Trial Outcomes & Findings for Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer (NCT NCT01802567)

Last Updated: 2024-08-06

Results Overview

Days to treatment is one data point that will be used in order to determine feasibility. The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and 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

52 participants

Primary outcome timeframe

Date from biopsy to completion of 1 cycle of therapy, generally about 30 days

Results posted on

2024-08-06

Participant Flow

Participant milestones

Participant milestones
Measure	Overall Study A total of up to 48 evaluable subjects (up to 52 enrolled) with childhood cancer that are refractory to or have relapsed on conventional therapy will be treated with molecular guided therapy. Subjects will be evaluated in 3 strata: * Stratum 1: 16 subjects with refractory/relapsed neuroblastoma * Stratum 2: 16 subjects with relapsed or refractory brain tumors Stratum 3: 16 subjects with refractory or relapsed rare tumors
Overall Study STARTED	52
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	18

Reasons for withdrawal

Reasons for withdrawal
Measure	Overall Study A total of up to 48 evaluable subjects (up to 52 enrolled) with childhood cancer that are refractory to or have relapsed on conventional therapy will be treated with molecular guided therapy. Subjects will be evaluated in 3 strata: * Stratum 1: 16 subjects with refractory/relapsed neuroblastoma * Stratum 2: 16 subjects with relapsed or refractory brain tumors Stratum 3: 16 subjects with refractory or relapsed rare tumors
Overall Study Withdrawal by Subject	2
Overall Study Physician Decision	1
Overall Study Lack of Efficacy	7
Overall Study Consented but did not start therapy	7
Overall Study Protocol Violation	1

Baseline Characteristics

Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Guided Therapy- Pediatric Gene Analysis Platform n=52 Participants A total of 48 pediatric cancer (neuroblastoma, brain tumor, and rare tumor) 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- Pediatric Gene Analysis Platform: A total of 48 neuroblastoma, brain tumor, and rare tumor 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).
Age, Categorical <=18 years	49 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	9.7 years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	39 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	37 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	6 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	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	40 Participants n=5 Participants
Race (NIH/OMB) More than one race	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Tumor Type refractory/relapsed neuroblastoma	22 Participants n=5 Participants
Tumor Type relapsed or refractory brain tumors	8 Participants n=5 Participants
Tumor Type refractory or relapsed rare tumors	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: Date from biopsy to completion of 1 cycle of therapy, generally about 30 days

Outcome measures

Outcome measures
Measure	Stratum 1 n=19 Participants Subjects with refractory/relapsed neuroblastoma	Stratum 2 n=7 Participants Subjects with relapsed or refractory brain tumors	Stratum 3 n=18 Participants Subjects with refractory or relapsed rare tumors
Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment	12.2 Days Interval 7.0 to 21.0	19.9 Days Interval 14.0 to 30.0	14.9 Days Interval 9.0 to 29.0

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 3 years.

Population: All subjects that started at least one dose of therapy. Outcome measure was reported as an overall study result, and was not reported by cohort.

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

Outcome measures

Outcome measures
Measure	Stratum 1 n=45 Participants Subjects with refractory/relapsed neuroblastoma	Stratum 2 Subjects with relapsed or refractory brain tumors	Stratum 3 Subjects with refractory or relapsed rare tumors
Number of Participants With Adverse Events as a Measure of Safety	43 Participants	—	—

SECONDARY outcome

Timeframe: Followed until off therapy, generally 3 years

Population: Analyzed population only includes evaluable subjects, defined as subjects that made it to an evaluation time point (scans at end of cycle 2), or had a documented progression of disease prior to evaluation time point.

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	Stratum 1 n=17 Participants Subjects with refractory/relapsed neuroblastoma	Stratum 2 n=7 Participants Subjects with relapsed or refractory brain tumors	Stratum 3 n=17 Participants Subjects with refractory or relapsed 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. Partial Response	0 Participants	0 Participants	1 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	1 Participants	2 Participants	2 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. Stable Disease	4 Participants	0 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. Progressive Disease	12 Participants	5 Participants	8 Participants

Adverse Events

Guided Therapy- Pediatric Gene Analysis Platform

Serious events: 21 serious events

Other events: 43 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Guided Therapy- Pediatric Gene Analysis Platform n=45 participants at risk A total of 48 pediatric cancer (neuroblastoma, brain tumor, and rare tumor) 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). Adverse events were reported as a whole for all subjects that started study therapy and were not reported by individual cohort.
Renal and urinary disorders Cystitis noninfective	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Blood and lymphatic system disorders Thrombocytopenia	4.4% 2/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Deydration	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Vascular disorders Epistaxis	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Infections and infestations Febrile neutropenia	11.1% 5/45 • Number of events 5 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
General disorders Fever	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Renal and urinary disorders Hematuria	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
General disorders Hypertension	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Hypocalcemia	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Infections and infestations Infection, lung	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Cardiac disorders Left ventricular systolic dysfunction	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Blood and lymphatic system disorders Neutropenia	6.7% 3/45 • Number of events 3 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Gastrointestinal disorders Oral mucocitis	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Gastrointestinal disorders Vomiting	2.2% 1/45 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.

Other adverse events

Other adverse events
Measure	Guided Therapy- Pediatric Gene Analysis Platform n=45 participants at risk A total of 48 pediatric cancer (neuroblastoma, brain tumor, and rare tumor) 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). Adverse events were reported as a whole for all subjects that started study therapy and were not reported by individual cohort.
Blood and lymphatic system disorders Neutropenia	46.7% 21/45 • Number of events 21 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Blood and lymphatic system disorders Anemia	35.6% 16/45 • Number of events 16 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Anorexia	17.8% 8/45 • Number of events 8 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Hepatobiliary disorders Aspartate Transferase Increase	6.7% 3/45 • Number of events 3 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Gastrointestinal disorders Constipation	11.1% 5/45 • Number of events 5 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Gastrointestinal disorders Diarrhea	13.3% 6/45 • Number of events 6 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Blood and lymphatic system disorders Leukopenia	42.2% 19/45 • Number of events 19 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Vascular disorders Epistaxis	8.9% 4/45 • Number of events 4 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
General disorders Fatigue	8.9% 4/45 • Number of events 4 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
General disorders Fever	8.9% 4/45 • Number of events 4 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
General disorders Hypertension	6.7% 3/45 • Number of events 3 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Hypocalcemia	15.6% 7/45 • Number of events 7 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Hypokalemia	8.9% 4/45 • Number of events 4 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Hyponatremia	8.9% 4/45 • Number of events 4 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Metabolism and nutrition disorders Hypophosphatemia	6.7% 3/45 • Number of events 3 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Infections and infestations Infection	15.6% 7/45 • Number of events 7 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Skin and subcutaneous tissue disorders Rash	8.9% 4/45 • Number of events 4 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Gastrointestinal disorders Nausea	22.2% 10/45 • Number of events 10 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
General disorders Pain	11.1% 5/45 • Number of events 5 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Gastrointestinal disorders Vomiting	15.6% 7/45 • Number of events 7 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.
Blood and lymphatic system disorders Thrombocytopenia	35.6% 16/45 • Number of events 16 • 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 3 years. Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.

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