Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age365-30
SponsorChildren's Oncology Group
About this trial
This phase III trial studies iobenguane I-131 or lorlatinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Lorlatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or lorlatinib and standard therapy may work better compared to lorlatinib and standard therapy alone in treating younger patients with neuroblastoma or ganglioneuroblastoma.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients must be enrolled on ANBL00B1 (NCT00904241) or APEC14B1 (NCT02402244) prior to enrollment on ANBL1531 (NCT03126916)
FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patient must be >= 365 days and =< 30 years of age at diagnosis
MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
Age > 547 days regardless of biologic features
Disqualifiers
FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients with INRG stage L2 tumors without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients with bone marrow failure syndromes
FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential
Trial design
Design model
Parallel
Treatments tested in this trial
Autologous Hematopoietic Stem Cell Transplantation
Procedure/SurgeryUndergo autologous HSCT
Biospecimen Collection
Procedure/SurgeryUndergo blood sample collection
Bone Marrow Aspiration and Biopsy
Procedure/SurgeryUndergo bone marrow aspiration and biopsy
Busulfan
DrugGiven IV
Carboplatin
DrugGiven IV
Cisplatin
DrugGiven IV
Computed Tomography
Procedure/SurgeryUndergo CT scan
Cyclophosphamide
DrugGiven IV
Dexrazoxane Hydrochloride
DrugGiven IV
Dinutuximab
Biological/VaccineGiven IV
Doxorubicin Hydrochloride
DrugGiven IV
Echocardiography Test
Procedure/SurgeryUndergo echocardiography
Etoposide Phosphate
DrugGiven IV
External Beam Radiation Therapy
RadiationUndergo EBRT
Iobenguane I-123
RadiationGiven 123 I-MIBG
Iobenguane I-131
RadiationGiven IV
Isotretinoin
DrugGiven PO
Lorlatinib
DrugGiven PO
Magnetic Resonance Imaging
Procedure/SurgeryUndergo MRI
Melphalan Hydrochloride
DrugGiven IV
Multigated Acquisition Scan
Procedure/SurgeryUndergo MUGA scan
Positron Emission Tomography
Procedure/SurgeryUndergo PET scan
Sargramostim
Biological/VaccineGiven SC
Therapeutic Conventional Surgery
Procedure/SurgeryUndergo standard of care surgery
Thiotepa
DrugGiven IV
Topotecan Hydrochloride
DrugGiven IV
Vincristine Sulfate
DrugGiven IV
Treatment groups
750 Participants
are divided into 5 treatment groupsGroup A: Arm A (chemotherapy, HSCT, EBRT)Experimental treatment 22 interventions
Group B: Arm B (Iobenguane I-131, chemotherapy, HSCT, EBRT)Experimental treatment 24 interventions
Group C: Arm C (Iobenguane I-131, chemotherapy, BuMel, HSCT, EBRT)Experimental treatment 24 interventions
Group D: Arm D (chemotherapy, HSCT, EBRT)Experimental treatment 22 interventions
Group E: Arm E (lorlatinib, chemotherapy, HSCT, EBRT)Experimental treatment 22 interventions
Trial outcomes
Primary outcomes
1
Event free survival (EFS) (Arm A, B, D, and E)
EFS time is calculated from date of randomization or assignment to first episode of disease relapse or progression, second malignancy, or death, or until last contact if no event has occurred.
Time frame
3 yearsSecondary outcomes
1
Incidence of adverse events
The proportion of patients with at least one Grade 3 or higher toxicity during protocol therapy, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0, will be reported.
Time frame
Up to 18 months for Arms A-D and 28 months for Arm E2
EFS (Arm C)
EFS time is calculated from date of randomization to first episode of disease relapse or progression, second malignancy, or death, or until last contact if no event has occurred.
Time frame
3 years3
Overall survival (OS)
OS time is calculated from date of randomization or assignment until death, or until last contact if patient is alive.
Time frame
3 years4
Response rate
The response rate will be calculated among all evaluable patients at end-Induction. Responders are defined as patients who achieve a \>= partial response (PR) per the revised International Neuroblastoma Response Criteria (INRC).
Time frame
Up to 6 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Children's Oncology Group
Lead sponsor
National Cancer Institute (NCI)
Collaborator
Source ClinicalTrials.gov