Haploidentical Stem Cell Transplantation in Neuroblastoma

High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma

Status

UNKNOWN

Phases

Early Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00790413

Enrollment

Registered

2008-11-13

Start date

2005-08-31

Completion date

2022-12-31

Last updated

2021-02-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuroblastoma

Keywords

Radiotherapy, Immunotherapy, Hematopoietic Stem Cell Transplantation

Brief 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.

Interventions

DRUGiodine I 131 metaiodobenzylguanidine

DRUGFludarabine

DRUGThiotepa

PROCEDURET-cell depletion

PROCEDUREHaploidentical stem cell transplantation

PROCEDUREDonor Lymphocyte Infusion

DRUGRituximab

PROCEDURECo-transplantation of mesenchymal stem cells

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Months to 21 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Engraftment rate	day 100

Secondary

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

Countries

Sweden

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026