Know Cancer

forgot password

A Pilot Study of Tumor Lysate-pulsed Dendritic Cell Vaccine for Immune Augmentation for High-risk Solid Tumor Patients Following Autologous Stem Cell Transplantation

Phase 2
30 Years
Open (Enrolling)
Sarcoma, Neuroblastoma, Wilm's Tumor

Thank you

Trial Information

A Pilot Study of Tumor Lysate-pulsed Dendritic Cell Vaccine for Immune Augmentation for High-risk Solid Tumor Patients Following Autologous Stem Cell Transplantation

Localized solid tumors such as, sarcoma, neuroblastoma, and Wilms' tumor, can generally be
effectively treated with a combination of surgery, radiation and chemotherapy. However,
patients with metastatic or relapsed disease have a very poor prognosis.

For the past decade, efforts to increase overall survival and progression-free survival for
patients with high-risk pediatric and young adult tumors, have evaluated the use of
high-dose chemotherapy and hematopoietic stem cell transplantation (HSCT). The proportion of
patients who enter a complete remission with HSCT is high, ranging from 81 to 90%. While
autologous HSCT renders a large proportion of patients temporarily disease-free, relapse
develops in the majority of patients.

Survival appears to have been most improved with this strategy for neuroblastoma, but
relapses occur in the majority of patients. Similar strategies have also been tried for
patients with advanced stage sarcoma and Wilms' tumor, but relapses are even more

New approaches to the management of these difficult groups of patients are needed. There is
evidence to suggest that solid tumors may be good candidates for immunotherapy approaches.
In fact, recent experimental evidence indicates that the period of lymphopenia that occurs
after HSCT may be an opportune time to use this treatment approach. In light of the very
poor prognosis of young patients with advanced solid tumors, this treatment approach
warrants further investigation.


Inclusion Criteria:

To participate in this study, it is necessary to collect sufficient tumor and peripheral
blood stem cells to both develop the vaccine and perform the autologous stem cell
transplant. Patients who also have previously had tumor or stem cells collected, which are
available and sufficient for this study, are eligible to participate as study subjects.

1. Patients must have a histologically verified diagnosis of neuroblastoma, Wilm's
tumor, or sarcoma, including rhabdomyosarcoma, a Ewing's sarcoma family tumor (ES,
PNET), synovial sarcoma, fibrosarcoma, or desmoplastic round cell tumor.

and must meet one of the following criteria:

1. have metastatic disease at diagnosis

2. have never achieved complete remission following frontline standard therapy

3. have relapsed after receiving standard therapy

2. Patients must have been < 30 years of age at the time of original diagnosis.

3. Patients must have a source of tumor tissue from which approximately 1 gram of viable
tumor can be obtained for vaccine development.

4. Patients must have a good performance status (>70% by Lansky or Karnofsky

5. Patients must have a life expectancy of at least 16 weeks.

6. Females of child-bearing age (>= 12 years old) must have a negative pregnancy test.

Patients may enroll on this study at various points in their treatment including
diagnosis, recurrence, or just prior to initiation of study mandated transplant
therapy. Because patients may enter this study prior to completion of retransplant
treatments, the below criteria must be met only to proceed to the high dose
chemotherapy and autologous stem cell transplant part of this study. These criteria
are not a requirement to enter this study in order to collect tumor or peripheral
blood stem cells.

7. Patients must have achieved complete response or very good partial response(>= 90%
decrease in tumor volume) before proceeding to transplant. For patients enrolling on
this study just prior to transplant a VGPR or CR must be achieved to be eligible.

8. Patients may have undergone prior autologous peripheral blood stem cell
transplantation, provided at least 12 months have elapsed prior to entry on this

9. Patients must have had a successful peripheral blood stem cell collection, with
cryopreservation of PBSCs for both engraftment and for generation of dendritic cells.

10. Adequate baseline organ function must be present:

hematologic parameters (not applicable if bone marrow is involved with tumor):

1. ANC > 500/mm3

2. platelet count > 50,000/mm3

3. serum creatinine < 1.5x upper limit of normal for age

4. serum hepatic transaminases (AST, ALT) < 3x upper limit of normal

5. serum bilirubin < 1.5x upper limit of normal

6. cardiac echocardiogram with either SF > 27% or EF > 50%. A comparable EF on a
MUGA scan will also meet eligibility criteria.

11. Give or obtain informed consent

Exclusion Criteria:

1. Patients who have received prior antitumor vaccines are ineligible

2. Patients who meet the response criteria but progress prior to study enrollment are

3. Patients with known autoimmune diseases or conditions are ineligible

4. Patients with HIV infection, AIDS, hepatitis B surface antigen positivity, ongoing
bleeding, or any significant uncontrolled medical or psychiatric illness are

5. Patients under treatment for infection must cleared by BMT physician prior to

6. Patients who are pregnant or nursing are ineligible

7. Prior allogenic transplant

Type of Study:


Study Design:

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

Outcome Measure:

To estimate the rate of immune response of this immunotherapy treatment

Outcome Time Frame:

70 days

Safety Issue:


Principal Investigator

James D Geiger, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Michigan, Department of Surgery, Pediatric Section


United States: Food and Drug Administration

Study ID:

UMCC 2005.050



Start Date:

June 2006

Completion Date:

December 2013

Related Keywords:

  • Sarcoma
  • Neuroblastoma
  • Wilm's Tumor
  • tumor vaccine
  • metastatic solid tumors
  • recurrent tumors
  • Wilms Tumor
  • Neuroblastoma
  • Sarcoma



University of Michigan, Department of Surgery, Pediatric Section Ann Arbor, Michigan  48170