Clinical trial NCT00004006: Combination Chemotherapy, Radiation Therapy, and Bone Marrow Transplantation in Treating Patients With Retinoblastoma
Official title: Treatment for Extrachoroidal or Metastatic Retinoblastoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinoblastoma
Keywords: intraocular retinoblastoma
extraocular retinoblastoma
recurrent retinoblastoma
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2003-07-18
Enrollment: 4
Sex: ALL
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Primary purpose: TREATMENT

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.

Detailed description: OBJECTIVES:

* 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.

Eligibility criteria: DISEASE CHARACTERISTICS:

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

PATIENT CHARACTERISTICS:

Age:

* 15 and under

Performance status:

* Not specified

Life expectancy:

* Not specified

Hematopoietic:

* Not specified

Hepatic:

* Not specified

Renal:

* Not specified

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* Not specified

Chemotherapy:

* Prior chemotherapy for low stage intraocular disease allowed

Endocrine therapy:

* Not specified

Radiotherapy:

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

Surgery:

* Not specified

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Carlos Rodriguez-Galindo, MD
role: STUDY_CHAIR
References: