Immunotherapy + Chemotherapy for High-Risk Neuroblastoma

Recruiting in Palo Alto (17 mi)

+9 other locations

Overseen bySara M Federico

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: National Cancer Institute (NCI)

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This phase II pilot trial studies the side effects and how well dinutuximab and sargramostim work when combined with chemotherapy in patients with high-risk neuroblastoma. Immunotherapy with monoclonal antibodies, such as dinutuximab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Sargramostim helps the body produce normal infection-fighting white blood cells. These cells also help the dinutuximab work better. Giving chemotherapy before a stem cell transplant, with drugs such as cisplatin, etoposide, vincristine, doxorubicin, cyclophosphamide, thiotepa, melphalan, etoposide, carboplatin, topotecan, and isotretinoin, helps kill cancer cells that are in the body and helps make room in a patient's bone marrow for new blood-forming cells (stem cells). Giving dinutuximab and sargramostim with combination chemotherapy may work better than combination chemotherapy alone in treating patients with high-risk neuroblastoma.

Eligibility Criteria

This trial is for children and young adults with high-risk neuroblastoma or ganglioneuroblastoma, confirmed by pathology analysis or bone marrow clumps with elevated urinary catecholamines. Participants must have certain heart function levels, adequate liver function, and kidney clearance rates. They should not have had extensive prior treatments except under specific circumstances and must be able to undergo stem cell collection. Pregnant females, patients over 18 months with non-amplified MYCN regardless of other features, those on immunosuppressants (except for allergies/adrenal therapy), breastfeeding females, and sexually active individuals not using contraception are excluded.

Inclusion Criteria

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:

Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features: MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features; OR Age > 547 days regardless of biologic features;

Creatinine clearance (CrCl) or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows: Age 1 month to < 6 months (male 0.4 mg/dL, female 0.4 mg/dL); Age 6 months to < 1 year (male 0.5 mg/dL, female 0.5 mg/dL); Age 1 to < 2 years (male 0.6 mg/dL, female 0.6 mg/dL); Age 2 to < 6 years (male 0.8 mg/dL, female 0.8 mg/dL); Age 6 to < 10 years (male 1 mg/dL, female 1 mg/dL); Age 10 to < 13 years (male 1.2 mg/dL, female 1.2 mg/dL); Age 13 to < 16 years (male 1.5 mg/dL, female 1.4 mg/dL); Age >= 16 years (male 1.7 mg/dL, female 1.4 mg/dL) (within 7 days prior to enrollment).

+28 more

Exclusion Criteria

Lactating females who plan to breastfeed their infants.

Female patients who are pregnant are ineligible 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.

Patients >18 months of age with INRG stage L2, MYCN non-amplified, regardless of additional biologic features.

+4 more

Participant Groups

The study tests the effectiveness of dinutuximab combined with sargramostim alongside a chemotherapy regimen before a stem cell transplant in treating high-risk neuroblastoma. The chemotherapy includes cisplatin, etoposide, vincristine, doxorubicin among others. This approach aims to improve immune response against cancer cells while making space in the bone marrow for new blood-forming cells.

1Treatment groups

Experimental Treatment

Group I: Treatment(chemotherapy, dinutuximab, sargramostim, ASCT, EBRT)Experimental Treatment15 Interventions

See Detailed Description

Carboplatin is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Paraplatin for: