Haploidentical Stem Cell Transplantation in Neuroblastoma

• Status: Active, not recruiting
• Trial end date: 2022-12-31
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Lund University Hospital
• Treatments: 3-Iodobenzylguanidine; Fludarabine; Fludarabine phosphate; Rituximab; Thiotepa

Criteria

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