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Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Phase 2
18 Years
Open (Enrolling)

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Trial Information

Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Delivering the chemotherapeutic agent in the arterial system through the ophthalmic artery
transforms the treatment of retinoblastoma from systemic chemotherapy to local chemotherapy.
Administration of the drug directly to the targeted site thus avoids the complications and
adverse events associated with toxicity from systemic, rather than local, chemotherapy.

Inclusion Criteria:

- Patients newborn to 18 years old.

- Patients with intraocular retinoblastoma, unilateral or bilateral, who would be
treated either by systemic chemotherapy, EBR, or enucleation would be considered for
this study.

Exclusion Criteria:

- Patients over the age of 18.

- Patients with small, localized intraocular Rb amenable to focal therapy (laser or

- Patients with extraocular disease evident on MRI (extension into the optic nerve),
massive choroidal/uveal invasion (grade IIC or IID per ARET0332) or disease outside
the globe evident on MRI or physical examination.

- Documented hypercoagulable disorders or vasculopathies.

- Laboratory exclusion criteria: GFR < 60 mL/min/1.73 m2

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of patients who complete therapy without the need for additional treatment including systemic chemotherapy, external beam radiation, or enucleation.

Outcome Description:

The primary objective of this study is to show that intra-arterial delivery of the chemotherapeutic agent is successful in treating intraocular retinoblastoma, defined as avoiding systemic chemotherapy, external beam radiation, and enucleation.

Outcome Time Frame:

Within the first six months after the initial treatment.

Safety Issue:


Principal Investigator

Monica Pearl, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

The Johns Hopkins Hospital


United States: Food and Drug Administration

Study ID:




Start Date:

March 2011

Completion Date:

August 2021

Related Keywords:

  • Retinoblastoma
  • Retinoblastoma
  • intra-arterial chemotherapy
  • angiography
  • Retinoblastoma



The Johns Hopkins HospitalBaltimore, Maryland  21205