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

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

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

Study Overview

• Status: Completed
• Conditions: Nasopharyngeal Cancer; Fibrosarcoma; Wilms Tumor; Brain Tumor, Recurrent; Carcinoma, Round Cell
• Intervention / Treatment: Procedure: Myeloablative Chemotherapy; Procedure: Stem Cell Rescue

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
• Enrollment (Actual): 25
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • The University of Michigan

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 21 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent of Participants With Progression Free Survival at 1 Year	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.	1 year post transplant

Collaborators and Investigators

• Sponsor: University of Michigan Rogel Cancer Center

Study record dates

Study Major Dates

• Study Start: January 1, 1997
• Primary Completion (ACTUAL): December 1, 2008
• Study Completion (ACTUAL): February 1, 2010

Study Registration Dates

• First Submitted: August 31, 2005
• First Submitted That Met QC Criteria: August 31, 2005
• First Posted (ESTIMATE): September 1, 2005

Study Record Updates

• Last Update Posted (ESTIMATE): June 20, 2014
• Last Update Submitted That Met QC Criteria: May 19, 2014
• Last Verified: May 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Kidney Diseases; Urologic Diseases; Carcinoma; Neoplasms, Glandular and Epithelial; Genetic Diseases, Inborn; Pharyngeal Neoplasms; Otorhinolaryngologic Neoplasms; Head and Neck Neoplasms; Nasopharyngeal Diseases; Pharyngeal Diseases; Stomatognathic Diseases; Otorhinolaryngologic Diseases; Kidney Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplastic Syndromes, Hereditary; Neoplasms, Complex and Mixed; Neoplasms, Connective Tissue; Sarcoma; Neoplasms, Fibrous Tissue; Nasopharyngeal Carcinoma; Nasopharyngeal Neoplasms; Brain Neoplasms; Wilms Tumor; Fibrosarcoma
• Other Study ID Numbers: UMCC 9626; IRB 1996-195