for people ages 6 months and up (full criteria)
at San Francisco, California and other locations
study started



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.

Official Title

A Multi-institutional Feasibility Study of Intra-arterial Chemotherapy Given in the Ophthalmic Artery of Children With Retinoblastoma



  1. To study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group D retinoblastoma, who would otherwise be considered for enucleation.


  1. To estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group D disease.

II. To evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy.

III. To evaluate vision outcomes in children treated with intra-arterial chemotherapy.

IV. To monitor the rate of the development of metastatic disease while on protocol therapy.


  1. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression.


Patients receive melphalan intra-arterially (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.


Unilateral Retinoblastoma, Retinoblastoma, Melphalan, Mechlorethamine, Nitrogen Mustard Compounds


You can join if…

Open to people ages 6 months and up

  • 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 m2 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

You CAN'T join if...

  • 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


  • UCSF Medical Center-Parnassus
    San Francisco California 94143 United States
  • UCSF Medical Center-Mission Bay
    San Francisco California 94158 United States


in progress, not accepting new patients
Start Date
Children's Oncology Group
Phase 1 Eye Cancer Research Study
Study Type
About 14 people participating
Last Updated