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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00602667

Registration number

NCT00602667

Ethics application status

Date submitted

10/01/2008

Date registered

28/01/2008

Date last updated

27/11/2024

Titles & IDs

Public title

Risk-Adapted Therapy for Young Children With Embryonal Brain Tumors, Choroid Plexus Carcinoma, High Grade Glioma or Ependymoma

Scientific title

Risk-Adapted Therapy for Young Children With Embryonal Brain Tumors, Choroid Plexus Carcinoma, High Grade Glioma or Ependymoma

Secondary ID [1]

R01CA154619

Secondary ID [2]

SJYC07

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Brain and Central Nervous System Tumors

Condition category

Condition code

Cancer

Brain

Cancer

Children's - Brain

Cancer

Neuroendocrine tumour (NET)

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Treatment: Drugs - Induction Chemotherapy
Treatment: Drugs - Low-Risk Therapy
Treatment: Drugs - High-Risk Therapy
Treatment: Drugs - Intermediate-Risk Therapy

Experimental: Low-Risk Patients - Patients with GTR/M0 medulloblastoma, nodular desmoplastic or high grade glioma histology will receive induction chemotherapy and low-risk therapy.

Note: Accrual to the low-risk medulloblastoma cohort is closed as of 12/2/2015. Accrual to the low-risk high grade glioma remains open.

Experimental: High-Risk Patients - Patients with CNS metastatic disease will receive induction chemotherapy and high-risk therapy.

Experimental: Intermediate-Risk Therapy - Patients with M0 medulloblastoma or nodular desmoplastic histology with less than a GTR, other histologic diagnoses with no metastatic disease, will receive induction chemotherapy and intermediate-risk therapy.

Treatment: Drugs: Induction Chemotherapy
All patients will receive 4 identical cycles of induction chemotherapy including highdose (5 g/m2 or 2.5g/m2 for patients less than or equal to 31 days of age at enrollment) intravenous methotrexate and standard dose vincristine, cisplatin, and cyclophosphamide.

Treatment: Drugs: Low-Risk Therapy
Induction will be followed by further conventional chemotherapy with carboplatin, cyclophosphamide, and etoposide. After consolidation, patients will receive 6 cycles of oral maintenance chemotherapy with cyclophosphamide, topotecan, and depending on the diagnosis, either erlotinib or etoposide (VP-16).

Treatment: Drugs: High-Risk Therapy
High risk patients will also receive vinblastine with each course of induction chemotherapy. Induction will be followed by either chemotherapy with targeted intravenous topotecan and cyclophosphamide or optional craniospinal irradiation (CSI). CSI will be offered only to patients who reach 3 years of age by the end of induction only. After consolidation, all patients will receive 6 cycles of oral maintenance chemotherapy with cyclophosphamide, topotecan, and depending on the diagnosis, either erlotinib or etoposide (VP-16).

Treatment: Drugs: Intermediate-Risk Therapy
Induction will be followed by consolidation focal radiotherapy (RT) to the tumor bed. Patients less than 12 months old upon completion of induction will receive low risk chemotherapy to delay RT until the age of 12 months. After consolidation, patients will receive 6 cycles of oral maintenance chemotherapy with cyclophosphamide, topotecan, and depending on the diagnosis, either erlotinib or etoposide (VP-16).

Note: The option to receive focal proton beam irradiation was suspended 10/29/2015. Focal photon beam irradiation continues as part of the treatment plan.

Intervention code [1]

Treatment: Drugs

Comparator / control treatment

Control group

Outcomes

Primary outcome [1]

Percent Probability of Progression-free Survival (PFS) for Medulloblastoma Patients

Timepoint [1]

From date on treatment until date of first progression or relapse or disease related death or date of last contact, estimated at 1 year after treatment

Primary outcome [2]

Percent Probability of Progression-free Survival (PFS) for Medulloblastoma Patients by DNA Methylation Subgroup

Timepoint [2]

From date on treatment to date of first progression or relapse or disease related death or date of last contact, estimated at 1 year after treatment

Primary outcome [3]

Percent Probability of Event-free Survival (EFS) for Medulloblastoma Patients

Timepoint [3]

From date on treatment to date of first progression, relapse, second malignancy or death from any cause or to date of last contact, estimated at 1 year after

Secondary outcome [1]

Number of Participants With Chromosomal Abnormalities

Timepoint [1]

Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment

Secondary outcome [2]

Numbers of Patients With Gene Alterations

Timepoint [2]

Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment

Secondary outcome [3]

Numbers of Patients With Molecular Abnormalities by Tumor Type

Timepoint [3]

Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment

Secondary outcome [4]

Number of Successful Collections for Frozen and Fixed Tumor Samples

Timepoint [4]

Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment

Secondary outcome [5]

Event-free Survival (EFS) Compared to Historical Controls

Timepoint [5]

From date on treatment until date of first event (progression, second malignancy or death) or until date of last contact, assessed up to 10 years

Secondary outcome [6]

Overall Survival (OS) Compared to Historical Controls

Timepoint [6]

1 year after treatment initiation of last patient

Secondary outcome [7]

Percentage of Patients With Objective Responses Rate to Induction Chemotherapy

Timepoint [7]

From on-study date to 2 months after completion of induction chemotherapy (up to 4 months after on-study date)

Secondary outcome [8]

Feasibility and Toxicity of Administering Vinblastine With Induction Chemotherapy for Patients With Metastatic Disease as Measured by the Percentage of Courses Delayed for More Than 7 Days Due to Toxicity

Timepoint [8]

From on-study date up to 4 months after on-study date

Secondary outcome [9]

Feasibility and Toxicity of Administering Consolidation Therapy Including Cyclophosphamide and Pharmacokinetically Targeted Topotecan to Patients With Metastatic Disease Based on the Percentage of Courses Delayed for More Than 7 Days Due to Toxicity

Timepoint [9]

At completion of consolidation therapy (up to 6 months after on-study date)

Secondary outcome [10]

Percent of Patients With Sustained Objective Responses Rate After Consolidation

Timepoint [10]

8 weeks after completion of consolidation therapy (up to 8 months after on-study date)

Secondary outcome [11]

Feasibility and Toxicity of Administering Oral Maintenance Therapy in Children <3 Years of Age as Measured by the Percentage of Total Scheduled Maintenance Doses Received

Timepoint [11]

From start of oral maintenance therapy (approximately 6 months after on-study date) to completion of oral maintenance therapy (up to 1 year after on-study date)

Secondary outcome [12]

Percent of PET Scans With Loss of Signal Intensity

Timepoint [12]

Up to 3 times during RT consolidation

Secondary outcome [13]

Concentration of Cerebrospinal Fluid Neurotransmitters

Timepoint [13]

Baseline, at the completion of therapy, and every 12 months up to 36 months after off therapy date

Secondary outcome [14]

Number and Type of Genetic Polymorphisms

Timepoint [14]

At study enrollment (Day 0)

Secondary outcome [15]

Pharmacogenetic Variation on Central Nervous System Transmitters

Timepoint [15]

At study enrollment (Day 0)

Secondary outcome [16]

Number of Participants With Endocrinopathy

Timepoint [16]

Baseline, end of therapy, and at 6- and 24-months after completion of therapy

Secondary outcome [17]

Longitudinal Change in Growth Hormone Secretion

Timepoint [17]

Baseline, end of therapy, and at 6- and 24-months after completion of therapy

Secondary outcome [18]

Methotrexate Clearance in Induction Cycle 1

Timepoint [18]

Pre-infusion and 6, 23, 42, 66 hours from start of Methotrexate (MTX)

Secondary outcome [19]

