N9: Pilot Study of Novel Shortened Induction Chemotherapy for High-Risk Neuroblastoma
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Enrollment
- 26
- Locations
- 1
- Primary Endpoint
- Assess safety of the N9 regimen in participants with HR-NB through toxicity assessment
Study Overview
Brief Summary
The purpose of the study is to find out whether N9 is a safe and effective treatment for children with neuroblastoma. N9 includes 3 different combinations of chemotherapy drugs that are given at different times - Cyclophosphamide, topotecan, and vincristine (CTV), Ifosfamide, carboplatin, and etoposide (ICE), Cyclophosphamide, doxorubicin, and vincristine (CDV).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 1 Year to 19 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Diagnosis of NB as defined by histopathology (confirmed by the MSK Department of Pathology), BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.
- •HR-NB, defined as MYCN-amplified stage 2/3/4/4S at any age and stage 4 in patients >18 months old.
- •No more than one prior cycle of chemotherapy.
- •Age <19 years old.
- •Signed informed consent indicating awareness of the investigational nature of this treatment.
Exclusion Criteria
- •Severe dysfunction of major organs, i.e., renal, cardiac, hepatic, neurologic, pulmonary, hematologic, or gastrointestinal toxicity ≥ grade
- •Inability to comply with protocol requirements.
- •Pregnancy is not an issue because all patients will be pre-adolescents.
Arms & Interventions
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Cyclophosphamide (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Topotecan (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Doxorubicin (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Vincristine (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Ifosfamide (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Etoposide (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Carboplatin (Drug)
Participants with newly-diagnosed HR-Neuroblastoma
This pilot study of N9 as induction chemotherapy will enroll 30 patients with newly-diagnosed HR-NB. A first cohort of >1 to 12-year old, and a second cohort of extended age <19 years old. Both cohorts will be analyzed together.
Intervention: Mesna (Drug)
Outcomes
Primary Outcomes
Assess safety of the N9 regimen in participants with HR-NB through toxicity assessment
Time Frame: 16 weeks
Adverse events will be graded using Common Toxicity Criteria Version 5.0 developed by the National Cancer Institute of the USA, events of all grades will be tabulated, for non-hematologic effects and hematologic effects on the patients in the safety set. The timing of cycles will also be described to assess any delay due to toxicity
Secondary Outcomes
No secondary outcomes reported