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Combination Chemotherapy, Radiation Therapy, and Bone Marrow Transplantation in Treating Patients With 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: NCT00004006
Recruitment Status : Completed
First Posted : July 18, 2003
Last Update Posted : October 4, 2011
National Cancer Institute (NCI)
Information provided by:
St. Jude Children's Research Hospital

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy plus radiation therapy followed by bone marrow transplantation in treating patients who have retinoblastoma.

Condition or disease Intervention/treatment Phase
Retinoblastoma Biological: filgrastim Drug: carboplatin Drug: cyclophosphamide Drug: doxorubicin hydrochloride Drug: etoposide Drug: topotecan hydrochloride Procedure: autologous bone marrow transplantation Radiation: radiation therapy Phase 2

Detailed Description:


  • Evaluate the feasibility of sequential therapy with carboplatin, etoposide, cyclophosphamide, doxorubicin, topotecan and radiotherapy followed by autologous bone marrow transplantation in patients with extrachoroidal or metastatic retinoblastoma.
  • Assess this treatment regimen in terms of response and toxicity before and after autologous bone marrow transplantation in this patient population.

OUTLINE: Patients receive carboplatin IV on day 1 and etoposide IV over 1 hour daily on days 1-3 of weeks 0, 6, and 12, plus cyclophosphamide IV or orally daily on days 1-7, doxorubicin IV on day 8 and carboplatin IV over 1 hour on day 10 on weeks 3, 9, and 15. Beginning on week 6, patients receive concurrent radiotherapy 5 days a week over 4-6 weeks. Patients with meningeal involvement receive topotecan intrathecally twice weekly for 3 weeks and then weekly for 3 weeks before starting radiotherapy. Beginning one day after each treatment course, patients receive filgrastim (G-CSF) subcutaneously daily for 10 days.

Patients undergo bone marrow collection before or after week 6. Following hematologic recovery, patients receive several days of high dose chemotherapy consisting of cyclophosphamide and topotecan followed by bone marrow reinfusion.

Patients are followed at 6, 9, and 12 months, and then every 6 months for 4 years.

PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Primary Purpose: Treatment
Official Title: Treatment for Extrachoroidal or Metastatic Retinoblastoma
Study Start Date : November 1997
Actual Primary Completion Date : September 2005
Actual Study Completion Date : September 2005

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of extrachoroidal or metastatic retinoblastoma, confirmed by histology, physical examination, or diagnostic imaging



  • 15 and under

Performance status:

  • Not specified

Life expectancy:

  • Not specified


  • Not specified


  • Not specified


  • Not specified


Biologic therapy:

  • Not specified


  • Prior chemotherapy for low stage intraocular disease allowed

Endocrine therapy:

  • Not specified


  • No prior radiotherapy except to eye(s) or orbit(s)


  • Not specified

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): NCT00004006

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United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105-2794
Sponsors and Collaborators
St. Jude Children's Research Hospital
National Cancer Institute (NCI)
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Study Chair: Carlos Rodriguez-Galindo, MD St. Jude Children's Research Hospital

Additional Information:
Layout table for additonal information Identifier: NCT00004006     History of Changes
Other Study ID Numbers: CDR0000067217
P30CA021765 ( U.S. NIH Grant/Contract )
First Posted: July 18, 2003    Key Record Dates
Last Update Posted: October 4, 2011
Last Verified: October 2011
Keywords provided by St. Jude Children's Research Hospital:
intraocular retinoblastoma
extraocular retinoblastoma
recurrent retinoblastoma
Additional relevant MeSH terms:
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Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Retinal Neoplasms
Eye Neoplasms
Neoplasms by Site
Eye Diseases, Hereditary
Eye Diseases
Retinal Diseases
Liposomal doxorubicin
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antibiotics, Antineoplastic