Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma
Status: | Active, not recruiting |
---|---|
Conditions: | Brain Cancer |
Therapuetic Areas: | Oncology |
Healthy: | No |
Age Range: | Any - 10 |
Updated: | 2/10/2019 |
Start Date: | February 4, 2008 |
A Trial of Intensive Multi-Modality Therapy for Extra-Ocular Retinoblastoma
This phase III trial is studying the side effects and how well giving combination
chemotherapy together with autologous stem cell transplant and/or radiation therapy works in
treating young patients with extraocular retinoblastoma. Giving chemotherapy before an
autologous stem cell transplant stops the growth of tumor cells by stopping them from
dividing or killing them. After treatment, stem cells are collected from the patient?s blood
and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the
stem cell transplant. The stem cells are then returned to the patient to replace the
blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high
energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy
and/or autologous stem cell transplant may kill any remaining tumor cells.
chemotherapy together with autologous stem cell transplant and/or radiation therapy works in
treating young patients with extraocular retinoblastoma. Giving chemotherapy before an
autologous stem cell transplant stops the growth of tumor cells by stopping them from
dividing or killing them. After treatment, stem cells are collected from the patient?s blood
and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the
stem cell transplant. The stem cells are then returned to the patient to replace the
blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high
energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy
and/or autologous stem cell transplant may kill any remaining tumor cells.
OBJECTIVES:
I. To estimate the proportion of 3 groups of patients with extraocular retinoblastoma (stage
2 and 3: regional extra-ocular disease;, stage 4a: disseminated metastatic disease not
involving the central nervous system [CNS]; or stage 4b: patients with CNS disease) who
achieve long-term event-free survival after treatment with aggressive multimodality therapy
compared to historical controls.
II. To estimate the response rate to the induction phase of the regimen. III. To evaluate the
toxicities associated with this regimen.
OUTLINE:
INDUCTION CHEMOTHERAPY: Patients receive vincristine intravenously (IV) on days 0, 7, and 14,
cisplatin IV over 6 hours on day 0, cyclophosphamide IV over 1 hour and etoposide IV over 1
hour on days 1 and 2, and filgrastim (G-CSF) subcutaneously (SC) beginning on day 3 and
continuing until blood counts recover.
Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or
unacceptable toxicity.
After completion of induction chemotherapy, patients with stage 2 or 3 disease who have at
least a partial response proceed to radiotherapy. Patients with stage 4a or 4b disease who
have at least a partial response proceed to high-dose consolidation chemotherapy and
autologous stem cell infusion.
STEM CELL HARVESTING (stage 4a or 4b disease only): Peripheral blood stem cells (preferred)
or bone marrow cells are collected after at least 1 course of induction chemotherapy.
HIGH-DOSE CONSOLIDATION CHEMOTHERAPY (stage 4a or 4b disease only): Patients receive
carboplatin IV over 4 hours on days -8 to -6 and thiotepa IV over 3 hours and etoposide IV
over 3 hours on days -5 to -3.
AUTOLOGOUS STEM CELL INFUSION (stage 4a or 4b disease only): Patients undergo autologous stem
cell infusion on day 0. Patients then receive filgrastim subcutaneously (SC) beginning on day
1 and continuing until blood counts recover.
RADIOTHERAPY: Patients with stage 2 or 3 disease (orbital and/or regional involvement)
undergo radiotherapy to sites that were initially involved beginning within 42 days after the
start of course 4 of induction chemotherapy. Patients with stage 4a or 4b disease undergo
radiotherapy to sites initially involved based on response beginning approximately 42 days
after autologous stem cell infusion. Patients with stage 4a disease who achieve a complete
response to induction chemotherapy or with less than 5 mm of residual tumor at the time of
planned irradiation, or patients with stage 4b disease who achieve a complete response to
induction chemotherapy do not undergo radiotherapy.
After completion of study therapy, patients are followed every 3 months for 1 year and then
annually thereafter for 9 years.
