Pilot Study of Umbilical Cord Blood Transplantation in Adult Patient With Advanced Hematopoietic Malignancies

August 13, 2013 updated by: University of California, San Francisco
This is a pilot study designed to evaluate the safety and feasibility of performing umbilical cord blood transplants in adults with high-risk hematopoietic malignancies. A novel myeloablative preparative regimen will be used. One, up to a maximum of three cord blood units will be administered to facilitate engraftment.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

This study intends to demonstrate an engraftment rate of >80% at day 100 post-transplantation and a transplant related mortality rate of < or equal to 50%. A TRM of >50% will be considered unacceptable. The present research will also:

  • Evaluate the toxicity of busulfan, fludarabine, and etoposide as preparative therapy prior to umbilical cord blood cell transplantation.
  • Evaluate neutrophil and platelet recovery following UCB transplantation.
  • Evaluate lineage-specific chimerism following transplantation and to assess the contribution of each individual CB unit to post-transplantation hematopoiesis.
  • Evaluate event free and overall survival.
  • Evaluate the incidence, severity and timing of acute and chronic GVHD following UCB transplantation.

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94143
        • University of California, San Francisco

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 55 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Age < or equal to 55
  • Availability of donor cord blood (one to three units) matching at least 4 of 6 HLA antigens (A, B, and DR). HLA class I antigens will be determined by serologic methods, and Class II antigens will be determined by high-resolution DNA typing. Typing will be confirmed by UCSF Immunogenetics Department following infusion. The UCB units must contain >2.5 x 10(7) TNC per kilogram recipient body weight. Cord blood units will be obtained from all available international banks.
  • HLA identical or 1 antigen mismatched related donors or potential HLA-matched unrelated donors (MUD) matching at >6/8 (A, B,C, DR) alleles must NOT be available.

  • Disease types:

    • Acute myeloid leukemia not expected to be curable with chemotherapy. This will include patients with high-risk cytogenetics (-7, -7q, -5, -5q, t(6,9), t(9,11), complex, Ph+), evolution from prior myelodysplasia or AML secondary to prior chemotherapy, failure to achieve remission, or second or subsequent remission. To ensure adequate time until disease progression, marrow blasts must be < or equal to 10%. This may be achieved using chemotherapy treatment.
    • Myelodysplasia with high-risk features. This will include patients with IPSS category INT2 or HI-risk MDS. Marrow blasts must be < or equal to 20%. If required, chemotherapy may be given to achieve target levels of blasts.
    • Acute lymphoblastic leukemia not expected to be curable with chemotherapy. This will include patients with high-risk cytogenetics (Ph+, t(4,11), 11q23 abnormalities, and monosomy 7), patients requiring more than one induction course to achieve remission, as well as patients failing to enter remission or in second or subsequent remission. To ensure adequate time until disease progression, marrow blasts must be < or equal to 10%. If required, chemotherapy may be given to achieve target levels of blasts.
    • Chronic myelogenous leukemia with advanced disease. This will include patients with accelerated or blastic phase or patients with chronic phase refractory to STI-5741. To ensure adequate time until disease progression, patients with blast crisis must show marrow blasts < or equal to 10%. If required, chemotherapy may be given to achieve target levels of blasts.
    • Multiple myeloma, stage II-III with >1st relapse or refractory disease or newly diagnosed with chromosome 13 abnormalities.
    • Lymphoma: diffuse large cell, mantle cell, peripheral T-cell, T-NK cell, or Hodgkin's disease which has failed to respond to primary therapy, progressed or recurred after prior therapy. Patients who have failed autologous transplant are eligible if they are >1 year post-transplant.
  • Patients must have an ECOG PS< or equal to 2
  • Laboratory requirements:
  • Creatinine <2.0mg/dL and creatinine clearance >40/m/min (calculated or based on 24 hour urine collection)
  • Bilirubin <2.0 mg/dL, AST/alkaline phosphatase <3x upper limit of normal
  • Patients with hepatitis C and active Hepatitis B are eligible only if a liver biopsy is performed and there is a < or equal to grade 2 inflammation or fibrosis.
  • Cardiac ejection fraction >40%
  • DLCO >40%
  • Negative pregnancy test (females of reproductive age)

Exclusion Criteria:

  • Active infection requiring ongoing antibiotic treatment
  • HIV infection
  • Poor performance status (ECOG >2)
  • Rapid progression of malignant disease
  • Opinion of BMT Committee that autologous transplant would be a preferable form of treatment
  • Organ function is below requirements
  • Pregnancy or breast-feeding

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
efficacy
Time Frame: 5 years
5 years
safety of umbilical cord transplant
Time Frame: 5 years
5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
safety of umbilical cord stem cell transplant
Time Frame: 5 years
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of California, San Francisco

Investigators

  • Principal Investigator: Thomas G. Martin, M.D., University of California, San Francisco

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2002

Primary Completion (Actual)

March 1, 2009

Study Completion (Actual)

March 1, 2009

Study Registration Dates

First Submitted

August 8, 2007

First Submitted That Met QC Criteria

August 9, 2007

First Posted (Estimate)

August 10, 2007

Study Record Updates

Last Update Posted (Estimate)

August 14, 2013

Last Update Submitted That Met QC Criteria

August 13, 2013

Last Verified

August 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UC2207

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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