Single Treatment With FT1050 of an Ex-vivo Modulated Umbilical Cord Blood Unit

September 9, 2016 updated by: Fate Therapeutics

A Phase I Trial of a Single FT1050 (16,16-Dimethyl Prostaglandin E2) Ex Vivo-Modulated Umbilical Cord Blood (CB) Unit Following a Reduced Intensity Conditioning Regimen For Adults With Hematologic Malignancies

This trial is a prospective, open-label, single-arm trial of the safety of a single FT1050-treated CB unit for hematopoietic reconstitution after a reduced-intensity conditioning regimen for hematologic malignancies. A maximum of 40 eligible adult subjects will be enrolled and treated in the trial at approximately 2-4 centers within the U.S.

Study Overview

Detailed Description

The trial will be conducted in three sequential cohorts of 6-12 evaluable subjects each.

Cohort 1 will enroll eligible subjects for whom a single CB unit has been identified that meets the minimum HLA-matching criteria and has a minimum pre-cryopreservation total nucleated cell (TNC) dose of at least 2.5 x 10^7 cells/kg. Cohort 2 is identical to Cohort 1, except that the TNC dose of the CB unit must be between 2.0 - <2.5 x 10^7 cells/kg. Finally, Cohort 3 is identical to Cohort 2, except that the TNC dose of the CB unit must be between 1.5 - <2.0 x 10^7 cells/kg. If no safety rules are triggered, the study will proceed to the next dosing cohort. Within a dosing cohort, no more than three subjects may be before Day 42 at any one time, unless they have already engrafted neutrophils. The final dosing cohort is defined as the last cohort where 12 evaluable subjects are treated and no stopping rules are triggered. The corresponding TNC dose level will be considered the minimally acceptable TNC dose level.

Study Type

Interventional

Enrollment (Actual)

10

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

    • Massachusetts
      • Boston, Massachusetts, United States, 02214
        • Massachusetts General Hospital
      • Boston, Massachusetts, United States, 02215
        • Dana Farber Cancer Institute-Hematopoietic Stem Cell Transplant Program
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State Univeristy Comprehensive Cancer Center

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:

  1. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Eligible diseases and stages include:

    • Non-Hodgkin's lymphoma or Hodgkin's lymphoma
    • Chronic lymphocytic leukemia (CLL)
    • Acute myelogenous leukemia (AML)
    • Chronic myelogenous leukemia (CML)
  2. Lack of 5-6/6 HLA-matched related or 8/8 HLA-A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe.

    • Identification of suitable backup CB unit(s) (single unit with pre-cryopreservation cell dose ≥ 2.5 x 10^7 TNC/kg or two units with pre-cryopreservation cell dose ≥ 1.5 x 10^7 TNC/kg each) and meeting minimum HLA match criteria.
    • An acceptable alternative to one or two backup CB unit(s) is the identification of an eligible related haploidentical donor that meets minimum HLA match criteria.
  3. Age 18-65 years.
  4. Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
  5. Signed IRB approved Informed Consent Form (ICF).

Exclusion Criteria:

  1. The following hematologic malignancies are excluded:

    • Myelofibrosis (Agnogenic Myeloid Metaplasia)
    • Aplastic anemia.
  2. Previous treatment that included an allogeneic transplant
  3. Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular
  4. dysfunction (Ejection fraction < 40%) as measured by gated radionucleotide ventriculogram or echocardiogram; active angina pectoris, or uncontrolled hypertension; history of myocardial infarction with depressed ejection fraction.
  5. Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected for hemoglobin.
  6. Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40 mL/min
  7. Hepatic disease: serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome or ongoing hemolytic anemia), SGOT or SGPT > 3 x upper limit of normal.
  8. Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other neuropsychiatric abnormalities believed to preclude transplantation.
  9. HIV antibody.
  10. Uncontrolled infection.
  11. Pregnancy or breast feeding mother.
  12. Inability to comply with the requirements for care after allogeneic stem cell transplantation.
  13. Participation in a concurrent clinical trial with a novel, unapproved investigational agent < 30 days prior to Day 0.

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: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single FT1050 treated UCB Unit
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Neutrophil engraftment/chimerism
Time Frame: Day 42
To determine the minimally effective TNC dose for a single FT1050-treated CB unit based on neutrophil engraftment/chimerism when used for hematopoietic reconstitution following a reduced-intensity conditioning regimen for hematologic malignancies.
Day 42

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety
Time Frame: Day 100
Define the safety profile of treatment with a single FT1050-treated CB unit. To define the preliminary efficacy of treatment with a single FT1050-treated CB unit.
Day 100
Immune reconstitution
Time Frame: 2 years
To assess immune reconstitution (B-, T-, and NK-cells).
2 years
Donor search
Time Frame: Day 0
To determine time from the initiation of donor search to transplantation.
Day 0

Collaborators and Investigators

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

Investigators

  • Study Director: Samuel Dychter, MD, Fate Therapeutics

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, 2012

Primary Completion (Actual)

October 1, 2013

Study Completion (Actual)

November 1, 2013

Study Registration Dates

First Submitted

February 1, 2012

First Submitted That Met QC Criteria

February 3, 2012

First Posted (Estimate)

February 7, 2012

Study Record Updates

Last Update Posted (Estimate)

September 12, 2016

Last Update Submitted That Met QC Criteria

September 9, 2016

Last Verified

January 1, 2015

More Information

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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