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Chemotherapy Treatment for Children With Intraocular Germ-Line Retinoblastoma

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified June 2011 by Ann & Robert H Lurie Children's Hospital of Chicago.
Recruitment status was:  Active, not recruiting
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago Identifier:
First received: September 12, 2005
Last updated: September 25, 2012
Last verified: June 2011

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.


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

Condition Intervention Phase
Retinoblastoma Retinal Neoplasms Drug: Carboplatin Drug: VP-16 Procedure: Local Surgery Procedure: Cryotherapy Procedure: Laser hyperthermia Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Chemotherapy Plus Local Surgical Treatment in Children With Intraocular Germ-Line Retinoblastoma

Resource links provided by NLM:

Further study details as provided by Ann & Robert H Lurie Children's Hospital of Chicago:

Primary Outcome Measures:
  • 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:
  • 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

Estimated Enrollment: 30
Study Start Date: April 1996
Estimated Study Completion Date: September 2006

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00179920

United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Principal Investigator: Stewart Goldman, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information Identifier: NCT00179920     History of Changes
Other Study ID Numbers: CNS 0294
Study First Received: September 12, 2005
Last Updated: September 25, 2012

Additional relevant MeSH terms:
Retinal Neoplasms
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Eye Neoplasms
Neoplasms by Site
Eye Diseases
Retinal Diseases
Antineoplastic Agents processed this record on June 23, 2017