by CTCAE v4.0
Time frame: 5 years
Plan to share: No
No publications or documents are linked to this record.
Ran from Jan 2018 to primary completion in Jan 2023, 60 months.
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This study is completed, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Topotecan
Hospital JP Garrahan