Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Johns Hopkins Medicine

Status and phase

Terminated

Phase 2

Conditions

Retinoblastoma

Treatments

Drug: Melphalan hydrochloride

Study type

Interventional

Funder types

Other

Identifiers

NCT01293539

J1071

NA_00040637 (Other Identifier)

Details and patient eligibility

About

The purpose of this study is to show that chemotherapy delivered directly through the artery supplying the eye (ophthalmic artery) to patients with retinoblastoma is a safe and effective treatment alternative to conventional systemic chemotherapy, external beam radiation, and surgical removal of the eye.

Full description

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.

Enrollment

10 patients

Sex

All

Ages

Under 18 years old

Volunteers

No Healthy Volunteers

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 cryotherapy).
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