Intravitreal Melphalan for Intraocular Retinoblastoma
Children's Oncology Group
Summary
This phase II trial tests the safety and side effects of adding melphalan (by injecting it into the eye) to standard chemotherapy in early treatment of patients with retinoblastoma (RB). RB is a type of cancer that forms in the tissues of the retina (the light-sensitive layers of nerve tissue at the back of the eye). It may be hereditary or nonhereditary (sporadic). RB is considered harder to treat (higher risk) when there are vitreous seeds present. Vitreous seeds are RB tumors in the jelly-like fluid of the eye (called the vitreous humor). The term, risk, refers to the chance of the cancer not responding to treatment or coming back after treatment. Melphalan is in a class of medications called alkylating agents. It may kill cancer cells by damaging their deoxyribonucleic acid (DNA) and stopping them from dividing. Other chemotherapy drugs given during this trial include carboplatin, vincristine, and etoposide. Carboplatin is in a class of medications known as platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin, but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing the growth of cancer cells. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Adding melphalan to standard chemotherapy early in treatment may improve the ability to treat vitreous seeds and may be better than standard chemotherapy alone in treating retinoblastoma.
Description
PRIMARY OBJECTIVE: I. To determine the feasibility of administering intravitreal melphalan by cycle 6 when given in combination with systemic carboplatin, vincristine, and etoposide (CVE) for the treatment of Group D retinoblastoma with vitreous seeding. SECONDARY OBJECTIVES: I. To determine the safety and toxicity profile associated with intravitreal melphalan in combination with systemic CVE for the treatment of Group D retinoblastoma with vitreous seeding. II. To evaluate the efficacy of intravitreal melphalan in conjunction with systemic chemotherapy in Group D intraocular retinoblasto…
Eligibility
- Age range
- Up to 18 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Patient must be \< 18 years of age at enrollment * Patient must have newly diagnosed intraocular (localized) retinoblastoma and meet one of the following criteria: * Unilateral Group D retinoblastoma with vitreous seeding; OR * Bilateral retinoblastoma with worst eye Group D, with vitreous seeding present and the contralateral eye is Group A-C; OR * Bilateral Group D retinoblastoma with at least one eye with vitreous seeding; OR * Bilateral retinoblastoma with one Group D eye with vitreous seeding and one Group E eye where the Group E eye has been enucleated pri…
Interventions
- ProcedureBiospecimen Collection
Undergo aqueous humor, tissue, and blood sample collection
- DrugCarboplatin
Given IV
- DrugEtoposide
Given IV
- ProcedureExamination Under Anesthesia
Undergo imaging of the eye during EUA
- ProcedureMagnetic Resonance Imaging
Undergo MRI
- DrugMelphalan
Given I-VITRE
- ProcedureUltrasound Biomicroscopy
Undergo UBM during EUA
Locations (20)
- Children's Hospital of AlabamaBirmingham, Alabama
- Children's Hospital Los AngelesLos Angeles, California
- Lucile Packard Children's Hospital Stanford UniversityPalo Alto, California
- Children's Hospital ColoradoAurora, Colorado
- Children's Healthcare of Atlanta - Arthur M Blank HospitalAtlanta, Georgia
- C S Mott Children's HospitalAnn Arbor, Michigan