Safety and Efficacy of ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematologic Malignancies.

October 7, 2013 updated by: Fate Therapeutics

A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematological Malignancies.

The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The purpose of this research study is to determine the safety and efficacy of a reduced intensity conditioning regimen during a double umbilical cord blood unit transplant with one of the cord blood units modulated with ProHema.

-As part of this research study cord units will be modulated in the laboratory with ProHema before it is given to the participant. Two different treatment groups will be tested. Group 1: will have the second cord blood unit modulated with ProHema; Group 2: will have the first cord blood unit modulated with ProHema.

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 1

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
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Dana-Farber Cancer Institute
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital

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 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate
  • Patient must be ineligible for traditional myeloablative transplantation according to treating physician
  • Lack of 6/6 or 5/6 HLA-matched related, 8/8 HLA-matched unrelated donor, or unrelated donor not available within a time frame necessary to perform a potentially curative stem cell transplant
  • 18-65 years of age
  • ECOG Performance Status 0-2

Exclusion Criteria:

  • The following hematologic malignancies are excluded:

    • Myelofibrosis unless there has been exposure to cytotoxic chemotherapy for the treatment of progression to acute myeloid leukemia
    • Chronic Myelogenous Leukemia, unless there has been exposure to cytotoxic chemotherapy for the treatment of blast phase, 3) Aplastic anemia, in the absence of transformation to Myelodysplastic disorder
  • Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular dysfunction as measured by gated radionucleotide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension
  • Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected hemoglobin
  • Renal disease: serum creatinine > 2.0mg/dl
  • Hepatic disease: serum bilirubin > 2.0mg/dl (expect in the case of Gilbert's syndrome or ongoing hemolytic anemia), SGOT or SGPT > 3 x upper limit of normal
  • Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation
  • HIV antibody
  • Uncontrolled infection
  • Pregnancy or breast feeding mother
  • Inability to comply with the requirements for care after allogeneic stem cell transplantation

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: NON_RANDOMIZED
  • Interventional Model: FACTORIAL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
ACTIVE_COMPARATOR: Group 1
2 umbilical cord units: Second cord blood unit modulated with ProHema
Drug: Melphalan
100 mg/m2/day IV x 1 day
Drug: Fludarabine
30mg/m2/day IV x 6 days
Drug: Antithymocyte Globulin
1mg/kg/day x 4 days
Drug: Sirolimus
GVHD Prophylaxis: Target range 3-12 ng/ml
Drug: Tacrolimus
GVHD Prophylaxis: Target range 5-10 ng/ml
ACTIVE_COMPARATOR: Group 2
2 umbilical cord units: First cord blood unit modulated with ProHema
Drug: Melphalan
100 mg/m2/day IV x 1 day
Drug: Fludarabine
30mg/m2/day IV x 6 days
Drug: Antithymocyte Globulin
1mg/kg/day x 4 days
Drug: Sirolimus
GVHD Prophylaxis: Target range 3-12 ng/ml
Drug: Tacrolimus
GVHD Prophylaxis: Target range 5-10 ng/ml

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Determine the safety of ProHema modulated umbilical cord blood units when used for transplantation in a reduced intensity, sequential umbilical cord blood transplantation model.
Time Frame: 2 years
2 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Time to engraftment of umbilical cord blood units
Time Frame: 2 years
2 years
Fractional chimerism of transplanted cord blood units
Time Frame: 2 years
2 years
Rates of acute and chronic GVHD
Time Frame: 2 years
2 years
30- and 100-day treatment related mortality
Time Frame: 2 years
2 years
Measures of immune reconstitution and relapse-free and overall survival at 1 and 2 years after transplantation
Time Frame: 2 years
2 years

Collaborators and Investigators

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

Sponsor

Fate Therapeutics

Collaborators

Dana-Farber Cancer Institute

Investigators

  • Principal Investigator: Corey Cutler, MD, MPH, FRCP(C), Dana-Farber Cancer Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

January 1, 2011

Primary Completion (ACTUAL)

May 1, 2013

Study Completion (ACTUAL)

October 1, 2013

Study Registration Dates

First Submitted

April 28, 2009

First Submitted That Met QC Criteria

April 28, 2009

First Posted (ESTIMATE)

April 29, 2009

Study Record Updates

Last Update Posted (ESTIMATE)

October 8, 2013

Last Update Submitted That Met QC Criteria

October 7, 2013

Last Verified

October 1, 2013

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FT1050-01

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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