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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
John Levine, MD, University of Michigan Cancer Center
ClinicalTrials.gov Identifier:
NCT00141765
First received: August 31, 2005
Last updated: May 19, 2014
Last verified: May 2014
  Purpose

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


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

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Myeloablative Chemotherapy With Stem Cell Rescue for Rare Poor-Prognosis Cancers

Resource links provided by NLM:


Further study details as provided by University of Michigan Cancer Center:

Primary Outcome Measures:
  • Percent of Participants With Progression Free Survival at 1 Year [ Time Frame: 1 year post transplant ] [ Designated as safety issue: No ]
    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.


Enrollment: 25
Study Start Date: January 1997
Study Completion Date: February 2010
Primary Completion Date: December 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00141765

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

No publications provided

Responsible Party: John Levine, MD, Professor of Pediatrics and of Internal Medicine, University of Michigan Cancer Center
ClinicalTrials.gov Identifier: NCT00141765     History of Changes
Other Study ID Numbers: UMCC 9626, IRB 1996-195
Study First Received: August 31, 2005
Results First Received: May 19, 2014
Last Updated: May 19, 2014
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on July 10, 2014