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Haploidentical Stem Cell Transplantation in Neuroblastoma

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ClinicalTrials.gov Identifier: NCT00790413
Recruitment Status : Active, not recruiting
First Posted : November 13, 2008
Last Update Posted : March 14, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment Phase
Neuroblastoma Drug: iodine I 131 metaiodobenzylguanidine Drug: Fludarabine Drug: Thiotepa Procedure: T-cell depletion Procedure: Haploidentical stem cell transplantation Procedure: Donor Lymphocyte Infusion Drug: Rituximab Procedure: Co-transplantation of mesenchymal stem cells Early Phase 1

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma
Study Start Date : August 2005
Estimated Primary Completion Date : December 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: High-dose MIBG with haploidentical stem cell transplantation
High-dose MIBG followed by Fludarabine, Thiotepa and Melfalan as conditioning Before haploidentical transplantation of T-cell depleted graft
Drug: iodine I 131 metaiodobenzylguanidine Drug: Fludarabine Drug: Thiotepa Procedure: T-cell depletion Procedure: Haploidentical stem cell transplantation Procedure: Donor Lymphocyte Infusion Drug: Rituximab Procedure: Co-transplantation of mesenchymal stem cells


Outcome Measures

Primary Outcome Measures :
  1. Engraftment rate [ Time Frame: day 100 ]

Secondary Outcome Measures :
  1. Overall survival [ Time Frame: 1 year ]
  2. Immunological reconstitution [ Time Frame: day 100 ]
  3. Incidence of acute graft versus host disease [ Time Frame: day 100 ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00790413


Locations
Sweden
Lund University Hospital, Department of Pediatric Oncology and Bone Marrow Transplantation
Lund, Sweden, 221 85
Sponsors and Collaborators
Lund University Hospital
Investigators
Principal Investigator: Jacek Toporski, MD, PhD Lund University Hospital, Department of Pediatric Oncology
More Information

Publications:

Responsible Party: Jacek Toporski, MD, PhD, Head, Section of Pediatric Oncology/Hematology, Lund University Hospital
ClinicalTrials.gov Identifier: NCT00790413     History of Changes
Other Study ID Numbers: 385/2005
First Posted: November 13, 2008    Key Record Dates
Last Update Posted: March 14, 2017
Last Verified: March 2017

Keywords provided by Jacek Toporski, Lund University Hospital:
Radiotherapy
Immunotherapy
Hematopoietic Stem Cell Transplantation

Additional relevant MeSH terms:
Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Fludarabine
Fludarabine phosphate
Rituximab
Thiotepa
3-Iodobenzylguanidine
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Enzyme Inhibitors
Radiopharmaceuticals