Methotrexate Clearance in Induction Cycle 2

Timepoint [19]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [20]

Methotrexate Clearance in Induction Cycle 3

Timepoint [20]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [21]

Methotrexate Clearance in Induction Cycle 4

Timepoint [21]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [22]

Methotrexate Volume of Central Compartment in Induction Cycle 1

Timepoint [22]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [23]

Methotrexate Volume of Central Compartment in Induction Cycle 2

Timepoint [23]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [24]

Methotrexate Volume of Central Compartment in Induction Cycle 3

Timepoint [24]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [25]

Methotrexate AUC0-66h in Induction Cycle 1

Timepoint [25]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [26]

Methotrexate AUC0-66h in Induction Cycle 2

Timepoint [26]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [27]

Methotrexate Volume of Central Compartment in Induction Cycle 4

Timepoint [27]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [28]

Methotrexate AUC0-66h in Induction Cycle 3

Timepoint [28]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [29]

Methotrexate AUC0-66h in Induction Cycle 4

Timepoint [29]

Pre-infusion and 6, 23, 42, 66 hours from start of MTX

Secondary outcome [30]

Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 1

Timepoint [30]

42 hours from start of MTX

Secondary outcome [31]

Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 2

Timepoint [31]

42 hours from start of MTX

Secondary outcome [32]

Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 3

Timepoint [32]

42 hours from start of MTX

Secondary outcome [33]

Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 4

Timepoint [33]

42 hours from start of MTX

Secondary outcome [34]

Cyclophosphamide Clearance in Induction Chemotherapy

Timepoint [34]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [35]

Cyclophosphamide Clearance in Consolidation Chemotherapy Cycle 1

Timepoint [35]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [36]

Cyclophosphamide Clearance in Consolidation Chemotherapy Cycle 2

Timepoint [36]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [37]

Cyclophosphamide Apparent Oral Clearance in Maintenance Chemotherapy Cycle A1

Timepoint [37]

Pre-dose, 0.5, 1.75, 3 and 6 hours post-dose

Secondary outcome [38]

Cyclophosphamide AUC0-24h in Induction Chemotherapy

Timepoint [38]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [39]

Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 1

Timepoint [39]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [40]

4-OH Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 1

Timepoint [40]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [41]

Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 2

Timepoint [41]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [42]

Cyclophosphamide AUC0-24h in Maintenance Chemotherapy Cycle A1

Timepoint [42]

Pre-dose, 0.5, 1.75, 3, 6, and 24 hours post-dose

Secondary outcome [43]

4-OH Cyclophosphamide AUC0-24h in Induction Chemotherapy

Timepoint [43]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [44]

4-OH Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 2

Timepoint [44]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [45]

4-OH Cyclophosphamide AUC0-24h in Maintenance Chemotherapy Cycle A1

Timepoint [45]

Pre-dose, 0.5, 1.75, 3, 6, and 24 hours post-dose

Secondary outcome [46]

CEPM AUC0-24h in Induction Chemotherapy

Timepoint [46]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [47]

CEPM AUC0-24h in Consolidation Chemotherapy Cycle 1

Timepoint [47]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [48]

CEPM AUC0-24h in Consolidation Chemotherapy Cycle 2

Timepoint [48]

Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion

Secondary outcome [49]

CEPM AUC0-24h in Maintenance Chemotherapy Cycle A1

Timepoint [49]

Pre-dose, 0.5, 1.75, 3, 6, and 24 hours post-dose

Secondary outcome [50]

Participants With Empirical Dosage Achieving Target System Exposure of Intravenous Topotecan

Timepoint [50]

Pre-infusion, 5 min., 1, and 3 hours from end of infusion

Secondary outcome [51]

Participants With PK-guided Dosage Adjustment Achieving Target System Exposure of Intravenous Topotecan

Timepoint [51]

Pre-infusion, 5 min., 1, and 3 hours from end of infusion

Secondary outcome [52]

