This trial is active, not recruiting.

Condition neuroblastoma
Treatments iodine i 131 metaiodobenzylguanidine, fludarabine, thiotepa, t-cell depletion, haploidentical stem cell transplantation, donor lymphocyte infusion, rituximab, co-transplantation of mesenchymal stem cells
Phase phase 0
Target CD20
Sponsor Lund University Hospital
Start date August 2005
End date December 2016
Trial size 15 participants
Trial identifier NCT00790413, 385/2005


Children with primary resistant or relapsed neuroblastoma who do not achieve remission with conventional chemotherapy have extremely dismal prognosis. A novel treatment strategy combining tumor targeted radioisotope treatment with metaiodobenzylguanidine (MIBG) and immunotherapeutic effect of haploidentical stem cell transplantation (haploSCT) followed by low-dose donor lymphocyte infusions will be piloted. The use of the isotope is aimed to decrease pre-transplant tumour burden. Reduced intensity conditioning containing Fludarabine, Thiotepa and Melfalan will enable sustained engraftment as well as will serve as additional anti-tumor treatment. A prompt natural killer (NK)-cell mediated tumour control may be achieved by haploidentical stem cell transplantation. The investigators hypothesize that tumour cells potentially evading NK-cell mediated immunity may be targeted by infused donor T-cells and eliminated by either MHC-dependent manner or through a bystander effect. The possible graft versus tumor effect will be evaluated in children with therapy resistant neuroblastoma.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
High-dose MIBG followed by Fludarabine, Thiotepa and Melfalan as conditioning Before haploidentical transplantation of T-cell depleted graft
iodine i 131 metaiodobenzylguanidine
t-cell depletion
haploidentical stem cell transplantation
donor lymphocyte infusion
co-transplantation of mesenchymal stem cells

Primary Outcomes

Engraftment rate
time frame: day 100

Secondary Outcomes

Overall survival
time frame: 1 year
Immunological reconstitution
time frame: day 100
Incidence of acute graft versus host disease
time frame: day 100

Eligibility Criteria

Male or female participants from 6 months up to 21 years old.

Inclusion Criteria: - Refractory neuroblastoma (any chemo/radiosensitive stable disease) - Relapse incl. autologous HSCT 3 m earlier - Primary induction failure - Cardiac output SF ≥25% - Creatinine clearance ≥40 cc/min/1.73 m2 - Performance score of ≥50% (Lansky or Karnofsky) - Available haploidentical family donor, aged ≥18 yrs, HIV-neg Exclusion Criteria: - Rapidly progressive disease - Pregnancy

Additional Information

Official title High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma
Principal investigator Jacek Toporski, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in March 2016.
Information provided to ClinicalTrials.gov by Lund University Hospital.