News & Events
07/11/18
A New Clinical trial is entitled, A Phase 3 Trial of Antibody hu3F8 and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients with Primary or Secondary Refracto
Y-mAbs Therapeutics Clinical Trial 201: A Phase 3 Trial of Antibody Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients with Primary or Secondary Refractory Osteomedullary Disease


Children and adults diagnosed with high risk neuroblastoma and refractory osteomedullary disease will be treated for up to 93 weeks with Naxitamab (also called hu3F8), a humanized monoclonal antibody targeting GD2, and GM-CSF. Participants will be followed for up to five years after the first dose.
Each cycle is started with 5 days of GM-CSF in advance of the start of Naxitamab. GM-CSF is also administered at a higher dose on days 1 to 5. Naxitamab is given on days 1, 3, and 5 of each cycle.

The first 5 cycles are repeated every 4 weeks up to week 21 and after that, the frequency decreases to every 8 weeks through a total of 93 weeks. End of treatment will take place around 8 weeks after the last cycle and thereafter long-term follow-up will continue.
 
Inclusion/Exclusion Criteria
  • Diagnosis of neuroblastoma
  • High risk neuroblastoma with either primary or secondary refractory osteomedullary disease (persistent neuroblastoma in the bone and/or bone marrow after prior treatment)
  • Older than 1 year of age
  • Acceptable hematologic status, liver and renal function
  • No chemotherapy/immunotherapy within 3 weeks of the start of Naxitamab
  • Any neuroblastoma outside of the bone and/or bone marrow must be removed prior to trial entry
Locations (As of March 2018)
  • Riley Hospital for Children is the only U.S. site recruiting right now
  • Other U.S. sites that will be opening but are not yet recruiting: Children’s Hospital Los Angeles, University of Florida, Memorial Sloan Kettering Cancer Center, Nationwide Children’s Hospital, and M.D. Anderson Cancer Center
  • Sites in Europe: Denmark and Spain are recruiting, United Kingdom is not yet recruiting
 
Study Director: Steen Lisby, MD (Chief Medical Officer at Y-mAbs Therapeutics)
Principal Investigator at Riley Hospital for Children: Jacquelyn Carter, MD
Contact at Riley Hospital for Children: Angie Myers at almyers2@iu.edu
 
Purpose
  • Primary Objective
    • To evaluate the response rate during Naxitamab treatment
  • Secondary Objectives (Not an all inclusive list)
    • To evaluate the duration of response, progression free survival, and overall survival