Topotecan Clearance in Consolidation Chemotherapy

Timepoint [52]

Pre-infusion, 5 min., 1, and 3 hours from end of infusion

Secondary outcome [53]

Topotecan Apparent Oral Clearance in Maintenance Chemotherapy

Timepoint [53]

Pre-dose, 0.25, 1.5 and 6 hours post-dose

Secondary outcome [54]

Topotecan AUC0-24h in Consolidation Chemotherapy

Timepoint [54]

Pre-infusion, 5 min., 1, 3, and 24 hours from end of infusion

Secondary outcome [55]

Topotecan AUC0-24h in Maintenance Chemotherapy

Timepoint [55]

Pre-dose, 0.25, 1.5, 6, and 24 hours post-dose

Secondary outcome [56]

Erlotinib Apparent Oral Clearance

Timepoint [56]

Pre-dose, 1, 2, 4, 8, and 24 hours post-dose

Secondary outcome [57]

Erlotinib Apparent Volume of Central Compartment

Timepoint [57]

Pre-dose, 1, 2, 4, 8, and 24 hours post-dose

Secondary outcome [58]

Erlotinib AUC0-24h

Timepoint [58]

Pre-dose, 1, 2, 4, 8, and 24 hours post-dose

Secondary outcome [59]

OSI-420 AUC0-24h

Timepoint [59]

Pre-dose, 1, 2, 4, 8, and 24 hours post-dose

Secondary outcome [60]

Rate of Local Disease Progression

Timepoint [60]

1 year after completion of radiation therapy for last patient

Secondary outcome [61]

Rate of Distant Disease Progression

Timepoint [61]

1 year after completion of radiation therapy for last patient

Secondary outcome [62]

Neurocognitive Performance Related to Global Cognitive Functioning as Measured by Cognitive Composite Scores and Estimated IQ Scores

Timepoint [62]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [63]

Neurocognitive Performance Related to Attention as Measured by Attention Problems T-scores

Timepoint [63]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [64]

Neurocognitive Performance Related to Processing Speed as Measured by Visual Matching Standard Scores

Timepoint [64]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [65]

Neurocognitive Performance Related to Executive Functioning as Measured by Global Executive Composite T-scores

Timepoint [65]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [66]

Neurocognitive Performance Related to Working Memory as Measured by Working Memory T-scores

Timepoint [66]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [67]

Neurocognitive Performance Related to Verbal Fluency as Measured by Retrieval Fluency Standard Scores

Timepoint [67]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [68]

Neurocognitive Performance Related to Visual-spatial Reasoning as Measured by Visual Motor Integration (VMI) T-scores

Timepoint [68]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [69]

Neurocognitive Performance Related to Visual-spatial Reasoning as Measured by Visual Perception T-scores

Timepoint [69]

Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy

Secondary outcome [70]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [70]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [71]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [71]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [72]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [72]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [73]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [73]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [74]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [74]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [75]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [75]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [76]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [76]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [77]

Change in Neurostructure, Especially White Matter Volume and Integrity

Timepoint [77]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [78]

Change in Quantitative Magnetic Resonance (MR) Measures in the Frontal Lobe

Timepoint [78]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Secondary outcome [79]

Change in Quantitative MR Measures in the Right Frontal-parietal Regions

Timepoint [79]

From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)

Eligibility

Key inclusion criteria

Histologically confirmed newly diagnosed CNS tumors of any of the following :

* Medulloblastoma (all histologic subtypes, including medullomyoblastoma and melanotic medulloblastoma)
* Supratentorial primitive neuroectodermal tumor (PNET) (including CNS neuroblastoma or ganglioneuroblastoma, medulloepithelioma, and ependymoblastoma)
* Pineoblastoma
* Atypical teratoid rhabdoid tumor (ATRT)
* Choroid plexus carcinoma
* High grade glioma (including anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic ganglioglioma, pleomorphic xanthoastrocytoma with anaplastic features, high-grade astroblastoma , anaplastic pilocytic astrocytoma, malignant glioneuronal tumor, glioblastoma multiforme), or gliosarcoma,
* Ependymoma (including all ependymoma histological variants)
* Age < 3 years at time of diagnosis for all histological diagnosis. Medulloblastoma patients = 3 and < 5years old at diagnosis who have non-metastatic disease with no more than 1cm2 of residual tumor are also eligible.