I. To estimate the proportion of 3 groups of patients with extraocular retinoblastoma (stage
2 and 3: regional extra-ocular disease;, stage 4a: disseminated metastatic disease not
involving the central nervous system [CNS]; or stage 4b: patients with CNS disease) who
achieve long-term event-free survival after treatment with aggressive multimodality therapy
compared to historical controls.
II. To estimate the response rate to the induction phase of the regimen. III. To evaluate the
toxicities associated with this regimen.
OUTLINE:
INDUCTION CHEMOTHERAPY: Patients receive vincristine intravenously (IV) on days 0, 7, and 14,
cisplatin IV over 6 hours on day 0, cyclophosphamide IV over 1 hour and etoposide IV over 1
hour on days 1 and 2, and filgrastim (G-CSF) subcutaneously (SC) beginning on day 3 and
continuing until blood counts recover.
Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or
unacceptable toxicity.
After completion of induction chemotherapy, patients with stage 2 or 3 disease who have at
least a partial response proceed to radiotherapy. Patients with stage 4a or 4b disease who
have at least a partial response proceed to high-dose consolidation chemotherapy and
autologous stem cell infusion.
STEM CELL HARVESTING (stage 4a or 4b disease only): Peripheral blood stem cells (preferred)
or bone marrow cells are collected after at least 1 course of induction chemotherapy.
HIGH-DOSE CONSOLIDATION CHEMOTHERAPY (stage 4a or 4b disease only): Patients receive
carboplatin IV over 4 hours on days -8 to -6 and thiotepa IV over 3 hours and etoposide IV
over 3 hours on days -5 to -3.
AUTOLOGOUS STEM CELL INFUSION (stage 4a or 4b disease only): Patients undergo autologous stem
cell infusion on day 0. Patients then receive filgrastim subcutaneously (SC) beginning on day
1 and continuing until blood counts recover.
RADIOTHERAPY: Patients with stage 2 or 3 disease (orbital and/or regional involvement)
undergo radiotherapy to sites that were initially involved beginning within 42 days after the
start of course 4 of induction chemotherapy. Patients with stage 4a or 4b disease undergo
radiotherapy to sites initially involved based on response beginning approximately 42 days
after autologous stem cell infusion. Patients with stage 4a disease who achieve a complete
response to induction chemotherapy or with less than 5 mm of residual tumor at the time of
planned irradiation, or patients with stage 4b disease who achieve a complete response to
induction chemotherapy do not undergo radiotherapy.
After completion of study therapy, patients are followed every 3 months for 1 year and then
annually thereafter for 9 years.
Inclusion Criteria:
- Patients must have histologic or cytologic verification of extra-ocular
retinoblastoma; extra-ocular disease includes orbital disease, optic nerve involvement
at the surgical margin, regional nodal disease, and/or overt distant metastatic
disease (at sites such as bone, bone marrow, liver and/or the central nervous system);
patients with trilateral retinoblastoma will also be included in this protocol
- Patients with a CNS lesion consistent with trilateral or stage 4b disease may be
enrolled without tissue confirmation if (1) unequivocal leptomeningeal disease is
present on brain or spine magnetic resonance imaging (MRI) scan and/or (2) the
primary tumor is at least 2 cm in diameter, predominantly solid, and demonstrates
enhancement on the post-gadolinium images; however, even in such cases surgery
should be given serious consideration
- Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age
- No prior chemotherapy or radiotherapy for the extra-ocular retinoblastoma may have
been administered prior to entering this study; prior treatment (chemotherapy and/or
radiation therapy) for intra-ocular retinoblastoma is permissible
- Peripheral absolute neutrophil count (ANC) >= 750/uL
- If the ANC and/or platelet count are not adequate, but due to bone marrow
metastatic disease, these criteria will be waived
- Platelet count >= 75,000/uL (transfusion independent)
- If the ANC and/or platelet count are not adequate, but due to bone marrow
metastatic disease, these criteria will be waived
- Creatinine clearance OR radioisotope glomerular filtration rate >= 70 mL/min/1.73 m^2
or a serum creatinine based on age/gender as follows:
- 0.4 mg/dL (1 month to < 6 months of age)
- 0.5 mg/dL (6 months to < 1 year of age)
- 0.6 mg/dL (1 years to < 2 years of age)
- 0.8 mg/dL (2 years to < 6 years of age)
- 1.0 mg/dL (6 years to < 10 years of age)
- 1.2 mg/dL (10 years to < 13 years of age)
- 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 years to < 16 years of age)
- 1.7 mg/dL (male) or 1.4 mg/dL (female) (>= 16 years of age)
- Total bilirubin =< 1.5 times upper limit of normal (ULN)
- Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or
serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x
upper limit of normal (ULN)
- All patients and/or their parents or legal guardians must sign a written informed
consent
- All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) for human studies must be met
We found this trial at
67
sites
529 West Markham Street
Little Rock, Arkansas 72205
Little Rock, Arkansas 72205
(501) 686-7000
University of Arkansas for Medical Sciences The University of Arkansas for Medical Sciences (UAMS) in...
