Combination Chemotherapy, Radiation Therapy, and Bone Marrow Transplantation in Treating Patients With Retinoblastoma

This study has been completed.
National Cancer Institute (NCI)
Information provided by:
St. Jude Children's Research Hospital Identifier:
First received: November 1, 1999
Last updated: October 3, 2011
Last verified: October 2011

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 Intervention Phase
Biological: filgrastim
Drug: carboplatin
Drug: cyclophosphamide
Drug: doxorubicin hydrochloride
Drug: etoposide
Drug: topotecan hydrochloride
Procedure: autologous bone marrow transplantation
Radiation: radiation therapy
Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: Treatment for Extrachoroidal or Metastatic Retinoblastoma

Resource links provided by NLM:

Further study details as provided by St. Jude Children's Research Hospital:

Enrollment: 4
Study Start Date: November 1997
Study Completion Date: September 2005
Primary Completion Date: September 2005 (Final data collection date for primary outcome measure)
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.


Ages Eligible for Study:   up to 15 Years
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004006

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)
Study Chair: Carlos Rodriguez-Galindo, MD St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided Identifier: NCT00004006     History of Changes
Other Study ID Numbers: CDR0000067217, P30CA021765, SJCRH: RET4, NCI-G99-1555
Study First Received: November 1, 1999
Last Updated: October 3, 2011
Health Authority: United States: Federal Government

Keywords provided by St. Jude Children's Research Hospital:
intraocular retinoblastoma
extraocular retinoblastoma
recurrent retinoblastoma

Additional relevant MeSH terms:
Eye Diseases
Eye Neoplasms
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Retinal Diseases
Retinal Neoplasms
Liposomal doxorubicin
Antibiotics, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses
Topoisomerase I Inhibitors
Topoisomerase II Inhibitors
Topoisomerase Inhibitors processed this record on October 08, 2015