Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00141765
Recruitment Status : Completed
First Posted : September 1, 2005
Results First Posted : June 20, 2014
Last Update Posted : June 20, 2014
Information provided by (Responsible Party):
John Levine, MD, University of Michigan Cancer Center

Brief Summary:
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.

Condition or disease Intervention/treatment Phase
Wilms Tumor Fibrosarcoma Carcinoma, Round Cell Nasopharyngeal Cancer Brain Tumor, Recurrent Procedure: Myeloablative Chemotherapy Procedure: Stem Cell Rescue Phase 2

Detailed Description:
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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Myeloablative Chemotherapy With Stem Cell Rescue for Rare Poor-Prognosis Cancers
Study Start Date : January 1997
Actual Primary Completion Date : December 2008
Actual Study Completion Date : February 2010

Arm Intervention/treatment
Experimental: Myeloablative Chemotherapy with Stem Cell Rescue
Myeloablative Chemotherapy, followed by stem cell rescue
Procedure: Myeloablative Chemotherapy
High dose chemotherapy (carboplatin and thiotepa) transplant rescue
Procedure: Stem Cell Rescue
autologous stem cell transplantation

Primary Outcome Measures :
  1. Percent of Participants With Progression Free Survival at 1 Year [ Time Frame: 1 year post transplant ]
    The primary outcome measure for this study was to improve the long-term disease-free survival of patients with rare cancers at high risk for lethal relapse.

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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, which:

    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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00141765

United States, Michigan
The University of Michigan
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
University of Michigan Cancer Center
Principal Investigator: John E. Levine, MS MD The Univeristy of Michigan

Responsible Party: John Levine, MD, Professor of Pediatrics and of Internal Medicine, University of Michigan Cancer Center Identifier: NCT00141765     History of Changes
Other Study ID Numbers: UMCC 9626
IRB 1996-195
First Posted: September 1, 2005    Key Record Dates
Results First Posted: June 20, 2014
Last Update Posted: June 20, 2014
Last Verified: May 2014

Additional relevant MeSH terms:
Wilms Tumor
Nasopharyngeal Neoplasms
Brain Neoplasms
Neoplasms, Complex and Mixed
Neoplasms by Histologic Type
Kidney Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Neoplastic Syndromes, Hereditary
Kidney Diseases
Urologic Diseases
Genetic Diseases, Inborn
Pharyngeal Neoplasms
Otorhinolaryngologic Neoplasms
Head and Neck Neoplasms
Nasopharyngeal Diseases
Pharyngeal Diseases
Stomatognathic Diseases
Otorhinolaryngologic Diseases
Neoplasms, Fibrous Tissue
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Central Nervous System Neoplasms
Nervous System Neoplasms
Brain Diseases
Central Nervous System Diseases