Chemotherapy Treatment for Children With Intraocular Germ-Line Retinoblastoma

August 3, 2020 updated by: Stewart Goldman, Ann & Robert H Lurie Children's Hospital of Chicago

Chemotherapy Plus Local Surgical Treatment in Children With Intraocular Germ-Line Retinoblastoma

Retinoblastoma is an unusual cancer of early childhood involving tumor is both eyes or, in certain circumstances, one eye only. This condition is the result of an abnormal gene which makes both retinas (the back of the eye) vulnerable to develop multiple tumors. Growths in the eye impair vision temporarily or permanently. These tumors are malignant, which means that they can grow within the eye, spread outside of the eye, and be fatal if untreated.

Standard therapy for bilateral retinoblastoma includes removal of one eye if vision cannot be save and radiation treatment of either eye in which vision might be saved. Radiation controls tumor growth in the majority of cases. Another standard method is cryotherapy (freezing a tumor to kill it). Chemotherapy (medicines used to kill tumor cells) has been used in the past for tumor in or outside the eye, but is not standard. Hyperthermia, increasing the temperature of a tumor to kill it, is widely performed, and can be done to a retinoblastoma tumor by a laser; this method is not standard.

The problem with removal of an eye is that any hope of vision is lost. The problems with radiation include incomplete control of tumor, injury to the eye or surrounding tissue with decreased growth, and that (due to the abnormal retinoblastoma gene) children are very susceptible to develop other tumors, especially in the tissue which was given radiation.

The doctors at Children's Memorial Hospital are using a newer form of treatment, including laser hyperthermia, chemotherapy and cryotherapy to decrease retinoblastoma tumors. Some may be controlled indefinitely, reducing the number of eyes that need radiation or removal.

OBJECTIVES

To find out how well chemotherapy plus cryotherapy and laser hyperthermia work on retinoblastoma tumors.
To find out whether vision can be saved and tumors controlled without radiation or removal of the eye.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment

Phase

Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

United States
- Illinois
  - Chicago, Illinois, United States, 60614
    - Children's Memorial Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

Child
Adult
Older Adult

Accepts Healthy Volunteers

Genders Eligible for Study

All

Description

Eligibility Criteria:

Initial clinical diagnosis of Retinoblastoma, with Bilateral tumors, OR Unilateral tumor with hope for salvage of vision AND Multiple primary tumors, or positive family history for retinoblastoma, or age < 12 months
No prior antitumor therapy (except enucleation of one eye)
At least one tumor exceeds 6 mm in diameter
No evidence of dissemination outside the globe
Signed IRB-approved informed consent
Patient/family available for follow-up

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Interventional Model: Single Group Assignment
Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
To determine if a multimodal approach can provide durable control of retinoblastoma and specifically:
Estimate the proportion of eyes in which radiation therapy can be omitted
Estimate the proportion of eyes in which vision can be retained

Secondary Outcome Measures

Outcome Measure
To estimate the response rate and duration for primary retinoblastoma tumors to combination therapy with carboplatin and VP-16
To estimate the response rate of qualifying in situ retinoblastoma tumors to local surgical therapies, specifically estimate tumor response and duration after laser hyperthermia concurrent with carboplatin
Estimate tumor response and duration after cryotherapy
To estimate the control rate of vitreous involvement by retinoblastoma to carboplatin and VP-16
To detect any toxicities resulting from multimodal therapy plus local surgical options

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 1996

Study Completion

September 1, 2006

Study Registration Dates

First Submitted

September 12, 2005

First Submitted That Met QC Criteria

September 12, 2005

First Posted (Estimate)

September 16, 2005

Study Record Updates

Last Update Posted (Actual)

August 5, 2020

Last Update Submitted That Met QC Criteria

August 3, 2020

Last Verified

August 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

CNS 0294

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.