NCT02097134

Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

A Multi-institutional Feasibility Study of Intra-arterial Chemotherapy Given in the Ophthalmic Artery of Children With Retinoblastoma

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Children's Oncology Group · Network
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

Summary

This pilot clinical trial studies whether unilateral group D retinoblastoma, or retinoblastoma affecting one eye that has spread to the inner jelly like part of the eye, can be treated with a new technique for delivering chemotherapy directly into the blood vessel that supplies the affected eye. This new technique is called intra-arterial injection. Giving melphalan via intra-arterial injection may make it less likely that children will need surgery to remove the eye and may reduce the amount of treatment side effects.

Detailed description

PRIMARY OBJECTIVES: I. To study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group D retinoblastoma, who would otherwise be considered for enucleation. SECONDARY OBJECTIVES: I. To estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group D disease. II. To evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy. III. To evaluate vision outcomes in children treated with intra-arterial chemotherapy. IV. To monitor the rate of the development of metastatic disease while on protocol therapy. TERTIARY OBJECTIVES: I. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression. OUTLINE: Patients receive melphalan intra-arterially (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.

Conditions

• Unilateral Retinoblastoma

Interventions

Type	Name	Description
DRUG	Melphalan	Given IA

Timeline

• Start date: 2014-10-31
• Primary completion: 2017-06-30
• Completion: 2023-09-30
• First posted: 2014-03-26
• Last updated: 2023-10-13
• Results posted: 2018-06-26

Locations

12 sites across 1 country: United States