Trial Outcomes & Findings for Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma (NCT NCT02212574)

Last Updated: 2021-11-05

Results Overview

To determine the feasibility of treating newly diagnosed children with non-metastatic, standard risk, Wnt positive medulloblastoma with a chemotherapy-only approach. Primary outcome measure of this study will be progression-free survival; the number of participants who with progression free survival.

Recruitment status

TERMINATED

Study phase

EARLY_PHASE1

Target enrollment

6 participants

Primary outcome timeframe

3 years

Results posted on

2021-11-05

Participant Flow

Participant milestones

Participant milestones
Measure	Chemotherapy Chemotherapy Cycle A Lomustine (CCNU) given by mouth on Day 1. Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, \& 15. Cisplatin given directly into a vein over 8 hours on Day 1. Cycle lasts 42 days. Chemotherapy Cycle B Cyclophosphamide given into a vein over 1 hour on Days 1 \& 2. MESNA, a drug to protect the bladder from the effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide and repeated at 3 \& 6 hours. Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 \& 8.Cycle lasts 28 days Lomustine: Chemotherapy Cycle A Lomustine (CCNU) given by mouth on Day Vincristine: Chemotherapy Cycle A Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, \& 15. Cisplatin: Chemotherapy Cycle A Cisplatin given directly into a vein over 8 hours on Day 1. Cyclophosphamide: Chemotherapy Cycle B Cyclophosphamide given into a vein over 1 hour on Days 1 \& 2. Mesna: Chemotherapy Cycle B MESNA, a drug to protect the bladder from the effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide and repeated at 3 \& 6 hours. Vincristine: Chemotherapy Cycle B Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 \& 8.
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Chemotherapy n=6 Participants Chemo Cycle A Lomustine (CCNU) given by mouth on Day 1. Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, \& 15. Cisplatin given directly into a vein over 8 hours on Day 1. Cycle lasts 42 days. Chemo Cycle B Cyclophosphamide given into a vein over 1 hour on Days 1 and 2. MESNA, a drug to protect the bladder from effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide, repeated at 3 and 6 hours. Vincristine given directly into a vein directly into the vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 \& 8. Cycle lasts 28 days Lomustine: Chemotherapy Cycle A Lomustine (CCNU) is given by mouth on Day 1. Vincristine: Chemo Cycle A Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1, 8, \& 15. Cisplatin: Chemo Cycle A Cisplatin is given directly into a vein over 8 hours on Day 1. Cyclophosphamide: Chemotherapy Cycle B Cyclophosphamide given into a vein over 1 hour on Days 1 \& 2. Mesna: Chemo Cycle B MESNA, a drug to protect the bladder from the effects of cyclophosphamide, will be given 15 minutes before each dose of cyclophosphamide, repeated at 3 \& 6 hours. Vincristine: Chemo Cycle B Vincristine given directly into a vein (IV) over one minute or using a minibag over several minutes by some institutions on Days 1 \& 8.
Age, Categorical <=18 years	6 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	7 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 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	3 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	6 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 years

Population: This outcome was not assessed because some patients had a relapse and study was abruptly terminated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 years

Population: This outcome was not assessed because some patients had a relapse and study was abruptly terminated.

To evaluate the patterns of failure in those children that do not have progressive disease, progression free survival and overall survival.

Outcome measures

Outcome data not reported

Adverse Events

All Participants

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Kenneth Cohen

Johns Hopkins University

Phone: (410) 614-5055

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place