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NeuroblastomaEarly Phase 1NCT00790413

Haploidentical Stem Cell Transplantation in Neuroblastoma

A Early Phase 1 study testing iodine I 131 metaiodobenzylguanidine, Fludarabine and Thiotepa in people with Neuroblastoma, run by Lund University Hospital at 1 site in Sweden.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 6 months to 21 years
Conditions the study is for
Neuroblastoma
What is being tested?
iodine I 131 metaiodobenzylguanidine, Fludarabine and Thiotepa
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteSweden
Study dates
Aug 2005to 31 Dec 2022
Phase
Early Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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

Who cannot join

  • Rapidly progressive disease
  • Pregnancy

Trial locations and contacts

1 site in 1 country.

Sweden1 site

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

    Lund, 221 85

Other trials in Sweden

About this study

Official title: 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.

Conditions
Neuroblastoma
Being tested
iodine I 131 metaiodobenzylguanidine, Fludarabine, Thiotepa, T-cell depletion, Haploidentical stem cell transplantation
Keywords
Radiotherapy, Immunotherapy, Hematopoietic Stem Cell Transplantation
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • iodine I 131 metaiodobenzylguanidineDrug

  • FludarabineDrug

  • ThiotepaDrug

  • T-cell depletionProcedure or surgery

  • Haploidentical stem cell transplantationProcedure or surgery

  • Donor Lymphocyte InfusionProcedure or surgery

  • RituximabDrug

  • Co-transplantation of mesenchymal stem cellsProcedure or surgery

Study groups in NCT00790413
Study groupRoleWhat they receive
High-dose MIBG with haploidentical stem cell transplantationExperimentalHigh-dose MIBG followed by Fludarabine, Thiotepa and Melfalan as conditioning Before haploidentical transplantation of T-cell depleted graft
Receives: 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

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Engraftment rate

    Measured: day 100

Secondary outcomes (3)

  1. Overall survival

    Measured: 1 year

  2. Immunological reconstitution

    Measured: day 100

  3. Incidence of acute graft versus host disease

    Measured: day 100

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Nov 2008 (estimated)
  2. Study startAug 2005
  3. Primary completion31 Dec 2022 (estimated)
  4. Study completion31 Dec 2022 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00790413
Sponsor's protocol ID
385/2005
Status last verified
Feb 2021
First submitted
12 Nov 2008
Last updated
21 Feb 2021
Results posted
No

Publications

  • Inoue M, Nakano T, Yoneda A, Nishikawa M, Nakayama M, Yumura-Yagi K, Sakata N, Yasui M, Okamura T, Kawa K. Graft-versus-tumor effect in a patient with advanced neuroblastoma who received HLA haplo-identical bone marrow transplantation. Bone Marrow Transplant. 2003 Jul;32(1):103-6. doi: 10.1038/sj.bmt.1704070.

    PubMed 12815485

  • Matthay KK, Tan JC, Villablanca JG, Yanik GA, Veatch J, Franc B, Twomey E, Horn B, Reynolds CP, Groshen S, Seeger RC, Maris JM. Phase I dose escalation of iodine-131-metaiodobenzylguanidine with myeloablative chemotherapy and autologous stem-cell transplantation in refractory neuroblastoma: a new approaches to Neuroblastoma Therapy Consortium Study. J Clin Oncol. 2006 Jan 20;24(3):500-6. doi: 10.1200/JCO.2005.03.6400.

    PubMed 16421427

  • Prigione I, Corrias MV, Airoldi I, Raffaghello L, Morandi F, Bocca P, Cocco C, Ferrone S, Pistoia V. Immunogenicity of human neuroblastoma. Ann N Y Acad Sci. 2004 Dec;1028:69-80. doi: 10.1196/annals.1322.008.

    PubMed 15650233

  • Neal ZC, Imboden M, Rakhmilevich AL, Kim KM, Hank JA, Surfus J, Dixon JR, Lode HN, Reisfeld RA, Gillies SD, Sondel PM. NXS2 murine neuroblastomas express increased levels of MHC class I antigens upon recurrence following NK-dependent immunotherapy. Cancer Immunol Immunother. 2004 Jan;53(1):41-52. doi: 10.1007/s00262-003-0435-2. Epub 2003 Sep 18.

    PubMed 14504825

  • Raffaghello L, Prigione I, Bocca P, Morandi F, Camoriano M, Gambini C, Wang X, Ferrone S, Pistoia V. Multiple defects of the antigen-processing machinery components in human neuroblastoma: immunotherapeutic implications. Oncogene. 2005 Jul 7;24(29):4634-44. doi: 10.1038/sj.onc.1208594.

    PubMed 15897905

  • Lang P, Pfeiffer M, Muller I, Schumm M, Ebinger M, Koscielniak E, Feuchtinger T, Foll J, Martin D, Handgretinger R. Haploidentical stem cell transplantation in patients with pediatric solid tumors: preliminary results of a pilot study and analysis of graft versus tumor effects. Klin Padiatr. 2006 Nov-Dec;218(6):321-6. doi: 10.1055/s-2006-942256.

    PubMed 17080334

  • Toporski J, Garkavij M, Tennvall J, Ora I, Gleisner KS, Dykes JH, Lenhoff S, Juliusson G, Scheding S, Turkiewicz D, Bekassy AN. High-dose iodine-131-metaiodobenzylguanidine with haploidentical stem cell transplantation and posttransplant immunotherapy in children with relapsed/refractory neuroblastoma. Biol Blood Marrow Transplant. 2009 Sep;15(9):1077-85. doi: 10.1016/j.bbmt.2009.05.007. Epub 2009 Jul 8.

    PubMed 19660720

  • Illhardt T, Toporski J, Feuchtinger T, Turkiewicz D, Teltschik HM, Ebinger M, Schwarze CP, Holzer U, Lode HN, Albert MH, Gruhn B, Urban C, Dykes JH, Teuffel O, Schumm M, Handgretinger R, Lang P. Haploidentical Stem Cell Transplantation for Refractory/Relapsed Neuroblastoma. Biol Blood Marrow Transplant. 2018 May;24(5):1005-1012. doi: 10.1016/j.bbmt.2017.12.805. Epub 2018 Jan 4.

    PubMed 29307718

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing iodine I 131 metaiodobenzylguanidine, Fludarabine and Thiotepa in people with Neuroblastoma, run by Lund University Hospital at 1 site in Sweden.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 21 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Engraftment rate (time frame: day 100). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00790413, which also lists 8 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2021.

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