Neoplasms

Eye Diseases

All Conditions

Diseases and Abnormalities at or Before Birth

Rare Diseases

Retinoblastoma

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Retinal Neoplasms

Eye Neoplasms

Eye Diseases, Hereditary

Retinal Diseases

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Melphalan

Antineoplastic Agents, Alkylating

Alkylating Agents

Immunosuppressive Agents

Immunologic Factors

Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral.

Drug administered intra-arterially (injection in the artery).

Standard dose:

2.5mg (3-6 month old) 3.0 mg (6-12 month old) 4.0 mg (1-3 year old) 5.0 mg (\>3 years old)

Dose modification: decrease standard dose by 25% if there are signs of toxicity. Increase the dose by 25% if there is inadequate tumor response.

Frequency: 2 treatment cycles at 3-4 week intervals, with a third treatment cycle administered if the tumor requires it.

Dose not to exceed 0.5mg/kg, per treatment cycle.

Melphalan hydrochloride

Monica Pearl, M.D.

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.

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.

Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

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.

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site.

Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; \>3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy.

Intra-arterial Chemotherapy

Age, Categorical

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.

Overall Study

Spots Global Cancer Trial Database for Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

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