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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 : February 21, 2021
Sponsor:
Information provided by (Responsible Party):
Jacek Toporski, Lund University Hospital

Tracking Information
First Submitted Date  ICMJE November 12, 2008
First Posted Date  ICMJE November 13, 2008
Last Update Posted Date February 21, 2021
Study Start Date  ICMJE August 2005
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 12, 2008)
Engraftment rate [ Time Frame: day 100 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 12, 2008)
  • Overall survival [ Time Frame: 1 year ]
  • Immunological reconstitution [ Time Frame: day 100 ]
  • Incidence of acute graft versus host disease [ Time Frame: day 100 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Haploidentical Stem Cell Transplantation in Neuroblastoma
Official Title  ICMJE High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Early Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Neuroblastoma
Intervention  ICMJE
  • 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
Study Arms  ICMJE 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
Interventions:
  • 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
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: November 12, 2008)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2022
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Sweden
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00790413
Other Study ID Numbers  ICMJE 385/2005
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Jacek Toporski, Lund University Hospital
Study Sponsor  ICMJE Lund University Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Jacek Toporski, MD, PhD Lund University Hospital, Department of Pediatric Oncology
PRS Account Lund University Hospital
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP