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



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


PRIMARY OBJECTIVES: I. 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. SECONDARY OBJECTIVES: I. 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. TERTIARY OBJECTIVES: I. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression. OUTLINE: 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
Completion Date
Children's Oncology Group
Study Type
About 14 people participating
Last Updated