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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/NCT02567435
Registration number
NCT02567435
Ethics application status
Date submitted
2/10/2015
Date registered
5/10/2015
Date last updated
23/04/2024
Titles & IDs
Public title
Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma
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Scientific title
A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
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Secondary ID [1]
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NCI-2015-01644
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Secondary ID [2]
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NCI-2015-01644
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Alveolar Rhabdomyosarcoma
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Botryoid-Type Embryonal Rhabdomyosarcoma
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Embryonal Rhabdomyosarcoma
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Rhabdomyosarcoma
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Sclerosing Rhabdomyosarcoma
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Spindle Cell Rhabdomyosarcoma
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Condition category
Condition code
Cancer
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Sarcoma (also see 'Bone') - soft tissue
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Cancer
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Bone
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Cancer
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Children's - Other
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Cyclophosphamide
Other interventions - Dactinomycin
Treatment: Drugs - Irinotecan Hydrochloride
Other interventions - Laboratory Biomarker Analysis
Other interventions - Questionnaire Administration
Treatment: Other - Radiation Therapy
Treatment: Drugs - Temsirolimus
Treatment: Drugs - Vincristine Sulfate
Treatment: Drugs - Vinorelbine
Experimental: Regimen A (VAC/VI) - Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37. Patients also undergo primary site RT beginning at week 13 or metastatic site RT beginning at week 43 for up to 6.5 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients then receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide PO QD on days 1-28. Cycles repeats every 28 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
Experimental: Regimen B (VAC/VI/temsirolimus) - Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-13, 16, 17, 19, 20, 22-26, 28, 31-34, 37, 38, and 40, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 7, 13, 22, 28, 34, and 40, irinotecan hydrochloride IV over 90 minutes on days 1-5 of weeks 4, 10, 16, 19, 25, 31, and 37 and temsirolimus IV over 30-60 minutes on day 1 of weeks 1-12 and 21-42. Patients also undergo RT as in Regimen A. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients then receive vinorelbine IV over 6-10 minutes on days 1, 8, and 15 and cyclophosphamide PO QD on days 1-28. Cycles repeats every 28 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
Experimental: Regimen C (FOXO1 fusion negative, VAC/VA) - Patients receive vincristine sulfate IV over 1 minute on day 1 of weeks 1-10 and 13-22, dactinomycin IV over 1-5 or 10-15 minutes on day 1 of weeks 1, 4, 7, 10, 13, 16, 19, and 22, cyclophosphamide IV over 60 minutes on day 1 of weeks 1, 4, 7, and 10. Patients undergo RT beginning at week 13 for up to 6.5 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
Treatment: Drugs: Cyclophosphamide
Given IV and PO
Other interventions: Dactinomycin
Given IV
Treatment: Drugs: Irinotecan Hydrochloride
Given IV
Other interventions: Laboratory Biomarker Analysis
Correlative studies
Other interventions: Questionnaire Administration
Ancillary studies
Treatment: Other: Radiation Therapy
Undergo RT
Treatment: Drugs: Temsirolimus
Given IV
Treatment: Drugs: Vincristine Sulfate
Given IV
Treatment: Drugs: Vinorelbine
Given IV
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Intervention code [3]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Event-free survival (EFS)
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Assessment method [1]
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EFS distributions will be estimated using the Kaplan-Meier method and will be compared between the randomized treatment groups using the log-rank test.
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Timepoint [1]
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Time from study enrollment to the first occurrence of progression, relapse, second malignant neoplasm or death as a first event
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Secondary outcome [1]
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Overall survival (OS)
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Assessment method [1]
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OS distributions will be estimated using the Kaplan-Meier method and will be compared between the randomized treatment groups using the log-rank test.
