Intra-arterial Chemotherapy for Children With Retinoblastoma

Phase 1-2 Study of Injection of Melphalan Into the Ophthalmic Artery in Children With Retinoblastoma

Retinoblastoma is a cancer of the eye that occurs exclusively in children. The treatment for retinoblastoma may include surgery, chemotherapy, radiation and local treatments to the eye such as freezing (cryotherapy) and local radiation (brachytherapy). In some cases, a child with retinoblastoma will have active cancer in a single remaining eye with useful vision. In such cases, it is sometimes necessary to remove this eye. In such cases, the injection of chemotherapy directly into the artery that supplies the eye and the tumor may lead to regression of the tumor without the need to remove the eye.

This form of treatment was pioneered by a group in New York (Abramson et al). In this study the investigators will assess the efficacy and safety of the technique in a group of children with retinoblastoma.

Study Overview

• Status: Unknown
• Conditions: Retinoblastoma
• Intervention / Treatment: Drug: Intra-arterial injection of melphalan; Drug: Injection of melphalan into the ophthalmic artery

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Michael Weintraub, MD; Phone Number: 972-2-6777408; Email: michaelw@hadassah.org.il
• Study Contact Backup: Name: Jacob Pe'er, MD; Phone Number: 972-26778431

Study Locations

• Israel
  • Jerusalem, Israel, 91120
    • Recruiting
    • Hadassah University Hospital
    • Contact: Michael Weintraub, MD; Email: michaelw@hadassah.org.il
    • Principal Investigator: Michael Weintraub, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Children with retinoblastoma in a single remaining eye with useful vision

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intra-arterial melphalan; The patients will be treated by injection of chemotherapy (melphalan) into the ophthalmic artery of an eye affected by retinoblastoma	Drug: Intra-arterial injection of melphalan; Injection of 5 milligrams of melphalan into the ophthalmic artery once every 3 weeks for a total of 6 courses; Drug: Injection of melphalan into the ophthalmic artery; Injection of 5 milligrams of melphalan into the ophthalmic artery in an eye affected by retinoblastoma

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Salvage of eye and vision	Three years from intervention

Collaborators and Investigators

• Sponsor: Hadassah Medical Organization

Study record dates

Study Major Dates

• Study Start: June 1, 2011
• Primary Completion (Anticipated): December 1, 2017
• Study Completion (Anticipated): December 1, 2017

Study Registration Dates

• First Submitted: May 19, 2009
• First Submitted That Met QC Criteria: May 20, 2009
• First Posted (Estimate): May 21, 2009

Study Record Updates

• Last Update Posted (Estimate): October 4, 2016
• Last Update Submitted That Met QC Criteria: October 1, 2016
• Last Verified: October 1, 2016

More Information

Terms related to this study

• Keywords: Retinoblastoma
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Eye Diseases; Retinal Diseases; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Eye Diseases, Hereditary; Eye Neoplasms; Retinal Neoplasms; Retinoblastoma; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Melphalan
• Other Study ID Numbers: 0058-09-HMO

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES