Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma
Overview
- Phase
- Phase 2
- Intervention
- Melphalan hydrochloride
- Conditions
- Retinoblastoma
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
- Status
- Terminated
- Last Updated
- 7 years ago
Overview
Brief Summary
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
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.
Investigators
Eligibility Criteria
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
- •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
Arms & Interventions
Intraocular Retinoblastoma Patients
Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral.
Intervention: Melphalan hydrochloride
Outcomes
Primary Outcomes
Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
Time Frame: Within the first six months after the initial treatment.
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.