Trial Outcomes & Findings for Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma (NCT NCT01355679)

Last Updated: 2024-08-06

Results Overview

Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."

Recruitment status

COMPLETED

Study phase

Target enrollment

16 participants

Primary outcome timeframe

1 year

Results posted on

2024-08-06

Participant Flow

This was an open label, multi-center prospective feasibility study in patients with refractory or recurrent neuroblastoma that enrolled at NMTRC centers across the country between 8/11/2011 and 11/26/2012.

Participant milestones

Participant milestones
Measure	Guided Therapy A total of 14 neuroblastoma patients who are refractory or relapsed on conventional therapy 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 14 neuroblastoma patients who are refractory or relapsed on conventional therapy 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 disease response, progression and safety. All patients will be
Overall Study STARTED	16
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Guided Therapy A total of 14 neuroblastoma patients who are refractory or relapsed on conventional therapy 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 14 neuroblastoma patients who are refractory or relapsed on conventional therapy 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 disease response, progression and safety. All patients will be
Overall Study benign tumor types found at biopsy	2

Baseline Characteristics

Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Guided Therapy n=16 Participants All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.
Age, Categorical <=18 years	15 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	9 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	1 Participants n=5 Participants
Race (NIH/OMB) White	11 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: All subjects had soft tissue disease in which biopsy was possible. Two subjects were deemed ineligible due to benign tumor type after biopsy.

Outcome measures

Outcome measures
Measure	Guided Therapy n=14 Participants All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.
Percentage of Participants That Are Able to Meet Feasibility Parameters.	100 percentage of participants

SECONDARY outcome

Timeframe: 1 year

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

Outcome measures

Outcome measures
Measure	Guided Therapy n=14 Participants All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.
Number of Participants With Adverse Events as a Measure of Safety	12 participants

SECONDARY outcome

Timeframe: 1 year

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Outcome measures

Outcome measures
Measure	Guided Therapy n=14 Participants All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.
Overall Response Rate (ORR) of Participants Using RECIST Criteria	7 percentage of participants with PR or CR

SECONDARY outcome

Timeframe: 1 year

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Outcome measures

Outcome measures
Measure	Guided Therapy n=14 Participants All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.
Activity of Treatments Chosen Based on Progression Free Survival (PFS)	59 Days Interval 43.0 to 59.0

Adverse Events

Guided Therapy

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Guided Therapy n=14 participants at risk A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy 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).
Gastrointestinal disorders Abdominal Pain	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Hepatobiliary disorders ALT elevation	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Hepatobiliary disorders AST Elevation	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Blood and lymphatic system disorders Anemia	42.9% 6/14 • Number of events 6 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Hepatobiliary disorders Elevated Bilirubin	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Gastrointestinal disorders Constipation	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Gastrointestinal disorders Dehydration	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
General disorders Fatigue	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Infections and infestations Fever	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Blood and lymphatic system disorders Hypoalbunemia	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
General disorders Hypocalcemia	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
General disorders Hypophosphatemia	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Infections and infestations Infection	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Blood and lymphatic system disorders Leukopenia	50.0% 7/14 • Number of events 7 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Blood and lymphatic system disorders Lymphocytopenia	21.4% 3/14 • Number of events 3 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Gastrointestinal disorders Mucositis	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
General disorders Myalgia	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Gastrointestinal disorders Nausea	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Blood and lymphatic system disorders Neutropenia	50.0% 7/14 • Number of events 7 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
General disorders Pain	14.3% 2/14 • Number of events 2 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Skin and subcutaneous tissue disorders Rash	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Cardiac disorders Tachycardia	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Blood and lymphatic system disorders Thrombocytopenia	57.1% 8/14 • Number of events 8 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
Gastrointestinal disorders Vomiting	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.
General disorders Weight Loss	7.1% 1/14 • Number of events 1 • Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.

Additional Information

Giselle Sholler, MD

NMTRC at Spectrum Health

Phone: 616-267-0334

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place