* Meets criteria for 1 of the following risk groups:
* Low-risk group:

* Histologically confirmed nodular desmoplastic medulloblastoma, including medulloblastoma with extensive nodularity

* Focal areas of anaplasia or other atypical features suggesting more aggressive phenotype in a tumor otherwise considered nodular desmoplastic should be treated on the intermediate-risk group, with final risk stratification at the discretion of principal investigator and study pathologist
* No evidence of CNS metastasis 7 to 28 days after surgery by MRI and cytologic examination of lumbar cerebrospinal fluid (CSF)

* Ventricular CSF from a shunt or Ommaya reservoir may be used to rule out M1 disease when lumbar puncture is medically contraindicated
* Intermediate-risk group assignment when there is no other evidence of metastasis and CSF sampling is not possible
* Gross total resection, defined as residual tumor or imaging abnormality (not definitive for residual tumor) with a size of < 1 cm2 confirmed on postoperative CT scan or MRI
* Brain stem invasion by the tumor in the absence of imaging evidence of residual tumor (tumor size < 1 cm2) and otherwise meets criteria for the low-risk group, the patient will be classified as low-risk
* Desmoplastic medulloblastoma patients who are =3 -<5 years of age will NOT be eligible for the low risk arm of the protocol.
* Intermediate-risk group:

* Histologically confirmed nodular desmoplastic medulloblastoma with less than gross total resection and no evidence of metastasis
* Any eligible histologic diagnosis other than desmoplastic medulloblastoma with no evidence of CNS metastasis
* Medulloblastoma patients who are =3 and < 5 yrs of age irrespective of histology and with no evidence of CNS metastasis
* High-risk group:

* Any eligible histologic diagnosis with evidence of CNS metastasis
* Patients with extraneural metastasis are eligible for treatment on the high-risk group

PATIENT CHARACTERISTICS:

* Lansky performance status = 30 (except for posterior fossa syndrome)
* WBC > 2,000/mm3
* Platelets > 50,000/mm3 (without support)
* Hemoglobin > 8 g/dL (with or without support)
* ANC > 500/mm3
* Serum creatinine < 3 times upper limit of normal (ULN)
* ALT < 5 times ULN
* Total bilirubin < 3 times ULN

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics
* No more than 31 days since prior definitive surgery
* No prior radiotherapy or chemotherapy other than corticosteroid therapy

Minimum age

No limit

Maximum age

5 Years

Sex

Both males and females

Can healthy volunteers participate?

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 2

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

17/12/2007

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

1/04/2026

Actual

Sample size

Target

Accrual to date

Final

293

Recruitment in Australia

Recruitment state(s)

QLD

Recruitment hospital [1]

Lady Cilento Children's Hospital, Brisbane - Brisbane

Recruitment postcode(s) [1]

4029 - Brisbane

Recruitment outside Australia

Country [1]

United States of America

State/province [1]

California

Country [2]

United States of America

State/province [2]

Minnesota

Country [3]

United States of America

State/province [3]

Tennessee

Country [4]

United States of America

State/province [4]

Texas

Funding & Sponsors

Primary sponsor type

Other

Name

St. Jude Children's Research Hospital

Address

Country

Other collaborator category [1]

Other

Name [1]

University of Florida

Address [1]

Country [1]

Other collaborator category [2]

Government body

Name [2]

National Cancer Institute (NCI)

Address [2]

Country [2]

Other collaborator category [3]