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Children's Hospital of Alabama Children
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
Cincinnati, Ohio 45229
1-513-636-4200
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Rainbow Babies and Children's Hospital UH Rainbow Babies & Children’s Hospital is a 244-bed, full-service...
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Medical City Dallas Hospital If you have concerns for your health, that of a family...
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Connecticut Children's Medical Center Connecticut Children’s Medical Center is a nationally recognized, 187-bed not-for-profit children’s...
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University of Mississippi Medical Center The University of Mississippi Medical Center, located in Jackson, is...
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Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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1717 South Orange Avenue # 100
Orlando, Florida 32806
Orlando, Florida 32806
(407) 650-7000
Nemours Children's Clinic - Orlando Located near downtown Orlando, Nemours Children’s Clinic, Orlando is a...
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Nemours Children's Clinic - Pensacola Nemours Children’s Clinic, Pensacola serves children and families in northwest...
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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401 College Street
Richmond, Virginia 23298
Richmond, Virginia 23298
(804) 828-0450
Virginia Commonwealth University Massey Cancer Center Founded in 1974, VCU Massey Cancer Center is a...
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7700 Floyd Curl Dr
San Antonio, Texas 78229
San Antonio, Texas 78229
(210) 575-7000
Methodist Children's Hospital of South Texas Methodist Children
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4502 Medical Drive
San Antonio, Texas 78284
San Antonio, Texas 78284
(210) 567-7000
University of Texas Health Science Center at San Antonio The University of Texas Health Science...
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New York Medical College The College was founded in 1860 by a group of New...
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Alfred I. duPont Hospital for Children Nemours began more than 70 years ago with the...
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Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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8901 Rockville Pike
Bethesda, Maryland 20889
Bethesda, Maryland 20889
(301) 295-4000
Walter Reed National Military Medical Center The Walter Reed National Military Medical Center is one...
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Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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Massachusetts General Hospital Cancer Center An integral part of one of the world
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Univ of Illinois A major research university in the heart of one of the world's...
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Cleveland Clinic Foundation The Cleveland Clinic (formally known as The Cleveland Clinic Foundation) is a...
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Denver, Colorado 80218
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Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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Cook Children's Medical Center Cook Children's Health Care System is a not-for-profit, nationally recognized pediatric...
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Riley Hospital for Children Riley Hospital for Children at IU Health is a place of...
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601 South Rancho Drive
Suite C-26
Las Vegas, Nevada 89106
Las Vegas, Nevada 89106
(702) 384-0013
Nevada Cancer Research Foundation CCOP The Nevada Cancer Research Foundation Community Clinical Oncology Program (NCRF-CCOP)...
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Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering Cancer Center — the world's oldest and...
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940 NE 13th St
Oklahoma City, Oklahoma 73190
Oklahoma City, Oklahoma 73190
(405) 271-6458
University of Oklahoma Health Sciences Center The OU Health Sciences Center is composed of seven...
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Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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725 Welch Rd
Palo Alto, California 94304
Palo Alto, California 94304
(650) 497-8000
Lucile Packard Children's Hospital Stanford University Stanford Children's Health is the only network in the...
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Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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660 S Euclid Ave
Saint Louis, Missouri 63110
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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111 Michigan Ave NW
Washington, District of Columbia
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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