A Study of Unilateral Retinoblastoma With and Without Histopathologic High-Risk Features and the Role of Adjuvant Chemotherapy
N/A
6 Years
Both
Intraocular Retinoblastoma
Trial Information
A Study of Unilateral Retinoblastoma With and Without Histopathologic High-Risk Features and the Role of Adjuvant Chemotherapy
OBJECTIVES:
I. Prospectively determine the prevalence of high-risk histopathologic features, such as
choroidal involvement, optic nerve invasion, and scleral and anterior segment involvement,
in patients with newly diagnosed unilateral retinoblastoma who have undergone enucleation.
II. Demonstrate that patients without certain high-risk features can be successfully treated
with enucleation alone by estimating the event-free survival (EFS) (where an event is
defined as the occurrence of extraocular or metastatic disease) and overall survival (OS).
III. Estimate the EFS and OS of patients with specific high-risk features who are uniformly
treated with adjuvant chemotherapy comprising vincristine, carboplatin, and etoposide.
IV. Estimate the incidence of toxicities associated with the proposed adjuvant chemotherapy
regimen.
OUTLINE: This is a prospective, nonrandomized, multicenter study. Patients are assigned to 1
of 2 groups according to presence of high-risk histopathologic features.
GROUP 1 (high-risk features): Patients receive vincristine IV and carboplatin IV over 1 hour
on day 1 and etoposide IV over 1 hour on days 1 and 2. Treatment repeats every 28 days for
up to 6 courses in the absence of disease progression or unacceptable toxicity.
GROUP 2 (no high-risk features): Patients undergo observation periodically for at least 5
years.
After completion of study treatment, patients in group 1 are followed periodically for at
least 5 years.
Inclusion Criteria:
- Newly diagnosed unilateral retinoblastoma
- Underwent enucleation as primary therapy within the past 5 weeks
- Must enroll and submit pathology slides within 21 days of enucleation
- Adjuvant chemotherapy must begin within 35 days after enucleation
- Disease with or without high-risk histopathologic features
- High-risk features are defined as any of the following:
- Posterior uveal invasion (includes choroidal invasion)
- Any degree of concomitant choroid and/or optic nerve involvement
- Tumor involving the optic nerve posterior to the lamina cribrosa as an
independent finding
- Scleral invasion
- Anterior chamber seeding
- Ciliary body infiltration
- Iris infiltration
- No evidence of extraocular retinoblastoma clinically, by CT scan, or by MRI of the
brain and orbits with and without gadolinium
- No tumor at the cut end of the optic nerve on any eye enucleated as evidenced by
histologic examination prior to study entry
- No systemic metastases as evidenced by bone marrow scan, bone scan, or any other
additional test at study entry
- Lansky performance status 50-100%
- Hemoglobin > 8 g/dL
- Absolute neutrophil count ≥ 1,000/mm³
- Platelet count ≥ 100,000/mm³
- Creatinine adjusted according to age as follows:
- No greater than 0.4 mg/dL (≤ 5 months)
- No greater than 0.5 mg/dL (6 months -11 months)
- No greater than 0.6 mg/dL (1 year-23 months)
- No greater than 0.8 mg/dL (2 years-5 years)
- No greater than 1.0 mg/dL (6 years-9 years)
- No greater than 1.2 mg/dL (10 years-12 years)
- No greater than 1.4 mg/dL (13 years and over [female])
- No greater than 1.5 mg/dL (13 years to 15 years [male])
- No greater than 1.7 mg/dL (16 years and over [male])
- Creatinine clearance or radioisotope glomerular filtration rate at least 70 mL/min
- Bilirubin ≤ 1.5 times upper limit of normal (ULN) for age
- AST or ALT < 2.5 times ULN for age
- No prior therapy other than enucleation
- No prior chemotherapy
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Event-free survival (EFS)
Outcome Description:
EFS distributions will be estimated by the Kaplan-Meier method for patients with high risk features (treated with adjuvant chemotherapy) and separately for subjects without high risk features (treated with enucleation alone).
