Chemotherapy Treatment for Children With Intraocular Germ-Line Retinoblastoma

NCT00179920 | Completed Phase 2

• 1 other identifier: CNS 0294
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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

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

Conditions

Retinoblastoma; Retinal Neoplasms

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (5)

• 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

Interventions

Carboplatin DRUG

VP-16 DRUG

Local Surgery PROCEDURE

Cryotherapy PROCEDURE

Laser hyperthermia PROCEDURE

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead

Study Sites (1)

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

MeSH Terms

Conditions

Retinoblastoma; Retinal Neoplasms

Interventions

Carboplatin; Etoposide; Cryotherapy

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Eye Neoplasms; Neoplasms by Site; Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Intervention Hierarchy (Ancestors)

Coordination Complexes; Organic Chemicals; Podophyllotoxin; Tetrahydronaphthalenes; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Polycyclic Compounds; Glucosides; Glycosides; Carbohydrates; Therapeutics

Study Officials

• Stewart Goldman, MD

  Ann & Robert H Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Division Head

Study Record Dates

• First Submitted: September 12, 2005
• First Posted: September 16, 2005
• Study Start: April 1, 1996
• Study Completion: September 1, 2006
• Last Updated: August 5, 2020

Record last verified: 2020-08

Locations

US (1)