Other

Name [3]

The Pew Charitable Trusts

Address [3]

Country [3]

Ethics approval

Ethics application status

Summary

Brief summary

RATIONALE: In this study a combination of anti-cancer drugs (chemotherapy) is used to treat brain tumors in young children. Using chemotherapy gives the brain more time to develop before radiation is given. The chemotherapy in this study includes the drug methotrexate. This drug was an important part of the two clinical trials which resulted in the best survival results for children less than 3 years of age with medulloblastoma. Most patients treated on this trial will also receive radiation which is carefully targeted to the area of the tumor. This type of radiation (focal conformal or proton beam radiotherapy) may result in fewer problems with thinking and learning than radiation to the whole brain and spinal cord.

PURPOSE: This clinical trial is studying how well giving combination chemotherapy together with radiation therapy works in treating young patients with newly diagnosed central nervous system tumors.

Trial website

https://clinicaltrials.gov/study/NCT00602667

Trial related presentations / publications

Ali JS, Ashford JM, Swain MA, Harder LL, Carlson-Green BL, Miller JM, Wallace J, Kaner RJ, Billups CA, Onar-Thomas A, Merchant TE, Gajjar A, Conklin HM. Predictors of Cognitive Performance Among Infants Treated for Brain Tumors: Findings From a Multisite, Prospective, Longitudinal Trial. J Clin Oncol. 2021 Jul 20;39(21):2350-2358. doi: 10.1200/JCO.20.01687. Epub 2021 May 4.
Kumar R, Smith KS, Deng M, Terhune C, Robinson GW, Orr BA, Liu APY, Lin T, Billups CA, Chintagumpala M, Bowers DC, Hassall TE, Hansford JR, Khuong-Quang DA, Crawford JR, Bendel AE, Gururangan S, Schroeder K, Bouffet E, Bartels U, Fisher MJ, Cohn R, Partap S, Kellie SJ, McCowage G, Paulino AC, Rutkowski S, Fleischhack G, Dhall G, Klesse LJ, Leary S, Nazarian J, Kool M, Wesseling P, Ryzhova M, Zheludkova O, Golanov AV, McLendon RE, Packer RJ, Dunham C, Hukin J, Fouladi M, Faria CC, Pimentel J, Walter AW, Jabado N, Cho YJ, Perreault S, Croul SE, Zapotocky M, Hawkins C, Tabori U, Taylor MD, Pfister SM, Klimo P Jr, Boop FA, Ellison DW, Merchant TE, Onar-Thomas A, Korshunov A, Jones DTW, Gajjar A, Ramaswamy V, Northcott PA. Clinical Outcomes and Patient-Matched Molecular Composition of Relapsed Medulloblastoma. J Clin Oncol. 2021 Mar 1;39(7):807-821. doi: 10.1200/JCO.20.01359. Epub 2021 Jan 27.
Robinson GW, Rudneva VA, Buchhalter I, Billups CA, Waszak SM, Smith KS, Bowers DC, Bendel A, Fisher PG, Partap S, Crawford JR, Hassall T, Indelicato DJ, Boop F, Klimo P, Sabin ND, Patay Z, Merchant TE, Stewart CF, Orr BA, Korbel JO, Jones DTW, Sharma T, Lichter P, Kool M, Korshunov A, Pfister SM, Gilbertson RJ, Sanders RP, Onar-Thomas A, Ellison DW, Gajjar A, Northcott PA. Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial. Lancet Oncol. 2018 Jun;19(6):768-784. doi: 10.1016/S1470-2045(18)30204-3. Epub 2018 May 16.

Public notes

Contacts

Principal investigator

Name

Amar Gajjar, MD

Address

St. Jude Children's Research Hospital

Country

Phone

Fax

Contact person for public queries

Name

Address

Country

Phone

Fax

Contact person for scientific queries

Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00602667

Trial Review

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For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00602667