For Healthcare Professionals

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)

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About the study

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.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

  1. Patients must be enrolled on ANBL00B1 (NCT00904241) or APEC14B1 (NCT02402244) prior to enrollment on ANBL1531 (NCT03126916)
  2. Patient must be >= 365 days and =< 30 years of age at diagnosis
  3. Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible:
  4. Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
  5. MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
  6. Age > 547 days regardless of biologic features
  7. Patients with INRG stage MS disease with MYCN amplification
  8. Patients with INRG stage L2 disease with MYCN amplification
  9. Patients > 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progressed to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M
  10. Patients >= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M
  11. Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing); patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible; patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible
  12. Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows:
  13. 1 to < 2 years: male = 0.6; female = 0.6
  14. 2 to < 6 years: male = 0.8; female = 0.8
  15. 6 to < 10 years: male = 1; female = 1
  16. 10 to < 13 years: male = 1.2; female = 1.2
  17. 13 to < 16 years: male = 1.5; female = 1.4
  18. >= 16 years: male = 1.7; female = 1.4
  19. Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
  20. Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN; for the purposes of this study, ULN for SGPT (ALT) is 45
  21. Shortening fraction of >= 27% by echocardiogram, or ejection fraction of > 50% by echocardiogram or radionuclide angiogram
  22. No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure

EXCLUSION CRITERIA

  1. 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)
  2. Patients with bone marrow failure syndromes
  3. Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
  4. 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
  5. Lactating females who plan to breastfeed their infants
  6. Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.

Study’s details


Contition

Childhood Ganglioneuroblastoma,Childhood Neuroblastoma,Ganglioneuroblastoma,INRG Stage L2,INRG Stage M,INRG Stage MS,NMYC Gene Amplification,Neuroblastoma,Recurrent Neuroblastoma

Age

365+

Phase

Phase 3

Participants needed

724

Est. Completion Date

Sep 2026

Treatment type

Interventional


Sponsor

Children's Oncology Group

ClinicalTrials.gov identifier

NCT03126916

Study number

ANBL1531

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