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A Phase I Study to Examine the Toxicity of Killer IG-Like Receptor (KIR) Mismatched Umbilical Cord Blood for Pediatric Patients With Malignant Solid Tumors

Phase 1
21 Years
Open (Enrolling)
Unspecified Childhood Solid Tumor, Protocol Specific

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Trial Information

A Phase I Study to Examine the Toxicity of Killer IG-Like Receptor (KIR) Mismatched Umbilical Cord Blood for Pediatric Patients With Malignant Solid Tumors


- Examine the impact of the use of killer cell immunoglobulin-like receptor
(KIR)-mismatched umbilical cord blood as a source of hematopoietic stem cells, after
busulfan, melphalan, and anti-thymocyte globulin in pediatric patients with relapsed or
refractory solid tumors.

- Determine the toxicity of this regimen, in terms of incidence of grade 3-4 acute
graft-versus-host disease, donor/host chimerism, and cellular immunity against tumor
cell lines, in these patients.


- Transplantation: Patients receive busulfan orally or IV every 6 hours on days -8 to -5,
anti-thymocyte globulin IV over 6 hours on days -4 to -1, and melphalan IV over 15-20
minutes on days -4 to -2. Patients undergo allogeneic umbilical cord blood stem cell
infusion on day 0. Patients receive sargramostim (GM-CSF) subcutaneously beginning on
day 7 and continuing until blood counts recover.

- Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 1 hour or
orally twice daily on days -1 to 180 and methylprednisolone IV or orally once or twice
daily on days 5 - 49.

Blood samples are collected periodically for immunophenotyping and flow cytometric analysis
(including interferon gamma and other TH1 and TH2 cytokines).

After completion of study treatment, patients are followed periodically.

PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.

Inclusion Criteria


- Diagnosis of malignant solid tumor

- Relapsed or refractory disease

- No isolated local recurrence of disease (in the site of the primary tumor) > 1
year after completing therapy

- No brain tumors or brain metastases

- Unrelated cord blood donor available

- May be HLA 6/6 matched (HLA-A, -B, -DR) OR mismatched for 1, 2, or 3 of these
HLA loci, but must be mismatched for HLA-C group as indicated by their following
killer cell immunoglobulin-like receptor (KIR) group specificity:


- Cw 2

- Cw 0307

- Cw 4, 5, 6

- Cw 0707, 0709

- Cw 1204, 1205

- All other Cw 15 alleles

- Cw 1602

- Cw 17

- Cw 18


- Cw 1

- All other Cw 3 alleles

- All other Cw 7 alleles

- Cw 8

- Cw 1202, 1203, 1206

- Cw 1301

- Cw 1402, 1403

- Cw 1507

- Cw 1601, 1604

- Cord blood specimen must have ≥ 1 x 10^7 nucleated cells/kg patient ideal body weight


- ECOG performance status (PS) 0-2 OR Lansky PS 70-100%

- Cardiac ejection fraction ≥ 50%

- Creatinine clearance ≥ 50%

- Bilirubin ≤ 3.0 mg/dL

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


- Prior autologous stem cell transplantation allowed

Type of Study:


Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:


Safety Issue:


Principal Investigator

Kenneth G. Lucas, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Milton S. Hershey Medical Center


United States: Federal Government

Study ID:




Start Date:

May 2004

Completion Date:

Related Keywords:

  • Unspecified Childhood Solid Tumor, Protocol Specific
  • unspecified childhood solid tumor, protocol specific
  • Neoplasms



Penn State Cancer Institute at Milton S. Hershey Medical Center Hershey, Pennsylvania  17033-0850