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Exploratory Study of Non-Myeloablative Allogeneic Stem Cell Transplantation and Donor Lymphocyte Infusions for Metastatic Neoplasms Refractory to Standard Therapy

10 Years
80 Years
Not Enrolling
Cholangiocarcinoma, Colon/Rectal Ca, Bladder Ca, Breast Ca, Esophageal/Gastric Ca, Hepatocellular Ca, Ovarian Ca, Prostate Ca, Pancreatic Ca, Bony/Soft Tissue Sarcoma

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Trial Information

Exploratory Study of Non-Myeloablative Allogeneic Stem Cell Transplantation and Donor Lymphocyte Infusions for Metastatic Neoplasms Refractory to Standard Therapy

The main objective of this study is to identify metastatic neoplasms, which may be
susceptible to the GVT effect. We will treat patients with progressive metastatic solid
tumors refractory to standard therapy with a non-myeloablative allogeneic PBSC transplant
from a family donor. A GVT effect from immunocompetent donor immune cells could extend life
expectancy and possibly cure such patients.

Eligible patients will be treated with an allogeneic peripheral blood stem cell transplant
from an HLA identical or single HLA antigen-mismatched family donor, using an intensive
immunosuppressive regimen without myeloablation ("mini-transplant") in an attempt to
decrease the transplant related toxicities while preserving the anti-malignancy and/or
anti-host marrow effect of the graft. The low intensity non-myeloablative conditioning
regimen should provide adequate immunosuppression to allow stem cell and lymphocyte
engraftment. A T-cell replete, donor-derived, granulocyte colony stimulating factor
(G-CSF)-mobilized peripheral blood stem cells (PBSC) will be used to establish hematopoietic
and lymphoid reconstitution. We will infuse lymphocytes in patients with < 100% donor
T-cell chimerism or with evidence of tumor progression in an attempt to prevent graft
rejection and enhance a graft-versus-malignancy effect, respectively.

This trial is open to several different types of metastatic, treatment-refractory, solid
neoplasms, breast, cholangiocarcinoma, small intestine/colon/rectal adenocarcinoma,
esophageal/gastric, hepatocellular, pancreatic, prostate, and bony/soft tissue sarcomas.
The trial design permits up to 10 patients with a specific tumor type to be enrolled to
screen for anti-tumor effects. A single complete response in a specific tumor type is an
indication to exclude further patients with that diagnosis from the study. Subsequently, a
new protocol which focuses on further defining a GVT effect in that disease category will be

Inclusion Criteria



Patients with metastatic solid tumors ( breast, cholangiocarcinoma, small
intestine/colon/rectal, adenocarcinoma, esophageal/gastric, hepatocellular, pancreatic,
prostate, bony/soft tissue sarcomas, which are histologically confirmed, progressive and

Due to low accrual, effective 12/19/2006, patients with adrenal, basal cell, transitional
cell carcinoma of the bladder or uroepithelium, ovarian, small cell lung cancer, non small
cell lung cancer, and adenocarcinomas of unknown primary origin are no longer eligible for
the trial.

Age greater than or equal to 10 to less than or equal to 80.

No known standard therapy for the patient's disease that is potentially curative or
definitely capable of extending life expectancy.

Metastatic disease, which is bi-dimensionally evaluable radiographically.

No prior treatment for neoplasm within 30 days.

Ability to comprehend the investigational nature of the study and provide informed

Availability of HLA identical or single HLA-locus mismatched family donor.

Willingness and availability to return to the NIH for scheduled follow-ups.


HLA identical or single HLA-locus mismatched family donor.

Age greater than or equal to 10 up to 80 years old.

Ability to comprehend the investigational nature of the study and provide informed



Pregnant or lactating.

Age less than 10 or greater than 80 years.

ECOG performance status of 3 or more.

Psychiatric disorder or mental deficiency severe as to make compliance with the BMT
treatment unlikely, and making informed consent impossible.

Major anticipated illness or organ failure incompatible with survival from PBSC

DLCO: less than 40% predicted.

Left ventricular ejection fraction: less than 30%.

Serum creatinine greater than 2.5mg/dl or creatinine clearance less than 50 cc/min by 24
hr urine collection.

Serum bilirubin greater than 4 mg/dl

Transaminases greater than 5 times the upper limit of normal.

Oral intake less than 1,200 calories/day.

Recent weight loss of greater than or equal to 10% of actual body weight.

Life expectancy less than 3 months

Therapy for malignancy within 4 weeks of beginning protocol.

CNS metastatic disease associated with intracranial bleeding, uncontrolled seizure
disorder or significant intracranial mass effect.

Other malignant diseases liable to relapse or progress within 5 years.

Uncontrolled infection.


Pregnant or lactating.

Donor unfit to receive G-CSF and undergo apheresis. (Uncontrolled hypertension, history
of congestive heart failure or unstable angina, thrombocytopenia).

Age less than 10 or greater than 80 years.

HIV positive. Donors who are positive for HBV, HCV or HTLV-I may be used at the
discretion of the investigator following counseling and approval from the recipient.

Type of Study:


Study Design:


Outcome Measure:

To identify an anti-tumor effect of allogeneic PBSC transplantation by induction of a graft-versus-tumor (GVT) effect in patients with a diversity of metastatic solid tumors, which are refractory to standard therapy.

Principal Investigator

Richard W Childs, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Heart, Lung, and Blood Institute (NHLBI)


United States: Federal Government

Study ID:




Start Date:

March 1999

Completion Date:

Related Keywords:

  • Cholangiocarcinoma
  • Colon/Rectal Ca
  • Bladder Ca
  • Breast Ca
  • Esophageal/Gastric Ca
  • Hepatocellular Ca
  • Ovarian Ca
  • Prostate CA
  • Pancreatic Ca
  • Bony/Soft Tissue Sarcoma
  • Graft-Versus-Tumor
  • Peripheral Blood Stem Cells
  • Non-Myeloablative Bone Marrow Transplantation
  • Metastatic Solid Tumors
  • Engraftment
  • Cyclophosphamide
  • Fludarabine
  • Donor Apheresis
  • Graft-Versus-Host Disease
  • Cancer
  • Tumor
  • Urinary Bladder Neoplasms
  • Breast Neoplasms
  • Esophageal Diseases
  • Ovarian Neoplasms
  • Prostatic Neoplasms
  • Cholangiocarcinoma
  • Sarcoma



National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland  20892