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Timepoint [1]
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Time from study enrollment to death from any cause, assessed up to 10 years
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Eligibility
Key inclusion criteria
- Feasibility Phase: Patients must be < 21 years of age at the time of enrollment;
please note: the feasibility phase is complete, effective with amendment #1
- Efficacy Phase: Patients must be =< 40 years of age at the time of enrollment
- Patients with newly diagnosed RMS of any subtype, except adult-type pleomorphic, based
upon institutional histopathologic classification, are eligible to enroll on the study
based upon stage, group, and age, as below
- RMS types included under embryonal rhabdomyosarcoma (ERMS) include those classified in
the 1995 International Classification of Rhabdomyosarcoma (ICR) as ERMS (classic,
spindle cell, and botryoid variants), which are reclassified in the 2013 World Health
Organization (WHO) classification as ERMS (classic, dense and botryoid variants) and
spindle cell/sclerosing RMS (encompassing the historical spindle cell ERMS variant and
the newly recognized sclerosing RMS variant); classification of alveolar
rhabdomyosarcoma (ARMS) in the 2013 WHO classification is the same as in the ICR and
includes classic and solid variants
- ERMS
- Stage 1, group III (non-orbit)
- Stage 3, group I/II
- Stage 2/3, group III
- Stage 4, group IV, < 10 years old
- ARMS:
- Stages 1-3, groups I-III
- Specimen Submission: Patients must have sufficient tissue available for the required
biology study
- Lansky performance status score >= 50 for patients =< 16 years of age; Karnofsky
performance status score >= 50 for patients > 16 years of age
- Peripheral absolute neutrophil count (ANC) >= 750/uL
- Platelet count >= 75,000/uL
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
- 1 month to < 6 months old: 0.4 mg/dl (male), 0.4 mg/dl (female)
- 6 months to < 1 year old: 0.5 mg/dl (male), 0.5 mg/dl (female)
- 1 to < 2 years old: 0.6 mg/dl (male), 0.6 mg/dl (female)
- 2 to < 6 years old: 0.8 mg/dl (male), 0.8 mg/dl (female)
- 6 to < 10 years old: 1 mg/dl (male), 1 mg/dl (female)
- 10 to < 13 years old: 1.2 mg/dl (male), 1.2 mg/dl (female)
- 13 to < 16 years old: 1.5 mg/dl (male), 1.4 mg/dl (female)
- >= 16 years old: 1.7 mg/dl (male), 1.4 mg/dl (female)
- Patients with an elevated serum creatinine due to obstructive hydronephrosis
secondary to tumor are still eligible; however, patients with urinary tract
obstruction by tumor must have unimpeded urinary flow established via diversion
(i.e. percutaneous nephrostomies or ureteric stents) of the urinary tract
- Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
- 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) requirements for human studies must be met.
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Minimum age
No limit
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Maximum age
40
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Patients who have previously received temsirolimus, another mTOR inhibitor, or any
other investigational agent
- Patients who have received any chemotherapy (excluding steroids) and/or RT prior to
this enrollment
- Patients with uncontrolled hyperglycemia
- Patients with uncontrolled hyperlipidemia
- Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation and for
at least 3 months after treatment is completed
- Female patients who are pregnant are not eligible since fetal toxicities or
teratogenic effects have been noted for several of the study drugs; Note: A pregnancy
test is required for female patients of childbearing potential prior to study entry
- Lactating females who plan to breastfeed their infants are not eligible
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/10/2024
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Actual
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Sample size
Target
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Accrual to date
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Final
325
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
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Recruitment hospital [1]
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John Hunter Children's Hospital - Hunter Regional Mail Centre
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Sydney Children's Hospital - Randwick
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The Children's Hospital at Westmead - Westmead
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Queensland Children's Hospital - South Brisbane
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Princess Margaret Hospital for Children - Perth
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Perth Children's Hospital - Perth
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2310 - Hunter Regional Mail Centre
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2031 - Randwick
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2145 - Westmead
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4101 - South Brisbane
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6008 - Perth
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Recruitment postcode(s) [6]
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6009 - Perth
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Funding & Sponsors
Primary sponsor type
Government body
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Name
National Cancer Institute (NCI)
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Ethics approval
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Summary
Brief summary
This randomized phase III trial studies how well combination chemotherapy (vincristine
sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan
hydrochloride or vinorelbine) works compared to combination chemotherapy plus temsirolimus in
treating patients with rhabdomyosarcoma (cancer that forms in the soft tissues, such as
muscle), and has an intermediate chance of coming back after treatment (intermediate risk).
Drugs used work in different ways to stop the growth of tumor cells, either by killing the
cells, by stopping them from dividing, or by stopping them from spreading. Combination
chemotherapy and temsirolimus may stop the growth of tumor cells by blocking some of the
enzymes needed for cell growth. It is not yet known whether chemotherapy plus temsirolimus is
more effective than chemotherapy alone in treating patients with intermediate-risk
rhabdomyosarcoma.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02567435
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Abha A Gupta
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Children's Oncology Group
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02567435
Download to PDF