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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00554788




Registration number
NCT00554788
Ethics application status
Date submitted
6/11/2007
Date registered
7/11/2007
Date last updated
2/10/2023

Titles & IDs
Public title
Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma
Scientific title
A Trial of Intensive Multi-Modality Therapy for Extra-Ocular Retinoblastoma
Secondary ID [1] 0 0
NCI-2009-00421
Secondary ID [2] 0 0
ARET0321
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Extraocular Retinoblastoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Children's - Other
Cancer 0 0 0 0
Head and neck
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - Autologous Bone Marrow Transplantation
Treatment: Surgery - Autologous Hematopoietic Stem Cell Transplantation
Treatment: Drugs - Carboplatin
Treatment: Drugs - Cisplatin
Treatment: Drugs - Cyclophosphamide
Treatment: Drugs - Etoposide
Other interventions - Filgrastim
Treatment: Surgery - In Vitro-Treated Peripheral Blood Stem Cell Transplantation
Treatment: Other - Radiation Therapy
Treatment: Drugs - Thiotepa
Treatment: Drugs - Vincristine Sulfate

Experimental: Treatment (chemotherapy, radiotherapy, autologous SCI) - INDUCTION: Patients receive vincristine IV; cisplatin IV; cyclophosphamide IV; and G-CSF SC beginning on day 3 and continuing until blood counts recover.
CONSOLIDATION (stage 4a or 4b disease only): Patients receive carboplatin IV; thiotepa IV; and etoposide IV.
AUTOLOGOUS STEM CELL INFUSION (stage 4a or 4b disease only): Patients undergo autologous stem cell infusion on day 0 and receive G-CSF 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.


Treatment: Surgery: Autologous Bone Marrow Transplantation
Undergo peripheral blood stem cell or bone marrow transplant

Treatment: Surgery: Autologous Hematopoietic Stem Cell Transplantation
Undergo peripheral blood stem cell or bone marrow transplant

Treatment: Drugs: Carboplatin
Given IV

Treatment: Drugs: Cisplatin
Given IV

Treatment: Drugs: Cyclophosphamide
Given IV

Treatment: Drugs: Etoposide
Given IV

Other interventions: Filgrastim
Given SC

Treatment: Surgery: In Vitro-Treated Peripheral Blood Stem Cell Transplantation
Undergo peripheral blood stem cell or bone marrow transplant

Treatment: Other: Radiation Therapy
Undergo radiotherapy

Treatment: Drugs: Thiotepa
Given IV

Treatment: Drugs: Vincristine Sulfate
Given IV
Intervention code [1] 0 0
Treatment: Surgery
Intervention code [2] 0 0
Treatment: Drugs
Intervention code [3] 0 0
Other interventions
Intervention code [4] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Event-free Survival (EFS)
Timepoint [1] 0 0
At 1 year
Secondary outcome [1] 0 0
Response Rate to the Induction Phase of the Regimen
Timepoint [1] 0 0
12 weeks after participant received the first dose
Secondary outcome [2] 0 0
Percentage of Participants With Adverse Events as Assessed by the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Timepoint [2] 0 0
Up to 30 days after completion of study treatment

Eligibility
Key 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
Minimum age
No limit
Maximum age
10 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/02/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2024
Actual
Sample size
Target
Accrual to date
Final
60
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
Arkansas
Country [4] 0 0
United States of America
State/province [4] 0 0
California
Country [5] 0 0
United States of America
State/province [5] 0 0
Colorado
Country [6] 0 0
United States of America
State/province [6] 0 0
Connecticut
Country [7] 0 0
United States of America
State/province [7] 0 0
Delaware
Country [8] 0 0
United States of America
State/province [8] 0 0
District of Columbia
Country [9] 0 0
United States of America
State/province [9] 0 0
Florida
Country [10] 0 0
United States of America
State/province [10] 0 0
Georgia
Country [11] 0 0
United States of America
State/province [11] 0 0
Illinois
Country [12] 0 0
United States of America
State/province [12] 0 0
Indiana
Country [13] 0 0
United States of America
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Iowa
Country [14] 0 0
United States of America
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Kentucky
Country [15] 0 0
United States of America
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Maryland
Country [16] 0 0
United States of America
State/province [16] 0 0
Massachusetts
Country [17] 0 0
United States of America
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Mississippi
Country [18] 0 0
United States of America
State/province [18] 0 0
Missouri
Country [19] 0 0
United States of America
State/province [19] 0 0
Nevada
Country [20] 0 0
United States of America
State/province [20] 0 0
New Jersey
Country [21] 0 0
United States of America
State/province [21] 0 0
New York
Country [22] 0 0
United States of America
State/province [22] 0 0
North Carolina
Country [23] 0 0
United States of America
State/province [23] 0 0
Ohio
Country [24] 0 0
United States of America
State/province [24] 0 0
Oklahoma
Country [25] 0 0
United States of America
State/province [25] 0 0
Pennsylvania
Country [26] 0 0
United States of America
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Tennessee
Country [27] 0 0
United States of America
State/province [27] 0 0
Texas
Country [28] 0 0
United States of America
State/province [28] 0 0
Virginia
Country [29] 0 0
United States of America
State/province [29] 0 0
Wisconsin
Country [30] 0 0
Argentina
State/province [30] 0 0
Buenos Aires
Country [31] 0 0
Brazil
State/province [31] 0 0
Sao Paulo
Country [32] 0 0
Canada
State/province [32] 0 0
Nova Scotia
Country [33] 0 0
Canada
State/province [33] 0 0
Quebec
Country [34] 0 0
Egypt
State/province [34] 0 0
Cairo

Funding & Sponsors
Primary sponsor type
Other
Name
Children's Oncology Group
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Cancer Institute (NCI)
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00554788
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ira J Dunkel
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00554788