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NCT02017964 · CITED IN SOURCE DOCUMENTS

Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
PHASE2
Status at capture
COMPLETED
Registry last update
2023-01-09

This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma. Drugs used in chemotherapy, such as vincristine sulfate, cyclophosphamide, methotrexate, etoposide, and carboplatin, 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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Progression-free Survival (PFS)

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outcome · POSTED

Overall Survival (OS)

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outcome · POSTED

Event-free Survival (EFS)

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outcome · POSTED

Percentage of Patients With Responses at 189 Days

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outcome · POSTED

Percentage of Patients With Responses at 273 Days

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outcome · NOT POSTED

Feasibility of Rapid Central Pathology Screening Review Defined as Success Rate of Timely Central Pathology Review Based on the Expectation That at Least 95% of the Cases Will be Reviewed Within 10 Days

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outcome · NOT POSTED

Molecular Profile

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outcome · NOT POSTED

Change in Neurocognitive and Adaptive Functioning Assessed Using Full Scale IQ Score (FSIQ) and General Adaptive Composite Score (GAC)

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * Patients must be newly diagnosed and have a confirmed histologic diagnosis of nodular desmoplastic (ND) medulloblastoma or medulloblastoma with extensive nodularity (MBEN) from rapid central pathology screening review; please note: patients with Gorlin syndrome are eligible * Patient must have negative lumbar cerebrospinal fluid (CSF) cytology (lumbar CSF must be obtained unless medically contraindicated); CSF cytology for staging should be performed preferably no sooner than 14 days post operatively to avoid false positive CSF, ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study; Note: patients with positive CSF cytology obtained prior to 14 days after surgery may have cytology repeated to determine eligibility and final CSF status * Patients must have: * Pre-operative cranial magnetic resonance imaging (MRI) (recommended with gadolinium) or pre-operative computed tomography (CT) (recommended with contrast) * Post-operative cranial MRI with and without gadolinium within 72 hours of surgery * Spinal MRI pre-op with and without gadolinium or post-op with and without gadolinium preferably within 72 hours of surgery * Patients must be enrolled on study within 31 days of definitive surgical resection at which time tissue is acquired to determine a diagnosis; patients must be enrolled before treatment begins; the date protocol therapy is projected to start must be no later than five (5) calendar days after the date of study enrollment; patients who are started on protocol therapy on a Phase II study prior to study enrollment will be considered ineligible * Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2; use Lansky for patients =\< 16 years of age * Patients must have a life expectancy of \>= 8 weeks * Patients who are receiving dexamethasone must be on a stable dose for at least 1 week prior to study entry * Peripheral absolute neutrophil count (ANC) \>= 1000/uL * Platelet count \>= 100,000/uL (transfusion independent) * Hemoglobin \>= 10.0 g/dL (may receive red blood cell \[RBC\] transfusions) * 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: 0.4 mg/dL * 6 months to \< 1 year: 0.5 mg/dL * 1 to \< 2 years: 0.6 mg/dL * 2 to \< 6 years: 0.8 mg/dL * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age * 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) for age * Central nervous system function defined as: * Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled * Patients must not be in status, coma or assisted ventilation prior to study enrollment Exclusion Criteria: * Patients with metastatic disease by either MRI evaluation (brain and spine) or lumbar CSF cytology are not eligible * Patients must not have received any prior tumor-directed therapy other than surgical intervention and corticosteroids
healthy Volunteers
false
maximum Age
47 Months
sex
ALL
std Ages
  1. CHILD
Treatment arms and interventions
arm Groups
  1. description
