Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma
Condition: Unilateral Retinoblastoma
MeSH terms: Retinoblastoma
Intervention: Melphalan
Intervention description: Given IA
Intervention MeSH terms: Melphalan; Mechlorethamine; Nitrogen Mustard Compounds
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 14.0
Sex: All
Minimum age: 6 Months
Healthy volunteers: No

Summary:

      This pilot clinical trial studies whether unilateral group D retinoblastoma, or
      retinoblastoma affecting one eye that has spread to the inner jelly like part of the eye, can
      be treated with a new technique for delivering chemotherapy directly into the blood vessel
      that supplies the affected eye. This new technique is called intra-arterial injection. Giving
      melphalan via intra-arterial injection may make it less likely that children will need
      surgery to remove the eye and may reduce the amount of treatment side effects.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Newly diagnosed patients with unilateral group D retinoblastoma

          -  Magnetic resonance imaging (MRI) (or computed tomography [CT] if MRI is not available)
             of the brain must be performed within 14 days prior to study entry

          -  Diagnostic examination under anesthesia (EUA) must be performed within 14 days prior
             to study entry

          -  Rapid central review confirmation of group D disease based on RetCam images from
             diagnostic EUA must be obtained before starting treatment

          -  Patients must have a performance status corresponding to Eastern Cooperative Oncology
             Group (ECOG) scores of 0, 1 or 2

          -  Patients must have a life expectancy of >= 8 weeks

          -  Patients must have adequate renal function, defined as:

               -  Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
                  mL/min/1.73 m^2 or

               -  A serum creatinine based on age/gender as follows:

                    -  1 month to < 6 months: 0.4 mg/dL

                    -  6 months to < 1 year: 0.5 mg/dL

                    -  1 to < 2 years: 0.6 mg/dL

                    -  2 to < 6 years: 0.8 mg/dL

                    -  6 to < 10 years: 1 mg/dL

                    -  10 to < 13 years: 1.2 mg/dL

                    -  13 to < 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)

                    -  >= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)

          -  Total bilirubin =< 1.5 x upper limit of normal (ULN) for age

          -  Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x
             upper limit of normal (ULN) for age

        Exclusion Criteria:

          -  Patients with bilateral disease

          -  Unilateral retinoblastoma with group A, B, C, or E eyes

          -  Prior chemotherapy or radiation therapy for this disease (laser and cryotherapy are
             allowed and are not considered exclusion criteria)

          -  Clinical or neuroimaging evidence of extraocular disease or orbital optic nerve
             involvement