A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease
N/A
30 Years
Both
Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Trial Information
A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease
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.
Inclusion 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
Type of Study:
Interventional
Study Design:
Masking: Open Label, Primary Purpose: Treatment
Principal Investigator
Kenneth G. Lucas, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Milton S. Hershey Medical Center
Authority:
United States: Federal Government
Study ID:
CDR0000430441
NCT ID:
NCT00112645
Start Date:
April 2005
Completion Date:
Related Keywords:
- Neuroblastoma
- Sarcoma
- Unspecified Childhood Solid Tumor, Protocol Specific
- unspecified childhood solid tumor, protocol specific
- recurrent neuroblastoma
- recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor
- recurrent childhood rhabdomyosarcoma
- Neuroblastoma
- Neoplasms
- Sarcoma
Name | Location |
|---|---|
| Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center | Hershey, Pennsylvania 17033-0850 |
