Trial Outcomes & Findings for Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma (NCT NCT02017964)

NCT ID: NCT02017964

Last Updated: 2023-01-09

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

26 participants

Primary outcome timeframe

2 years from diagnosis

Results posted on

2023-01-09

Participant Flow

This study opened for entry on December 24, 2013, and the first patient was enrolled on April 22, 2014. The study enrolled 26 patients before being closed early due to inferior efficacy as of July 27, 2016. 25 of the 26 patients enrolled were deemed to be eligible

Participant milestones

Participant milestones
Measure	All Patients (Combination Chemotherapy) All patients enrolled on the study. Patients received 3-5 cycles of therapy which included cyclophosphamide (800 mg/m2/day), vincristine (1.5 mg/m2/day), methotrexate (5,000 mg/m2), etoposide (150 mg/m2/dose) and carboplatin (200 mg/m2/day IV).
Overall Study STARTED	26
Overall Study COMPLETED	25
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients (Combination Chemotherapy) All patients enrolled on the study. Patients received 3-5 cycles of therapy which included cyclophosphamide (800 mg/m2/day), vincristine (1.5 mg/m2/day), methotrexate (5,000 mg/m2), etoposide (150 mg/m2/dose) and carboplatin (200 mg/m2/day IV).
Overall Study Patient deemed ineligible	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Eligible Patients (Combination Chemotherapy) n=26 Participants All eligible patients enrolled on the study
Age, Categorical <=18 years	26 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	19.7 months (at time of dx) n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	20 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	23 participants n=5 Participants
Region of Enrollment Canada	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years from diagnosis

Population: 26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient was excluded from all analyses.

Outcome measures

Outcome measures
Measure	All Eligible Patients (Combination Chemotherapy) n=25 Participants All eligible patients enrolled on the study
Progression-free Survival (PFS)	52.0 percent probability Interval 32.4 to 71.6

PRIMARY outcome

Timeframe: Assessed up to 72 months, reported at 2 years from diagnosis

Population: 26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient was excluded from all analyses.

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.

Outcome measures

Outcome measures
Measure	All Eligible Patients (Combination Chemotherapy) n=25 Participants All eligible patients enrolled on the study
Overall Survival (OS)	92.0 percent probability Interval 80.8 to 100.0

PRIMARY outcome

Timeframe: 2 years from diagnosis

Population: 26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient is excluded from all analyses.

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.

Outcome measures

Outcome measures
Measure	All Eligible Patients (Combination Chemotherapy) n=25 Participants All eligible patients enrolled on the study
Event-free Survival (EFS)	52.0 percent probability Interval 32.4 to 71.6

PRIMARY outcome

Timeframe: 189 days from start of treatment

Population: 26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient was excluded from all analyses.

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.

Outcome measures

Outcome measures
Measure	All Eligible Patients (Combination Chemotherapy) n=25 Participants All eligible patients enrolled on the study
Percentage of Patients With Responses at 189 Days	92.0 Percentage of patients Interval 75.6 to 98.6

PRIMARY outcome

Timeframe: 273 days from start of treatment

Population: 26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient is excluded from all analyses.

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.

Outcome measures

Outcome measures
Measure	All Eligible Patients (Combination Chemotherapy) n=25 Participants All eligible patients enrolled on the study
Percentage of Patients With Responses at 273 Days	88.0 Percentage of patients Interval 69.7 to 96.7

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 10 days

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.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 2 years

Contingency table analysis will be used to examine the association of molecular profile with histology.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to up to 60 months

Changes will be described via paired tests and confidence intervals both for FSIQ and GAC in order to capture any deterioration over time.

Outcome measures

Outcome data not reported

Adverse Events

All Eligible Patients (Combination Chemotherapy)

Serious events: 2 serious events

Other events: 17 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	All Eligible Patients (Combination Chemotherapy) n=25 participants at risk All eligible patients enrolled on the study
General disorders Death NOS	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Other, Progression Of Medulloblastoma	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.

Other adverse events

Other adverse events
Measure	All Eligible Patients (Combination Chemotherapy) n=25 participants at risk All eligible patients enrolled on the study
Blood and lymphatic system disorders Febrile neutropenia	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Blood and lymphatic system disorders Leukocytosis	4.0% 1/25 • Number of events 4 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Ear and labyrinth disorders Hearing impaired	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Infections and infestations Meningitis	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Investigations Lymphocyte count decreased	28.0% 7/25 • Number of events 18 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Investigations Neutrophil count decreased	56.0% 14/25 • Number of events 36 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Investigations Platelet count decreased	28.0% 7/25 • Number of events 13 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Investigations White blood cell decreased	44.0% 11/25 • Number of events 24 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Metabolism and nutrition disorders Hypernatremia	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
Metabolism and nutrition disorders Hypokalemia	4.0% 1/25 • Number of events 1 • 1 year Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 626-446-0064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Must obtain prior Sponsor approval
Publication restrictions are in place

Restriction type: OTHER