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A Phase I Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas

Phase 1
2 Years
21 Years
Open (Enrolling)
Ewing Sarcoma, Gastrointestinal Tumor, Germ Cell Tumor, Hepatic Tumor, Lymphoma, Wilms Tumor, Rhabdoid Tumor, Clear Cell Carcinoma, Renal Cell Carcinoma, Melanoma, Neuroblastoma, Rhabdomyosarcoma, Non-rhabdomyosarcoma

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Trial Information

A Phase I Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas

Twelve participants and 12 donors will be enrolled on this study. Donors will undergo seven
days of hematopoietic stem cell (HSC) mobilization followed by two apheresis collections.
Each apheresis collection will be processed by the CliniMACS system.

DONORS: A mobilization regimen of granulocyte colony stimulating factor (G-CSF) will be
used to obtain a peripheral blood stem cell (PBSC) product from the donor. Apheresis will
be performed for a minimum of two consecutive days, including one day for each cell product

STUDY PARTICIPANTS: Participants will undergo a two-stage haploidentical cell infusion
following myeloablative conditioning. The first cell infusion will be a CD3-depleted product
and the second infusion will be a CD45RA-depleted product.

Primary Objective:

- To determine the feasibility of haploidentical HSCT using two infusions engineered by
negative selection on the Miltenyi CliniMACS system- the first by selective depletion
of CD3+ cells, followed by a second depleted of CD45RA+ cells, in children with
relapsed or refractory solid tumors or lymphomas.

Secondary Objectives:

- To estimate hematopoietic cell recovery and engraftment rates for the patients.

- To estimate infection rates and complications.

- To estimate the one-year overall survival (OS) and event-free survival (EFS) for the
study patients.

Inclusion Criteria

Inclusion Criteria - Transplant Recipients:

- At least 2 years of age and less than or equal to 21 years of age.

- Histologically confirmed solid tumor or lymphoma at original diagnosis:

- Ewing Sarcoma Family of Tumors (ESFT)

- Gastrointestinal tumors

- Germ Cell tumors

- Hepatic tumors (including hepatocellular carcinoma and hepatoblastoma)

- Lymphoma (including Hodgkin and non-Hodgkin lymphoma)

- Kidney tumors (including Wilms tumor, rhabdoid tumors, clear cell carcinoma, and
renal cell carcinoma)

- Melanoma

- Neuroblastoma

- Soft tissue sarcoma (including rhabdomyosarcoma and non-rhabdomyosarcoma soft
tissue sarcoma)

- Malignancy has no reasonable expectation of cure with available alternative salvage

- Has a suitable human leukocyte antigen (HLA) haploidentical donor available.

- At least two weeks since receipt of any biological therapy, chemotherapy, and/or
radiation therapy.

- Has recovered from all acute NCI Common Toxicity Criteria grade II-IV acute
non-hematologic toxicities from prior therapy per the judgment of the PI.

- Shortening fraction greater than or equal to 25%.

- Creatinine clearance greater than or equal to 50 mL/min/1.73 m2.

- Pulse oximetry greater than or equal to 92% on room air

- Alanine aminotransferase (ALT) and aspartate transaminase (AST) less than or equal
to3 times the upper limit of the institution-established normal range.

- Direct bilirubin less than or equal to 3.0 mg/dL.

- Karnofsky or Lansky performance score of greater than or equal to 50.

Exclusion Criteria - Transplant Recipients:

- Newly diagnosed patients with no prior attempt at curative therapy.

- Any primary or active central nervous system (CNS) malignancy, including metastatic

- Any active or prior malignant or pre-malignant condition of the bone marrow,
excluding metastasis of the primary malignancy.

- Prior allogeneic hematopoietic stem cell transplant.

- Prior autologous stem cell transplant within previous 3 months.

- Allergy to murine products or positive human anti-mouse antibody (HAMA).

- (Female only) Known pregnancy (negative serum or urine pregnancy test to be conducted
within 7 days prior to enrollment).

- (Female only) Lactating.

Inclusion Criteria - Donors:

- At least 18 years of age.

- Partially HLA matched family member.

- Human immunodeficiency virus (HIV) negative.

Exclusion Criteria - Donors:

- (Female only) Known pregnancy (negative serum or urine pregnancy test to be conducted
within 7 days prior to enrollment).

- (Female only) Lactating.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Feasibility of haploidentical HSCT

Outcome Description:

Feasibility is defined as engraftment (ANC≥ 500/mm3 for 3 consecutive tests performed on different days) evaluated before day +30.

Outcome Time Frame:

30 days post transplantation

Safety Issue:


Principal Investigator

David R. Shook, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

St. Jude Children's Research Hospital


United States: Food and Drug Administration

Study ID:




Start Date:

July 2012

Completion Date:

December 2015

Related Keywords:

  • Ewing Sarcoma
  • Gastrointestinal Tumor
  • Germ Cell Tumor
  • Hepatic Tumor
  • Lymphoma
  • Wilms Tumor
  • Rhabdoid Tumor
  • Clear Cell Carcinoma
  • Renal Cell Carcinoma
  • Melanoma
  • Neuroblastoma
  • Rhabdomyosarcoma
  • Non-rhabdomyosarcoma
  • Carcinoma
  • Carcinoma, Renal Cell
  • Digestive System Neoplasms
  • Gastrointestinal Neoplasms
  • Liver Neoplasms
  • Lymphoma
  • Melanoma
  • Wilms Tumor
  • Neuroblastoma
  • Rhabdomyosarcoma
  • Neoplasms, Germ Cell and Embryonal
  • Adenocarcinoma, Clear Cell
  • Adenomyoepithelioma
  • Rhabdoid Tumor
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma



St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794