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

This study has been completed.
Sponsor:
Information provided by:
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00112645
First received: June 2, 2005
Last updated: July 17, 2013
Last verified: February 2011
  Purpose

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 Intervention 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

Study Type: Interventional
Study Design: Masking: 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

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Estimated Enrollment: 10
Study Start Date: April 2005
Study Completion Date: February 2011
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Detailed Description:

OBJECTIVES:

  • 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.

OUTLINE:

  • 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.

  Eligibility

Ages Eligible for Study:   up to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • 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

PATIENT CHARACTERISTICS:

Age

  • 30 and under

Performance status

  • ECOG 0-1 OR
  • Lansky 80-100%

Life expectancy

  • Not specified

Hematopoietic

  • Not specified

Hepatic

  • Bilirubin ≤ 3.0 mg/dL

Renal

  • Creatinine clearance ≥ 50 mL/min

Cardiovascular

  • Ejection fraction ≥ 50%

Pulmonary

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

Other

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

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Prior autologous stem cell transplantation allowed

Chemotherapy

  • Not specified

Endocrine therapy

  • Not specified

Radiotherapy

  • Not specified

Surgery

  • Not specified
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00112645

Locations
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
Investigators
Principal Investigator: Kenneth G. Lucas, MD Milton S. Hershey Medical Center
  More Information

Additional Information:
No publications provided

ClinicalTrials.gov Identifier: NCT00112645     History of Changes
Other Study ID Numbers: PSCI-20615, CDR0000430441
Study First Received: June 2, 2005
Last Updated: July 17, 2013
Health Authority: United States: Federal Government

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:
Neuroblastoma
Neoplasms
Sarcoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Connective and Soft Tissue
Antilymphocyte Serum
Busulfan
Cyclosporins
Cyclosporine
Melphalan
Methotrexate
Methylprednisolone Hemisuccinate
Prednisolone
Methylprednisolone acetate
Prednisolone acetate
Methylprednisolone
Prednisolone hemisuccinate
Prednisolone phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents, Alkylating

ClinicalTrials.gov processed this record on April 14, 2014