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Haploidentical Stem Cell Transplantation in Neuroblastoma

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2010 by Lund University Hospital.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Lund University Hospital
ClinicalTrials.gov Identifier:
NCT00790413
First received: November 12, 2008
Last updated: November 17, 2010
Last verified: November 2010
  Purpose

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 Intervention 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
Phase 0

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Lund University Hospital:

Primary Outcome Measures:
  • Engraftment rate [ Time Frame: day 100 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Overall survival [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Immunological reconstitution [ Time Frame: day 100 ] [ Designated as safety issue: Yes ]
  • Incidence of acute graft versus host disease [ Time Frame: day 100 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 15
Study Start Date: August 2005
Estimated Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: High-dose MIBG with haploidentical stem cell transplantation 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

  Eligibility

Ages Eligible for Study:   6 Months to 21 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00790413

Contacts
Contact: Jacek Toporski, MD, PhD 004646178089 jacek.toporski@med.lu.se
Contact: Dominik Turkiewicz, MD, PhD 004646178064 dominik.turkiewicz@skane.se

Locations
Sweden
Lund University Hospital, Department of Pediatric Oncology and Bone Marrow Transplantation Recruiting
Lund, Sweden, 221 85
Contact: Jacek Toporski, MD PhD    004646178089    jacek.toporski@med.lu.se   
Principal Investigator: Jacek Toporski, MD, PhD         
Sub-Investigator: Albert N Bekassy, MD         
Sub-Investigator: Dominik Turkiewicz, MD, PhD         
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, Lund University Hospital, Department of Pediatric Oncology
ClinicalTrials.gov Identifier: NCT00790413     History of Changes
Other Study ID Numbers: 385/2005
Study First Received: November 12, 2008
Last Updated: November 17, 2010
Health Authority: Sweden: Regional Ethical Review Board

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

Additional relevant MeSH terms:
Neuroblastoma
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neuroectodermal Tumors, Primitive
Neuroectodermal Tumors, Primitive, Peripheral
3-Iodobenzylguanidine
Antineoplastic Agents
Diagnostic Uses of Chemicals
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Radiopharmaceuticals
Therapeutic Uses

ClinicalTrials.gov processed this record on November 25, 2014