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Myeloablative Chemotherapy With Stem Cell Rescue for Rare Poor-Prognosis Cancers

Phase 2
21 Years
Open (Enrolling)
Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer, Brain Tumor, Recurrent

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Trial Information

Myeloablative Chemotherapy With Stem Cell Rescue for Rare Poor-Prognosis Cancers

This is a phase II trial designed to provide a transplant option for patients with rare
poor-prognosis cancers. The protocol is only open to patients with metastatic or relapsed
cancers for whom the probability of remaining free of progressive disease for one year after
being brought into remission is < 25%. Patients eligible for this study have been diagnosed
with a form of cancer that leads to death more than 75% of the time when treated with
standard therapy doses of chemotherapy and/ or radiation therapy. Under this treatment
intensification protocol the expectation is that the one year progression-free survival for
this group of patients will rise to 40%. Patients eligible for this protocol will be
followed for one year post-transplant. Patients alive and free of progressive disease at
the end of this period will be considered successes.

Inclusion Criteria:

- Patients must be ineligible for other IRB-approved myeloablative regimens, be 21
years old or younger, and must have a histologically-confirmed Wilms' tumor, liver
cancer, recurrent brain tumor of childhood, nasopharyngeal carcinoma, fibrosarcoma,
desmoplastic small round cell tumor, germ cell tumor or other small round cell tumor,

1. is metastatic and has < 25% cure rate with conventional treatment; or

2. progressed after prior chemotherapy and has < 25% salvage rate with
non-myeloablative therapies.

- Disease status: Within 3 weeks of initiation of this protocol, patients must:

1. be in a complete or good partial remission (section 7.4); or

2. have a "chemosensitive" tumor, which is defined as a > 50% decrease in at least
one measurable tumor parameter attributable to prior chemotherapy, without
evidence of progressive disease by any other parameter.

- Prior chemotherapy: Before entry to this protocol, patients must have derived maximal
benefit from conventional, i.e., nonmyeloablative, doses of combination chemotherapy.
Conventional therapy should be continued until either a complete remission is
achieved, no further benefit from non-myeloablative dosing can be appreciated, or
toxicity from conventional therapy is perceived as limiting in the absence of stem
cell rescue. The cancer must be proven to be sensitive to alkylating agents. This
means that, in addition to, or as part of, the appropriate chemotherapy protocol for
the specific cancer in question, all patients must have received and responded to a
minimum of:

1. 2 courses of high-dose cyclophosphamide, totaling > 4200 mg/m2; or

2. courses of high-dose ifosfamide totaling > 12 gm/m2.

3. 1 course of "a)" above, plus 1 course of 'b)" above.

4. Equivalent high dose alkylating agents as described in 3.3 a, b, and c.

- Patients must have adequate renal hepatic, and cardiac function (sections 4.4-4.6).

- Patients must meet at least one of the following stem cell requirements (Peripheral
blood collection is to be preferred when available as an option):

1. Harvested bone marrow must contain 1 x 108 nucleated cells per kg of body
weight, or,

2. Peripheral blood collection should include at least 2 x 106 CD34+ cells/kg.

- Informed consent must be signed indicating patient and/or parental awareness of the
investigational nature of this program

Type of Study:


Study Design:

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

Outcome Measure:

To improve the long-term disease-free survival of patients with rare cancers at high risk for lethal relapse.

Outcome Time Frame:

subject's lifetime

Safety Issue:


Principal Investigator

John E. Levine, MS MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

The Univeristy of Michigan


United States: Institutional Review Board

Study ID:

UMCC 9626



Start Date:

January 1997

Completion Date:

January 2014

Related Keywords:

  • Wilms Tumor
  • Fibrosarcoma
  • Carcinoma, Round Cell
  • Nasopharyngeal Cancer
  • Brain Tumor, Recurrent
  • Brain Neoplasms
  • Carcinoma
  • Fibrosarcoma
  • Wilms Tumor
  • Nasopharyngeal Neoplasms



The University of Michigan Ann Arbor, Michigan  48109