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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00075582




Registration number
NCT00075582
Ethics application status
Date submitted
9/01/2004
Date registered
12/01/2004
Date last updated
4/11/2016

Titles & IDs
Public title
Vincristine, Dactinomycin, and Cyclophosphamide With or Without Radiation Therapy in Treating Patients With Newly Diagnosed Low-Risk Rhabdomyosarcoma
Scientific title
Vincristine, Dactinomycin, and Lower Doses of Cyclophosphamide With or Without Radiation Therapy for Patients With Newly Diagnosed Low-Risk Embryonal/Botryoid/Spindle Cell Rhabdomyosarcoma
Secondary ID [1] 0 0
NCI-2009-00425
Secondary ID [2] 0 0
ARST0331
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Adult Rhabdomyosarcoma 0 0
Embryonal Childhood Rhabdomyosarcoma 0 0
Embryonal-botryoid Childhood Rhabdomyosarcoma 0 0
Previously Untreated Childhood Rhabdomyosarcoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Sarcoma (also see 'Bone') - soft tissue
Cancer 0 0 0 0
Bone
Cancer 0 0 0 0
Children's - Other

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - conventional surgery
Treatment: Drugs - dactinomycin
Treatment: Drugs - cyclophosphamide
Treatment: Drugs - vincristine sulfate
Treatment: Other - radiation therapy

Experimental: Regimen I (chemotherapy, radiotherapy) - Patients receive VAC chemotherapy comprising vincristine sulfate IV over 1 minute on day 1 of weeks 1-9 and dactinomycin IV over 1 minute and cyclophosphamide IV over 1 hour on day 1 of weeks 1, 4, 7, and 10; VA chemotherapy comprising vincristine sulfate IV over 1 minute on day 1 of weeks 13-21 and dactinomycin IV over 1 minute on day 1 of weeks 13, 16, 19, and 22 (dactinomycin is omitted during radiation therapy); and radiation therapy, 5 days a week, beginning on week 13 and continuing for 4-7 weeks, depending on prescribed dose. Some patients do not receive radiation therapy; some start it at week 24. (closed to accrual as of 08/13/2010)

Experimental: Regimen II (chemotherapy, radiotherapy, surgery) - Patients receive VAC chemotherapy and radiation therapy as in regimen I and VA chemotherapy comprising vincristine sulfate IV over 1 minute on day 1 of weeks 13-21, 25-33, and 37-45 and dactinomycin IV over 1 minute on day 1 of weeks 13, 16, 19, 22, 25, 28, 31, 34, 37, 40, 43, and 46 (dactinomycin is omitted during radiation therapy). Some patients do not receive radiation therapy; some start it at week 13 and some at week 24. Some patients have conventional surgery (second-look) at Week 13 (closed to accrual as of 9/23/2011).


Treatment: Surgery: conventional surgery
Some patients may undergo second-look surgery

Treatment: Drugs: dactinomycin
Given IV

Treatment: Drugs: cyclophosphamide
Given IV

Treatment: Drugs: vincristine sulfate
Given IV

Treatment: Other: radiation therapy
Undergo radiotherapy

Intervention code [1] 0 0
Treatment: Surgery
Intervention code [2] 0 0
Treatment: Drugs
Intervention code [3] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Estimated Percentage of Patients Failure Free at 5 Years (95% Confidence Interval) - Failure-free survival: Time to disease recurrence or death as a first event. An analysis plan based on the method of Woolson (1981) will be used to monitor outcome for these patients.
Timepoint [1] 0 0
5 years from study enrollment
Primary outcome [2] 0 0
Estimated Percentage of Patients With Stage 1, Clinical Group IIB or C (Node Positive) or Stage 2 Group I or Stage 2 Group II Disease Treated With Regimen 1 Failure-free at 5 Years - Failure-free survival: Time to disease recurrence or death as a first event. An analysis plan based on the method of Woolson (1981) will be used to monitor outcome for these patients.
Timepoint [2] 0 0
5 years from study enrollment
Primary outcome [3] 0 0
Estimated Percentage of Patients With Low-risk Rhabdomyosarcoma Treated With Regimen 2 Therapy Failure Free at 5 Years (95% Confidence Interval). - Failure free survival: Time to disease recurrence or death as a first event. An analysis plan based on the method of Woolson (1981) will be used to monitor outcome for these patients.
Timepoint [3] 0 0
5 years from study enrollment
Secondary outcome [1] 0 0
Rate of Local Failure for Patients Who Receive Reduced Doses of Radiation Therapy - The local failure rate will be estimated using cumulative incidence curves.
Timepoint [1] 0 0
Up to 10 years
Secondary outcome [2] 0 0
Rate of Second-look Surgery and the Proportion of Patients Who Are Tumor-free or With Microscopic Tumor Only Following Second-look Surgeries - The decision to perform second-look surgery should be based on the physical examination and imaging studies at Week 12 and should only be considered if a reasonable functional and cosmetic result is anticipated.
Timepoint [2] 0 0
At 13 weeks
Secondary outcome [3] 0 0
Rate of Local Failure for Patients With Clinical Group III Disease When the Radiotherapy Dose is Reduced After Second-look Surgical Resection. - The local failure rate will be estimated using cumulative incidence curves.
Timepoint [3] 0 0
Up to 20 weeks

