This protocol will evaluate the addition of the anti-GD2 mAb, ch14.18 (dinutuximab) and sargramostim (GM-CSF) to standard Induction chemotherapy Cycles 3 to 5 in children and young adults with newly-diagnosed neuroblastoma.
Intensification of Consolidation therapy with tandem autologous stem cell transplants and the addition of post-Consolidation immunotherapy targeting the disialoganglioside GD2 present on neuroblastoma cells have led to improved event-free survival rates for patients with high-risk neuroblastoma. However, a substantial number of children still experience disease progression during Induction therapy, show evidence of persistent metastatic disease or relapse after completion of treatment.
Recent studies conducted in patients with recurrent or refractory neuroblastoma have demonstrated objective clinical responses following treatment with the combination of an anti-GD2 monoclonal antibody (mAb) plus chemotherapy and GM-CSF.
- To assess the feasibility and tolerability of administering ch14.18 (dinutuximab) and sargramostim (GM-CSF) in combination with a multi-agent chemotherapy regimen during Cycles 3-5 of the Induction phase for patients with newly-diagnosed high-risk neuroblastoma
Inclusion criteria include:
- Previously enrolled on ANBL00B1 (ANBLB1) or APEC14B1
- Diagnosis of neuroblastoma or ganglioneuroblastoma (nodular)
- 30 years old or younger
- No prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis within allowed timing)
Exclusion Criteria include:
- Older than 18 months with INRG stage L2, MYCN non-amplified, regardless of additional biologic features
- Between 12 and 18 months old with INRG stage M and all 3 favorable biologic features
- Has a bone marrow failure syndrome
- On immunosuppressive medications
St. Jude Children’s Research Hospital, Memphis, Tennessee
Collaborating sites in and outside the U.S.
About this study
Neuroblastoma is a type of cancer that occurs in nerve cells outside the brain. Neuroblastoma is considered high-risk disease when it has spread or is hard to treat.
This clinical trial will study a new treatment in patients with high-risk neuroblastoma. This treatment will add two drugs to standard combination chemotherapy. Those drugs are dinutuximab (ch14.18) and sargramostim (GM-CSF).
Dinutuximab has been approved by the U.S. Food and Drug Administration (FDA) to treat high-risk neuroblastoma at the end of standard therapy. However, it is not approved by the FDA to treat high-risk neuroblastoma at the beginning of therapy. In this study, dinutuximab will be considered experimental because it will be given at the beginning of therapy.
Sargramostim helps the body produce infection-fighting white blood cells. Research suggests sargramostim may increase the anti-cancer effect of monoclonal antibodies like dinutuximab. It is not considered experimental.
Purpose of this clinical trial
The main goal of this study is to learn the good and bad effects of dinutuximab when given with sargramostim and combination chemotherapy to patients with high-risk neuroblastoma.
- Diagnosis of neuroblastoma or ganglioneuroblastoma
- 30 years old or younger
ANBL17P1 Quick View Sponsor National Cancer Institute ClinicalTrials.gov identifier NCT03786783 Trial start date
45 Study type Interventional Study phase Phase II Conditions Neuroblastoma Ages Up to 30 years old Principal investigator Sara Federico, MD Study site St. Jude Children’s Research Hospital and collaborating sites in and outside the U.S. For a consultation or to discuss ANBL17P1 St. Jude Physician/Patient Referral Office
St. Jude Children’s Research Hospital
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Memphis, TN 38105 USA
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