Donor Stem Cell Transplant in Treating Young Patients With Relapsed or Refractory Solid Tumors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00112645
Recruitment Status : Completed
First Posted : June 3, 2005
Last Update Posted : July 18, 2013
Information provided by:
National Cancer Institute (NCI)

June 2, 2005
June 3, 2005
July 18, 2013
April 2005
April 2010   (Final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00112645 on Archive Site
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Donor Stem Cell Transplant in Treating Young Patients With Relapsed or Refractory Solid Tumors
A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease

RATIONALE: A peripheral stem cell, bone marrow, or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving busulfan and melphalan with or without antithymocyte globulin before transplant and cyclosporine with methylprednisolone or methotrexate after transplant may stop this from happening.

PURPOSE: This phase I trial is studying the side effects of donor stem cell transplant in treating young patients with relapsed or refractory solid tumors.


  • Determine the toxicity of allogeneic hematopoietic stem cell transplantation, in terms of the incidence of grade 3-4 acute graft-versus-host disease, in young patients with relapsed or refractory solid tumors.
  • Determine the incidence of transplant-related mortality at 100 days post-transplantation in these patients.


  • Conditioning: Patients receive busulfan IV or orally 4 times daily on days -8 to -5 (a total of 16 doses) and melphalan IV over 15-20 minutes on days -4 to -2. Patients with an unrelated donor also receive anti-thymocyte globulin IV on days -4 to -2.
  • Allogeneic hematopoietic stem cell transplantation (SCT): Patients undergo allogeneic hematopoietic SCT on day 0.
  • Post-transplant graft-versus-host disease (GVHD) prophylaxis: Patients who undergo cord blood SCT receive cyclosporine and methylprednisolone for graft-versus-host disease (GVHD) prophylaxis. Patients who undergo peripheral blood or bone marrow SCT receive cyclosporine and methotrexate (short course) for GVHD prophylaxis.

After completion of study treatment, patients are followed at 1, 3, 6, and 12 months and then annually thereafter.

PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study within 4 years.

Phase 1
Masking: None (Open Label)
Primary Purpose: Treatment
  • Neuroblastoma
  • Sarcoma
  • Unspecified Childhood Solid Tumor, Protocol Specific
  • Biological: anti-thymocyte globulin
  • Drug: busulfan
  • Drug: cyclosporine
  • Drug: melphalan
  • Drug: methotrexate
  • Drug: methylprednisolone
  • Procedure: allogeneic bone marrow transplantation
  • Procedure: peripheral blood stem cell transplantation
  • Procedure: umbilical cord blood transplantation
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
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February 2011
April 2010   (Final data collection date for primary outcome measure)


  • Diagnosis of relapsed or refractory solid tumor, including, but not limited to, relapsed neuroblastoma, relapsed Ewing's sarcoma, and relapsed rhabdomyosarcoma
  • No isolated local disease recurrence at the site of the primary tumor > 1 year after completion of prior therapy
  • No brain tumor or brain metastases
  • Related or unrelated hematopoietic stem cell donor available, meeting 1 of the following criteria:

    • Matched for HLA-A, -B, -C, -DR, and -DQ (9/10 or 10/10 allelles) (marrow or peripheral blood)
    • Matched for HLA-A, -B, and -DR (5/6 or 6/6 allelles) (cord blood)

      • Cord blood specimen must contain ≥ 2 x 10 ^7 nucleated cells/kg body weight



  • 30 and under

Performance status

  • ECOG 0-1 OR
  • Lansky 80-100%

Life expectancy

  • Not specified


  • Not specified


  • Bilirubin ≤ 3.0 mg/dL


  • Creatinine clearance ≥ 50 mL/min


  • Ejection fraction ≥ 50%


  • DLCO ≥ 70% OR
  • O_2 saturation ≥ 95% on room air


  • Not pregnant
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • HIV negative


Biologic therapy

  • Prior autologous stem cell transplantation allowed


  • Not specified

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified
Sexes Eligible for Study: All
up to 30 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
CDR0000430441 ( Registry Identifier: PDQ (Physician Data Query) )
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Milton S. Hershey Medical Center
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Principal Investigator: Kenneth G. Lucas, MD Milton S. Hershey Medical Center
National Cancer Institute (NCI)
February 2011

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