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No enrollment and competing studies
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The investigators are testing subtenon carboplatin in combination with vincristine and topotecan given by vein in the hopes of finding a drug combination that may be effective against retinoblastoma that has come back (recurrent) or is resistant to other treatment (refractory).
The goals of this study are:
In this study, the investigators are also testing a new experimental way of giving carboplatin "subtenon carboplatin". The carboplatin will be given directly in the eye through a needle placed under the covering of the eye. This is to try to get more carboplatin to the retinoblastoma inside the eye.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Topotecan/Vincristine with subtenon Carboplatin | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| subtenon Carboplatin | Drug | Available in 50mg, 150mg, 450 mg, 600mg vials. It is provided as a premixed aqueous solution or lyophilized powder for injection given on day 1/week 10 (cycles 1-3). |
| Measure | Description | Time Frame |
|---|---|---|
| To estimate the event-free survival, where an event is defined at the ocular level as the need for non-protocol therapy defined as additional non-protocol chemotherapy, external beam radiation, or enucleation. | at 1 year | |
| To estimate the patterns of response, and response rate of locally refractory retinoblastoma to combination intravenous and peri-ocular chemotherapy. | at 1 yr |
| Measure | Description | Time Frame |
|---|---|---|
| To describe the toxicities associated with the proposed regimen. | at 1 yr | |
| To describe the histologic findings in the eyes ultimately requiring enucleation. | at 1 yr | |
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Inclusion Criteria:
Age: ≤ 10 years of age
Diagnosis: Patients must have a history of bilateral Retinoblastoma AND Recurrent/Refractory Intra-Ocular Retinoblastoma considered not amenable to cure with local therapies alone (photocoagulation with argon laser, cryotherapy, transpupillary thermotherapy, radioactive plaque therapy).
Therapeutic Options: Patient's disease status is one for which there are no known options proven to provide a high chance for ocular salvage or cure other than external beam radiation or enucleation.
Remaining visual function in target eye (s) is required
Life Expectancy of > 8 weeks
Lansky ≥ 50
Prior Therapy: Patients must have local relapsed/refractory disease after receiving standard upfront therapy involving at least one chemotherapeutic regimen. There is no limit to prior chemotherapeutic regimens permitted. Patients may have received previous intravenous carboplatin, but may not have received prior subtenon carboplatin or intravenous topotecan.
Patients must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study, as described below:
Radiation therapy: Four weeks must have elapsed since external beam radiation therapy, if given.
Adequate Bone Marrow Function Defined as:
Adequate Renal Function Defined as:
Adequate Liver Function Defined As:
Exclusion Criteria:
Extra-ocular retinoblastoma
Asynchronous involvement of the contralateral eye, previously untreated
Uncontrolled infection at time of protocol entry
Concomitant Medications:
Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
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| Name | Affiliation | Role |
|---|---|---|
| James Geller, MD | Children's Hospital Medical Center, Cincinnati | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
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| Topotecan Hydrochloride | Drug | Each vial contains topotecan hydrochloride equivalent to 4 mg of topotecan as free base. Given intravenously on days 1-5 of each week (cycles 1-6). |
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| Vincristine | Drug | Each vial contains vincristine sulfate, 1 mg; mannitol, 100 mg; sterile water for injection; Acetic acid and sodium acetate are added for pH control. Given intravenously on day 1/week 1 (cycles 1-6). |
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| Filgrastim | Drug | Supplied as a clear solution in 300 mcg/ml 1 ml or 1.6 ml vials and prefilled syringes containing 300mcg/0.5mL or 480mcg/0.8mL. Given intravenously on day 6 of each week (cycles 1-6). |
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| To describe the visual acuity of patients with advanced intraocular retinoblastoma before and after the proposed therapy. |
| at 1 yr |
| To describe primary visual cortex function in patients with retinoblastoma via functional Magnetic Resonance Imaging (fMRI) technique both before and after the proposed therapy. | at 1 yr |
| ID | Term |
|---|---|
| D012175 | Retinoblastoma |
| ID | Term |
|---|---|
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
| D019572 | Retinal Neoplasms |
| D005134 | Eye Neoplasms |
| D009371 | Neoplasms by Site |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012164 | Retinal Diseases |
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| ID | Term |
|---|---|
| D016190 | Carboplatin |
| D019772 | Topotecan |
| D014750 | Vincristine |
| D000069585 | Filgrastim |
| ID | Term |
|---|---|
| D056831 | Coordination Complexes |
| D009930 | Organic Chemicals |
| D002166 | Camptothecin |
| D000470 | Alkaloids |
| D006571 | Heterocyclic Compounds |
| D014748 | Vinca Alkaloids |
| D046948 | Secologanin Tryptamine Alkaloids |
| D026121 | Indole Alkaloids |
| D007211 | Indoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D054836 | Indolizidines |
| D007212 | Indolizines |
| D016179 | Granulocyte Colony-Stimulating Factor |
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
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