Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma

Status: Recruiting
Phase: Phase 3
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Solid Tumors
NCT ID: NCT00554788 (View complete trial on
DFCI Protocol ID: 13-090


This phase III trial is studying the side effects and how well giving combination chemotherapy together with autologous stem cell transplant and/or radiation therapy works in treating young patients with extraocular retinoblastoma. Giving chemotherapy before an autologous stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy and/or autologous stem cell transplant may kill any remaining tumor cells.


Conducting Institutions:
Dana-Farber Cancer Institute, Children's Hospital Boston, Massachusetts General Hospital

Overall PI:
Carlos Rodriguez-Galindo, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:
Eric Grabowski, MD, Massachusetts General Hospital

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,
Massachusetts General Hospital: Cancer Trials Call Center, 877-789-6100

Eligibility Criteria

DISEASE CHARACTERISTICS: - Histologically or cytologically confirmed extraocular retinoblastoma, meeting 1 of the following criteria: - Stage 2 or 3 disease (regional extraocular disease) - Stage 4a disease (disseminated metastatic disease not involving the CNS, including extradural/dural disease without parenchymal or leptomeningeal disease) - Stage 4b disease (CNS disease, including trilateral retinoblastoma) - Extraocular disease includes any of the following: - Orbital disease - Optic nerve involvement at the surgical margin - Regional nodal disease - Overt distant metastatic disease (at sites such as bone, bone marrow, liver, and/or the CNS) PATIENT CHARACTERISTICS: - ANC ≥ 750/μL* - Platelet count ≥ 75,000/μL* (transfusion independent) - Creatinine clearance OR radioisotope glomerular filtration rate ≥ 70 mL/min/1.73 m^2 - Total bilirubin ≤ 1.5 times upper limit of normal (ULN) - AST or ALT < 2.5 times ULN NOTE: *Inadequate ANC and/or platelet count due to bone marrow metastatic disease allowed PRIOR CONCURRENT THERAPY: - No prior chemotherapy or radiotherapy for extraocular retinoblastoma - Prior chemotherapy and/or radiation therapy for intraocular retinoblastoma allowed - No other concurrent anticancer chemotherapy, radiotherapy, or immunomodulating agents (including steroids) - Corticosteroid therapy is allowed only for treatment of increased intracranial pressure in patients with CNS tumors - Dexamethasone should not be prescribed as an anti-emetic
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