Outcome Time Frame:
At 2 years
Safety Issue:
No
Principal Investigator
Murali Chintagumpala
Investigator Role:
Principal Investigator
Investigator Affiliation:
Children's Oncology Group
Authority:
United States: Institutional Review Board
Study ID:
ARET0332
NCT ID:
NCT00335738
Start Date:
December 2005
Completion Date:
Related Keywords:
- Intraocular Retinoblastoma
- Retinoblastoma
Name | Location |
|---|---|
| Baylor College of Medicine | Houston, Texas 77030 |
| Johns Hopkins University | Baltimore, Maryland 21205 |
| Cleveland Clinic Foundation | Cleveland, Ohio 44195 |
| Mayo Clinic | Rochester, Minnesota 55905 |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania 19104 |
| University of Iowa Hospitals and Clinics | Iowa City, Iowa 52242 |
| Washington University School of Medicine | Saint Louis, Missouri 63110 |
| University of Texas Health Science Center at San Antonio | San Antonio, Texas 78284-7811 |
| Midwest Children's Cancer Center | Milwaukee, Wisconsin 53226 |
| Vanderbilt-Ingram Cancer Center | Nashville, Tennessee 37232-6838 |
| Massachusetts General Hospital Cancer Center | Boston, Massachusetts 02114 |
| Marshfield Clinic | Marshfield, Wisconsin 54449 |
| Dana-Farber Cancer Institute | Boston, Massachusetts 02115 |
| Children's Hospital Los Angeles | Los Angeles, California 90027-0700 |
| Children's National Medical Center | Washington, District of Columbia 20010-2970 |
| Maine Children's Cancer Program | Scarborough, Maine 04074-9308 |
| Carolinas Medical Center | Charlotte, North Carolina 28232-2861 |
| Scott and White Memorial Hospital | Temple, Texas 76508 |
| Southern California Permanente Medical Group | Downey, California 90242 |
| Kosair Children's Hospital | Louisville, Kentucky 40202-3830 |
| Brooklyn Hospital Center | Brooklyn, New York 11201 |
| Covenant Children's Hospital | Lubbock, Texas 79410 |
| Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio 45229-3039 |
| Primary Children's Medical Center | Salt Lake City, Utah 84113-1100 |
| Rady Children's Hospital - San Diego | San Diego, California 92123-4282 |
| University of New Mexico Cancer Center | Albuquerque, New Mexico 87131-5636 |
| Nationwide Children's Hospital | Columbus, Ohio 43205-2696 |
| Lehigh Valley Hospital - Muhlenberg | Bethlehem, Pennsylvania 18017 |
| University of Alabama at Birmingham | Birmingham, Alabama 35294-3300 |
| Duke University Medical Center | Durham, North Carolina 27710 |
| Wayne State University | Detroit, Michigan 48202 |
| University of Arizona Health Sciences Center | Tucson, Arizona 85724 |
| University of Kentucky | Lexington, Kentucky 40536-0098 |
| M D Anderson Cancer Center | Houston, Texas 77030 |
| Childrens Memorial Hospital | Chicago, Illinois 60614 |
| Lombardi Comprehensive Cancer Center at Georgetown University | Washington, District of Columbia 20057 |
| University of Illinois | Chicago, Illinois 60612 |
| Cook Children's Medical Center | Fort Worth, Texas 76104 |
| The Children's Medical Center of Dayton | Dayton, Ohio 45404 |
| University of Miami Miller School of Medicine-Sylvester Cancer Center | Miami, Florida 33136 |
| University of Minnesota Medical Center-Fairview | Minneapolis, Minnesota 55455 |
| Children's Oncology Group | Arcadia, California 91006-3776 |
| The Childrens Mercy Hospital | Kansas City, Missouri 64108 |
| Rainbow Babies and Childrens Hospital | Cleveland, Ohio 44106 |
| Children's Hospital and Medical Center of Omaha | Omaha, Nebraska 68114 |
| Childrens Hospital-King's Daughters | Norfolk, Virginia 23507 |
| Children's Hospital Colorado | Aurora, Colorado 80045 |
| University of California San Francisco Medical Center-Parnassus | San Francisco, California 94143 |