    INDUCTION THERAPY: Patients receive vincristine sulfate IV over 1 minute or infused via minibag on days 1, 15, and 29; cyclophosphamide IV over 1 hour on days 1-3; methotrexate IV over 24 hours on days 15 and 29; etoposide IV over 60-120 minutes on days 43-45; and carboplatin IV over 1 hour on days 43-45. Treatment repeats every 63 days for 3 courses in the absence of disease progression or unacceptable toxicity. CONTINUATION THERAPY: Patients receive vincristine sulfate IV over 1 minute or infused via minibag on day 1, cyclophosphamide IV over 1 hour on days 1-3, etoposide IV over 60-120 minutes on days 21-23, and carboplatin IV over 1 hour on days 21-23. Treatment repeats every 42 days for 2 courses in the absence of disease progression or unacceptable toxicity.
    intervention Names
    1. Drug: Carboplatin
    2. Other: Cognitive Assessment
    3. Drug: Cyclophosphamide
    4. Drug: Etoposide
    5. Other: Laboratory Biomarker Analysis
    6. Drug: Methotrexate
    7. Drug: Vincristine Sulfate
    label
    Treatment (combination chemotherapy)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Given IV
    name
    Carboplatin
    other Names
    1. Blastocarb
    2. Carboplat
    3. Carboplatin Hexal
    4. Carboplatino
    5. Carboplatinum
    6. Carbosin
    7. Carbosol
    8. Carbotec
    9. CBDCA
    10. Displata
    11. Ercar
    12. JM-8
    type
    DRUG
  2. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Optional ancillary studies
    name
    Cognitive Assessment
    type
    OTHER
  3. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Given IV
    name
    Cyclophosphamide
    other Names
    1. (-)-Cyclophosphamide
    2. 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate
    3. Carloxan
    4. Ciclofosfamida
    5. Ciclofosfamide
    6. Cicloxal
    7. Clafen
    8. Claphene
    9. CP monohydrate
    10. CTX
    11. CYCLO-cell
    12. Cycloblastin
    type
    DRUG
  4. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Given IV
    name
    Etoposide
    other Names
    1. Demethyl Epipodophyllotoxin Ethylidine Glucoside
    2. EPEG
    3. Lastet
    4. Toposar
    5. Vepesid
    6. VP 16
    7. VP 16-213
    8. VP-16
    9. VP-16-213
    10. VP16
    type
    DRUG
  5. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Correlative studies
    name
    Laboratory Biomarker Analysis
    type
    OTHER
  6. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Given IV
    name
    Methotrexate
    other Names
    1. Abitrexate
    2. Alpha-Methopterin
    3. Amethopterin
    4. Brimexate
    5. CL 14377
    6. CL-14377
    7. Emtexate
    8. Emthexat
    9. Emthexate
    10. Farmitrexat
    11. Fauldexato
    12. Folex
    type
    DRUG
  7. arm Group Labels
    1. Treatment (combination chemotherapy)
    description
    Given IV
    name
    Vincristine Sulfate
    other Names
    1. Kyocristine
    2. Leurocristine Sulfate
    3. Leurocristine, sulfate
    4. Oncovin
    5. Vincasar
    6. Vincosid
    7. Vincrex
    8. Vincristine, sulfate
    type
    DRUG
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
26
type
ACTUAL
Registered outcome plans (not posted results)
other Outcomes
  1. description
    At the end of the trial the feasibility of such a prescreening process will be assessed by reporting the percentage and the associated confidence interval of cases where the central review was obtained within 10 days from receipt of slides.
    measure
    Feasibility of Rapid Central Pathology Screening Review Defined as Success Rate of Timely Central Pathology Review Based on the Expectation That at Least 95% of the Cases Will be Reviewed Within 10 Days
    time Frame
    Up to 10 days
  2. description
    Contingency table analysis will be used to examine the association of molecular profile with histology.
    measure
    Molecular Profile
    time Frame
    Up to 2 years
  3. description
    Changes will be described via paired tests and confidence intervals both for FSIQ and GAC in order to capture any deterioration over time.
    measure
    Change in Neurocognitive and Adaptive Functioning Assessed Using Full Scale IQ Score (FSIQ) and General Adaptive Composite Score (GAC)
    time Frame
    Baseline to up to 60 months
primary Outcomes
  1. description
    Progression-free survival (PFS) is defined as the time interval from diagnosis to the earliest of disease progression/recurrence or death from any cause, or to the date of last follow-up for patients without events. PFS was estimated using the method of Kaplan and Meier. 2-year estimates are reported with 95% CI's.
    measure
    Progression-free Survival (PFS)
    time Frame
    2 years from diagnosis
  2. description
    Overall Survival (OS) is defined as the time from diagnosis to death from any cause, or to the date of last follow-up for survivors. OS was estimated using the method of Kaplan and Meier. 2-year estimates are reported with 95% CI's, as the data are not mature to 72 months.
    measure
    Overall Survival (OS)
    time Frame
    Assessed up to 72 months, reported at 2 years from diagnosis
  3. description
    Event-free survival (EFS) is defined as the time from diagnosis to the earliest of disease progression/recurrence, second malignancy or death from any cause, or to the date of last follow-up for patients without events. EFS was estimated using the method of Kaplan and Meier. 2-year estimates are reported with 95% CI's.
    measure
    Event-free Survival (EFS)
    time Frame
    2 years from diagnosis
  4. description
    The percentage of patients with complete response (CR) at the end of induction (\~189 days) was reported and presented with the associated exact 95% confidence interval.
    measure
    Percentage of Patients With Responses at 189 Days
    time Frame
    189 days from start of treatment
  5. description
    The percentage of patients with complete response (CR) at the end of therapy (\~273 days) was reported and presented with the associated exact 95% confidence interval.
    measure
    Percentage of Patients With Responses at 273 Days
    time Frame
    273 days from start of treatment
Full study description
brief Summary
This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma. Drugs used in chemotherapy, such as vincristine sulfate, cyclophosphamide, methotrexate, etoposide, and carboplatin, 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.
detailed Description
PRIMARY OBJECTIVES: I. Estimate of the progression free survival (PFS) distribution for patients 0-\<4 years of age with M0 desmoplastic medulloblastoma (nodular desmoplastic or medulloblastoma with extensive nodularity) treated with the modified HIT SKK regimen (excluding the use of intraventricular methotrexate). OTHER PRE-SPECIFIED OBJECTIVES: I. Evaluate the feasibility of a rapid central pathology screening review for treatment allocation according to histology and assess agreement between institutional and central pathology review diagnoses as well as among central pathology review diagnoses. II. Prospectively evaluate the molecular profile of nodular desmoplastic (ND)/medulloblastoma with extensive nodularity (MBEN) medulloblastoma in young children. III. Monitor and describe the neurocognitive and adaptive functioning of young children with ND/MBEN medulloblastoma treated on this protocol using the ALTE07C1 protocol. OUTLINE: INDUCTION THERAPY: Patients receive vincristine sulfate intravenously (IV) over 1 minute or infused via minibag on days 1, 15, and 29; cyclophosphamide IV over 1 hour on days 1-3; methotrexate IV over 24 hours on days 15 and 29; etoposide IV over 60-120 minutes on days 43-45; and carboplatin IV over 1 hour on days 43-45. Treatment repeats every 63 days for 3 courses in the absence of disease progression or unacceptable toxicity. CONTINUATION THERAPY: Patients receive vincristine sulfate IV over 1 minute or infused via minibag on day 1, cyclophosphamide IV over 1 hour on days 1-3, etoposide IV over 60-120 minutes on days 21-23, and carboplatin IV over 1 hour on days 21-23. Treatment repeats every 42 days for 2 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 3, 6, 9, 12, 16, 20, 24, 36, 48, 60, and 72 months.
Source references
Source notices and limitations
    Discovery and provenance
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      occurrences
      1. context exact
        177;6%) for patients in the nondesmoplastic group (<em>P</em> &lt; .0001 for both EFS and OS comparisons).</li></ul></div></li><li>The <a href="/clinicaltrials/NCT02017964">COG-ACNS1221 (NCT02017964)</a> study tested systemic chemotherapy that was identical to the chemotherapy used in the German HIT 2000 trial, except for the omi
        end
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      2. context exact
        nondesmoplastic group (<em>P</em> &lt; .0001 for both EFS and OS comparisons).</li></ul></div></li><li>The <a href="/clinicaltrials/NCT02017964">COG-ACNS1221 (NCT02017964)</a> study tested systemic chemotherapy that was identical to the chemotherapy used in the German HIT 2000 trial, except for the omission of intraventricular m
        end
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      source retrieved at
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      source string sha256
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      source title
      Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Health Professional Version
      source update dates
      1. context
        Updated: April 11, 2025
        datetime
        2025-04-11T12:00:00Z
        display
        April 11, 2025

    Preserved source evidence · Not medical advice