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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.
ClinicalTrials.gov Identifier: NCT00112645
Recruitment Status : Completed
First Posted : June 3, 2005
Last Update Posted : July 18, 2013
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
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 Phase 1

Detailed Description:


  • 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease
Study Start Date : April 2005
Actual Primary Completion Date : April 2010
Actual Study Completion Date : February 2011

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00112645

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United States, Pennsylvania
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033-0850
Sponsors and Collaborators
Milton S. Hershey Medical Center
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Principal Investigator: Kenneth G. Lucas, MD Milton S. Hershey Medical Center
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ClinicalTrials.gov Identifier: NCT00112645    
Other Study ID Numbers: PSCI-20615
CDR0000430441 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: June 3, 2005    Key Record Dates
Last Update Posted: July 18, 2013
Last Verified: February 2011
Keywords provided by National Cancer Institute (NCI):
unspecified childhood solid tumor, protocol specific
recurrent neuroblastoma
recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor
recurrent childhood rhabdomyosarcoma
Additional relevant MeSH terms:
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Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Antilymphocyte Serum
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Physiological Effects of Drugs
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists