NCT02017964|CompletedPhase 2Results

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

A Phase II Study for the Treatment of Non-metastatic Nodular Desmoplastic Medulloblastoma in Children Less Than 4 Years of Age

Children's Oncology Group ClinicalTrials.gov

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6 other identifiers

ACNS1221NCI-2013-02379COG-ACNS1221s15-00476U10CA180886U10CA098543

Study Type

interventional

Target

Locations

4 countries

Sites

173

Timeline

RegisteredDec 2013

StartedDec 2013

CompletionDec 2022

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_2

Timeline

Completed

Started Dec 2013

Longer than P75 for phase_2

Geographic Reach

4 countries

173 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

9 years study duration

First Submitted

Initial submission to the registry

December 13, 2013

Completed

10 days until next milestone

First Posted

Study publicly available on registry

December 23, 2013

Completed

1 day until next milestone

Study Start

First participant enrolled

December 24, 2013

Completed

4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2018

Completed

1 year until next milestone

Results Posted

Study results publicly available

April 4, 2019

Completed

3.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2022

Completed

Last Updated

January 9, 2023

Status Verified

January 1, 2023

Enrollment Period

4.3 years

First QC Date

December 13, 2013

Results QC Date

March 12, 2019

Last Update Submit

January 4, 2023

Conditions

Desmoplastic/Nodular Medulloblastoma Medulloblastoma Medulloblastoma With Extensive Nodularity Nevoid Basal Cell Carcinoma Syndrome

Outcome Measures

Primary Outcomes (5)

Progression-free Survival (PFS)
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.
2 years from diagnosis
Overall Survival (OS)
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.
Assessed up to 72 months, reported at 2 years from diagnosis
Event-free Survival (EFS)
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.
2 years from diagnosis
Percentage of Patients With Responses at 189 Days
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.
189 days from start of treatment
Percentage of Patients With Responses at 273 Days
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.
273 days from start of treatment

Other Outcomes (3)

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
Up to 10 days
Molecular Profile
Up to 2 years
Change in Neurocognitive and Adaptive Functioning Assessed Using Full Scale IQ Score (FSIQ) and General Adaptive Composite Score (GAC)
Baseline to up to 60 months

Study Arms (1)

Treatment (combination chemotherapy)

EXPERIMENTAL

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.

Drug: CarboplatinOther: Cognitive AssessmentDrug: CyclophosphamideDrug: EtoposideOther: Laboratory Biomarker AnalysisDrug: MethotrexateDrug: Vincristine Sulfate

Interventions

CarboplatinDRUG

Given IV

Also known as: Blastocarb, Carboplat, Carboplatin Hexal, Carboplatino, Carboplatinum, Carbosin, Carbosol, Carbotec, CBDCA, Displata, Ercar, JM-8, Nealorin, Novoplatinum, Paraplatin, Paraplatin AQ, Paraplatine, Platinwas, Ribocarbo