Naxitamab for High-risk Neuroblastoma

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Levine Children's Hospital, Charlotte, NC
High-risk Neuroblastoma+1 More
Naxitamab - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy. The initial chemotherapy will include 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles. Subjects with an ALK mutation or amplification will have ceritinib added to their treatment regimen as soon as results are available. We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.

Eligible Conditions

  • High-risk Neuroblastoma

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 6 Secondary · Reporting Duration: Year 7

Month 12
Number of participants with a Very Good Partial Response (VGPR)(+) rate (VGPR + Complete Response (CR) rate)
Objective Response Rate (ORR)
Month 4
Objective Response Rate (ORR) after two cycles
Year 1
Morphine equivalent daily dose (MEDD) per subject
Year 2
Number of participants with treatment-related adverse events
Objective Response Rate (ORR) after five cycles
Year 7
Number of days that subjects remain alive
Number of days that subjects remain in remission

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

2 Treatment Groups

Subjects with ALK Wildtype or Unknown
1 of 2
Subjects with ALK aberration
1 of 2
Experimental Treatment

76 Total Participants · 2 Treatment Groups

Primary Treatment: Naxitamab · No Placebo Group · Phase 2

Subjects with ALK Wildtype or Unknown
Drug
Experimental Group · 1 Intervention: Naxitamab · Intervention Types: Drug
Subjects with ALK aberrationExperimental Group · 2 Interventions: Naxitamab, Ceritinib · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ceritinib
2015
Completed Phase 2
~310

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: year 7
Closest Location: Levine Children's Hospital · Charlotte, NC
Photo of charlotte 1Photo of charlotte 2Photo of charlotte 3
2011First Recorded Clinical Trial
1 TrialsResearching High-risk Neuroblastoma
29 CompletedClinical Trials

Who is running the clinical trial?

Wake Forest University Health SciencesLead Sponsor
1,100 Previous Clinical Trials
1,128,949 Total Patients Enrolled
Jaqueline Kraveka, DOStudy ChairMedical University of South Carolina

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Subjects with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) Stage 4 are eligible with the following: i
Subjects with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the following: i
You are 12 months of age or older.
Subjects with newly diagnosed neuroblastoma with INSS Stage 2A/2B with MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features.
You are under 21 years of age at initial diagnosis.
Ability to tolerate Peripheral blood stem cell (PBSC) collection: No known contraindication to PBSC collection.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.