Trial Outcomes & Findings for Molecular-Guided Therapy for Childhood Cancer (NCT NCT02162732)

Last Updated: 2025-11-03

Results Overview

The definition of feasibility is: Enrollment onto study, genomic profile, analysis and report generation completed, tumor board held with treatment decision, treatment review completed, start of treatment, and completion of 1 cycle of therapy.

Recruitment status

COMPLETED

Study phase

Target enrollment

186 participants

Primary outcome timeframe

2 years

Results posted on

2025-11-03

Participant Flow

Participant milestones

Participant milestones
Measure	Guided Therapy A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). Guided Therapy: A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
Overall Study STARTED	186
Overall Study COMPLETED	168
Overall Study NOT COMPLETED	18

Reasons for withdrawal

Reasons for withdrawal
Measure	Guided Therapy A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). Guided Therapy: A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
Overall Study Biopsy not performed.	18

Baseline Characteristics

Molecular-Guided Therapy for Childhood Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Guided Therapy n=144 Participants A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). Guided Therapy: A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
Race (NIH/OMB) Asian	5 Participants n=3 Participants
Age, Categorical <=18 years	134 Participants n=3 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=3 Participants
Age, Categorical >=65 years	0 Participants n=3 Participants
Age, Continuous	11.04 years n=3 Participants
Sex: Female, Male Female	62 Participants n=3 Participants
Sex: Female, Male Male	82 Participants n=3 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	13 Participants n=3 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	108 Participants n=3 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	23 Participants n=3 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=3 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=3 Participants
Race (NIH/OMB) Black or African American	10 Participants n=3 Participants
Race (NIH/OMB) White	93 Participants n=3 Participants
Race (NIH/OMB) More than one race	0 Participants n=3 Participants
Race (NIH/OMB) Unknown or Not Reported	36 Participants n=3 Participants
Tumor Type Neuroblastoma	31 Participants n=3 Participants
Tumor Type CNS Tumor	41 Participants n=3 Participants
Tumor Type Rare Tumor	72 Participants n=3 Participants

PRIMARY outcome

Timeframe: 2 years

Outcome measures

Outcome measures
Measure	Neuroblastoma n=31 Participants Subjects on study with a diagnosis of Neuroblastoma	CNS Tumors n=41 Participants Subjects on study with a diagnosis of a CNS Tumor which included, but was not limited to, Ependymoma, Glioblastoma, Diffuse Midline Glioma, and Other CNS Tumors.	Rare Tumors n=72 Participants Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors.
Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers.	31 Days Interval 18.0 to 75.0	48 Days Interval 18.0 to 146.0	35 Days Interval 20.0 to 98.0

SECONDARY outcome

Timeframe: Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.

Population: All subjects that received at least one dose of study therapy.

To determine the safety of allowing a molecular tumor board to determine individualized treatment plans

Outcome measures

Outcome measures
Measure	Neuroblastoma n=144 Participants Subjects on study with a diagnosis of Neuroblastoma	CNS Tumors Subjects on study with a diagnosis of a CNS Tumor which included, but was not limited to, Ependymoma, Glioblastoma, Diffuse Midline Glioma, and Other CNS Tumors.	Rare Tumors Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors.
Number of Participants With an Unexpected Adverse Events as a Measure of Safety	6 Participants	—	—

SECONDARY outcome

Timeframe: Followed until off therapy, generally 4 years

Population: Having at least one disease evaluation following completion of at least one cycle of molecular-guided therapy

To determine the activity of treatments chosen based on Overall response rate (ORR) using RESIST criteria. The assessment of response will include the initial measurable targets and will be performed after cycle 2, then after every other cycle. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT or MRI imaging and/or by MIBG or PET scans: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease, At least a 20% increase in the sum of the disease measurements for measurable lesions, Stable Disease, Neither sufficient decrease to qualify for PR or sufficient increase to qualify for PD from study entry. Overall Response (OR) = CR + PR.

Outcome measures

Outcome measures
Measure	Neuroblastoma n=27 Participants Subjects on study with a diagnosis of Neuroblastoma	CNS Tumors n=37 Participants Subjects on study with a diagnosis of a CNS Tumor which included, but was not limited to, Ependymoma, Glioblastoma, Diffuse Midline Glioma, and Other CNS Tumors.	Rare Tumors n=60 Participants Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors.
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans. Stable Disease	13 Participants	10 Participants	18 Participants
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans. Progressive Disease	9 Participants	10 Participants	24 Participants
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans. Complete Response	4 Participants	2 Participants	5 Participants
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans. No Evidence of Disease	1 Participants	11 Participants	6 Participants
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans. Partial Response	0 Participants	4 Participants	7 Participants

SECONDARY outcome

Timeframe: From date of start of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 4 years

Population: Time on study was measured in days from the date of initial biopsy to date of last administration of study therapy. For subjects with \>1 biopsies and \>1 MGT regimens, the off-therapy date from their last treatment was used.

Time to progression (PFS), defined as the period from the start of the treatment until the criteria for progression are met taking as reference the screening measurements. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as at least a 20% increase in the sum of the disease measurements for measurable lesions, taking as reference the smallest disease measurement recorded since the start of treatment (nadir), and minimum 5 mm increase over the nadir or the appearance of one or more new lesions or appearance of positive bone marrow.

Outcome measures

Outcome measures
Measure	Neuroblastoma n=27 Participants Subjects on study with a diagnosis of Neuroblastoma	CNS Tumors n=37 Participants Subjects on study with a diagnosis of a CNS Tumor which included, but was not limited to, Ependymoma, Glioblastoma, Diffuse Midline Glioma, and Other CNS Tumors.	Rare Tumors n=60 Participants Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors.
Progression Free Survival (PFS) Interval Will be Measured by Days and Compared to the PFS of Previous Chemotherapy Regimens Since Relapse for Each Patient.	153 Days Interval 45.0 to 556.0	283 Days Interval 50.0 to 1415.0	163 Days Interval 28.0 to 1317.0

Adverse Events

Guided Therapy

Serious events: 0 serious events

Other events: 6 other events

Deaths: 3 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Guided Therapy n=144 participants at risk A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles). Guided Therapy: A total of 200 neuroblastoma, brain tumor, and rare tumor patients will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
Blood and lymphatic system disorders Leukopenia	0.69% 1/144 • Number of events 1 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
Hepatobiliary disorders Alanine Transaminase increased	0.69% 1/144 • Number of events 1 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
Hepatobiliary disorders Aspartate Transferase	0.69% 1/144 • Number of events 1 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
Metabolism and nutrition disorders Dehydration	0.69% 1/144 • Number of events 1 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
Infections and infestations Infection	1.4% 2/144 • Number of events 2 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
Gastrointestinal disorders Oral Mucositis	0.69% 1/144 • Number of events 1 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
Hepatobiliary disorders Pancreatitis	0.69% 1/144 • Number of events 1 • Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.

Additional Information

Giselle Sholler, MD

Penn State Health Children's Hospital

Phone: 7175310003

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60