Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Conditions
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Retinoblastoma

Intervention / Treatment
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* Drug: Melphalan hydrochloride


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Intraocular Retinoblastoma Patients<br>Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral. | Drug: Melphalan hydrochloride<br>* 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.<br>* Other names: Alkeran;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation. | 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. | Within the first six months after the initial treatment. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Retinoblastoma, intra-arterial chemotherapy, angiography