Haploidentical Stem Cell Transplantation in Neuroblastoma

February 17, 2021 updated by: Jacek Toporski, Lund University Hospital

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

15

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Sweden
      • Lund, Sweden, 221 85
        • Lund University Hospital, Department of Pediatric Oncology and Bone Marrow Transplantation

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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: Rituximab
Drug: Fludarabine
Drug: Thiotepa
Drug: iodine I 131 metaiodobenzylguanidine
Procedure: T-cell depletion
Procedure: Haploidentical stem cell transplantation
Procedure: Donor Lymphocyte Infusion
Procedure: Co-transplantation of mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Engraftment rate
Time Frame: day 100
day 100

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Lund University Hospital

Investigators

  • Principal Investigator: Jacek Toporski, MD, PhD, Lund University Hospital, Department of Pediatric Oncology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2005

Primary Completion (Anticipated)

December 31, 2022

Study Completion (Anticipated)

December 31, 2022

Study Registration Dates

First Submitted

November 12, 2008

First Submitted That Met QC Criteria

November 12, 2008

First Posted (Estimate)

November 13, 2008

Study Record Updates

Last Update Posted (Actual)

February 21, 2021

Last Update Submitted That Met QC Criteria

February 17, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 385/2005

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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