Trial Outcomes & Findings for High-Risk Neuroblastoma Chemotherapy Without G-CSF (NCT NCT02786719)

NCT ID: NCT02786719

Last Updated: 2020-03-12

Results Overview

Incidence of infections in chemotherapy cycles NOT followed by hematopoietic growth factors

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 13 participants
• Primary outcome timeframe: through study completion, approximately 5 months
• Results posted on: 2020-03-12

Participant Flow

Participant milestones

Measure	High Risk Neuroblastoma Patients This was a single arm study- all participants enrolled in single arm which provided 6 cycles of chemotherapy without prophylactic G-CSF for 4 cycles (1,2,4 and 6), and with G-CSF for stem cell collection after cycle 3, and GM-CSF administration after cycle 5 in preparation for surgical resection.
Overall Study STARTED	13
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

High-Risk Neuroblastoma Chemotherapy Without G-CSF

Baseline characteristics by cohort

Measure	Single Arm n=12 Participants Single arm study- all participants enrolled in single arm
Age, Categorical <=18 years	12 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	2.9 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 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	3 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants
International Neuroblastoma Risk Group Stage L2 (localized with image defined risk factors)	2 Participants n=5 Participants
International Neuroblastoma Risk Group Stage M (metastatic)	10 Participants n=5 Participants
MYCN gene amplification Yes	6 Participants n=5 Participants
MYCN gene amplification No	6 Participants n=5 Participants
Marrow involvement Yes	9 Participants n=5 Participants
Marrow involvement No	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: through study completion, approximately 5 months

Population: Includes all patients evaluable for endpoint

Incidence of infections in chemotherapy cycles NOT followed by hematopoietic growth factors

Outcome measures

Measure	High Risk Neuroblastoma Patients n=12 Participants Single arm study- all participants enrolled in single arm
Incidence of Infection	6 infections

SECONDARY outcome

Timeframe: through study completion, approximately 5 months

Population: 58 cycles of chemotherapy administered to 12 participants

incidence of delay in chemotherapy administration due to prolonged neutrophil recovery

Outcome measures

Measure	High Risk Neuroblastoma Patients n=58 Chemotherapy cycles Single arm study- all participants enrolled in single arm
Delay in Chemotherapy Administration Due to Prolonged Neutrophil Recovery	9 chemotherapy cycles

SECONDARY outcome

Timeframe: through study completion, approximately 5 months

Population: Completed all 6 cycles of induction chemotherapy on study

Response rate in the participants that completed all 6 cycles of induction chemotherapy on study. Response rate as categorize by International neuroblastoma response criteria.

• Complete response (CR): No evidence of primary tumor; no evidence of metastases (chest, abdomen, liver, bone, bone marrow, nodes, etc.), and urine catecholamines homovanillic acid (HVA)/ vanillylmandelic acid (VMA) normal. MIBG scan must be negative to qualify for CR.
• Very good partial response (VGPR): Greater than 90% reduction in primary tumor; no metastatic tumor (as above except bone); no new bone lesions, all pre-existing lesions improved, HVA/VMA normal
• Partial Response (PR): 50-90% reduction of primary tumor; 50% or greater reduction in measurable sites of metastases; 0-1 bone marrow samples with tumor; number of positive bone sites decreased by 50%

Outcome measures

Measure	High Risk Neuroblastoma Patients n=8 Participants Single arm study- all participants enrolled in single arm
the Response Rate Following Induction Chemotherapy Without Prophylactic Granulocyte Colony Stimulating Factor (G-CSF) Partial response	2 Participants
the Response Rate Following Induction Chemotherapy Without Prophylactic Granulocyte Colony Stimulating Factor (G-CSF) Very good partial response	2 Participants
the Response Rate Following Induction Chemotherapy Without Prophylactic Granulocyte Colony Stimulating Factor (G-CSF) Complete Response	4 Participants

Adverse Events

High Risk Neuroblastoma Patients

Serious events: 11 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	High Risk Neuroblastoma Patients n=12 participants at risk Single arm study- all participants enrolled in single arm
Blood and lymphatic system disorders Febrile Neutropenia	75.0% 9/12 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Infections and infestations bacteremia	33.3% 4/12 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Infections and infestations Skin/soft tissue infection	25.0% 3/12 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Infections and infestations Urinary tract infection	16.7% 2/12 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Respiratory, thoracic and mediastinal disorders Respiratory failure	8.3% 1/12 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Hepatobiliary disorders Sinusoidal obstruction syndrome	8.3% 1/12 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.

Other adverse events

Measure	High Risk Neuroblastoma Patients n=12 participants at risk Single arm study- all participants enrolled in single arm
Hepatobiliary disorders ALT increased	8.3% 1/12 • Number of events 1 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Hepatobiliary disorders Alk phos increased	8.3% 1/12 • Number of events 1 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Blood and lymphatic system disorders Anemia	100.0% 12/12 • Number of events 73 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Hepatobiliary disorders AST increased	8.3% 1/12 • Number of events 1 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Blood and lymphatic system disorders Neutropenia	100.0% 12/12 • Number of events 100 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Musculoskeletal and connective tissue disorders Pain	16.7% 2/12 • Number of events 2 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Blood and lymphatic system disorders Thrombocytopenia	100.0% 12/12 • Number of events 93 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Respiratory, thoracic and mediastinal disorders Pleural effusion	8.3% 1/12 • Number of events 1 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Skin and subcutaneous tissue disorders Rash	8.3% 1/12 • Number of events 1 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
General disorders Weight loss	8.3% 1/12 • Number of events 1 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.
Blood and lymphatic system disorders WBC decreased	100.0% 12/12 • Number of events 119 • Reporting period for each patient was from enrolling on trial until ANC recovery >750 from final cycle of chemotherapy administered while patient on study (max of 6 cycles). A systematic assessment was performed using regular laboratory assessment as well as regular investigator assessment of clinical records for AEs. All-Cause Mortality was assessed for all 13 participants who signed the informed consent. Serious and Other (Not Including Serious) Adverse Events were only assessed for the 12 participants who received treatment.

Additional Information

Sarah Whittle, MD MS

Baylor College of Medicine

Phone: 8328241471

Email: whittle@bcm.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place