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Chemotherapy Treatment for Children With Intraocular Germ-Line Retinoblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00179920
Recruitment Status : Completed
First Posted : September 16, 2005
Last Update Posted : August 5, 2020
Information provided by (Responsible Party):
Stewart Goldman, Ann & Robert H Lurie Children's Hospital of Chicago

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.


  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 or disease Intervention/treatment Phase
Retinoblastoma Retinal Neoplasms Drug: Carboplatin Drug: VP-16 Procedure: Local Surgery Procedure: Cryotherapy Procedure: Laser hyperthermia Phase 2

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Study Type : Interventional  (Clinical Trial)
Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Chemotherapy Plus Local Surgical Treatment in Children With Intraocular Germ-Line Retinoblastoma
Study Start Date : April 1996
Study Completion Date : September 2006

Resource links provided by the National Library of Medicine

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

Secondary Outcome Measures :
  1. To estimate the response rate and duration for primary retinoblastoma tumors to combination therapy with carboplatin and VP-16
  2. 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
  3. Estimate tumor response and duration after cryotherapy
  4. To estimate the control rate of vitreous involvement by retinoblastoma to carboplatin and VP-16
  5. To detect any toxicities resulting from multimodal therapy plus local surgical options

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00179920

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United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
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Principal Investigator: Stewart Goldman, MD Ann & Robert H Lurie Children's Hospital of Chicago
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Responsible Party: Stewart Goldman, Division Head, Ann & Robert H Lurie Children's Hospital of Chicago Identifier: NCT00179920    
Other Study ID Numbers: CNS 0294
First Posted: September 16, 2005    Key Record Dates
Last Update Posted: August 5, 2020
Last Verified: August 2020
Additional relevant MeSH terms:
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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, Hereditary
Eye Diseases
Retinal Diseases
Antineoplastic Agents