Eligibility
Key inclusion criteria
- Histologically confirmed newly diagnosed embryonal rhabdomyosarcoma (RMS), botryoid or
spindle cell variants of embryonal RMS, or embryonal ectomesenchymoma, meeting
criteria for 1 of the following subsets:

- Subset 1, defined by meeting 1 of the following criteria (closed to accrual as of
08/13/2010):

- Stage 1 and clinical group I (completely resected) or II (microscopic
residual disease and/or regional lymph node involvement) disease

- Stage 1 and clinical group III (gross residual disease) disease arising in
the orbit

- Stage 2 and clinical group I or II disease

- Subset 2, defined by meeting 1 of the following criteria (closed to accrual as of
09/23/2011):

- Stage 1 and clinical group III disease arising in a non-orbit site

- Stage 3 and clinical group I or II disease

- Prior staging ipsilateral retroperitoneal lymph node dissection required for all
patients age 10 and over with paratesticular tumors and patients under 10 years of age
with clinically or radiographically involved lymph nodes (except when extensive lymph
node involvement is identified by imaging studies)

- If there is extensive gross node involvement only confirmatory node biopsy is
recommended and the patient is classified as Clinical Group III

- Prior regional lymph node sampling required for patients with extremity tumors

- None of the following diagnoses:

- Intermediate-risk embryonal RMS

- Metastatic embryonal RMS

- Alveolar RMS

- Undifferentiated sarcoma

- RMS not otherwise specified (NOS)

- Other soft tissue sarcoma, including sarcoma NOS

- Prior enrollment on clinical trial COG-D9902

- Performance status - ECOG 0-2

- Performance status - Karnofsky 50-100% (= 16 years old)

- Performance status - Lansky 50-100% (< 16 years old)

- Absolute neutrophil count at least 750/mm^3

- Platelet count at least 75,000/mm^3 (transfusion independent)

- Bilirubin no greater than 1.5 times upper limit of normal (ULN)*

- Creatinine* based on age/gender as follows:

- No greater than 0.8 mg/dL for patients age 5 and under

- No greater than 1.0 mg/dL for patients age 6 to 9

- No greater than 1.2 mg/dL for patients age 10 to 12

- No greater than 1.4 mg/dL for female patients age 13 and over

- No greater than 1.5 mg/dL for male patients age 13 to 15

- No greater than 1.7 mg/dL for male patients age 16 and over

- Creatinine clearance* or radioisotope glomerular filtration rate at least 70
mL/min/1.73 m^2

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No uncontrolled infection

- No prior chemotherapy (except for patients treated on the related intermediate-risk
study)

- Prior steroids allowed

- No prior radiotherapy
Minimum age
No limit
Maximum age
49 Years
Gender
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
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Unknown status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
The Children's Hospital at Westmead - Sydney
Recruitment hospital [3] 0 0
Sydney West Area Health Service-Westmead Hospital - Westmead
Recruitment hospital [4] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [5] 0 0
Women's and Children's Hospital-Adelaide - North Adelaide
Recruitment hospital [6] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [7] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
2145 - Sydney
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment postcode(s) [4] 0 0
4029 - Herston
Recruitment postcode(s) [5] 0 0
5006 - North Adelaide
Recruitment postcode(s) [6] 0 0
3052 - Parkville
Recruitment postcode(s) [7] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
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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
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Connecticut
Country [7] 0 0
United States of America
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District of Columbia
Country [8] 0 0
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Florida
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United States of America
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Georgia
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United States of America
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Hawaii
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Idaho
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Illinois
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Indiana
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Iowa
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Kentucky
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Louisiana
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Maine
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Mississippi
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Missouri
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Nebraska
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Nevada
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New Hampshire
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New Jersey
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New Mexico
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New York
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United States of America
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North Carolina
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Ohio
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Oregon
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United States of America
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Pennsylvania
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South Carolina
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Tennessee
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Texas
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Utah
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Vermont
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Virginia
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Washington
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West Virginia
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Wisconsin
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Canada
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Alberta
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Canada
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British Columbia
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Canada
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Manitoba
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Canada
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Newfoundland and Labrador
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Canada
State/province [47] 0 0
Ontario
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Canada
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Quebec
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Canada
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Saskatchewan
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New Zealand
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Auckland
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New Zealand
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Christchurch
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Puerto Rico
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Santurce
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Switzerland
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Geneva
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Switzerland
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Lausanne

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 how well combination chemotherapy and radiation therapy work
in treating patients with newly diagnosed low-risk rhabdomyosarcoma. Drugs used in
chemotherapy, such as vincristine, dactinomycin, and cyclophosphamide, work in 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. Combining chemotherapy with radiation therapy may
kill more tumor cells. It is not yet known which treatment regimen is more effective in
treating low-risk rhabdomyosarcoma.
Trial website
https://clinicaltrials.gov/show/NCT00075582
Trial related presentations / publications
Woolson RF (1981) Rank-tests and a one-sample log-rank test for comparing observed survival-data to a standard population. Biometrics 37: 687-696.
Public notes

Contacts
Principal investigator
Name 0 0
David Walterhouse, MD
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/show/NCT00075582