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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01527838
Recruitment Status : Completed
First Posted : February 7, 2012
Last Update Posted : September 12, 2016
Information provided by (Responsible Party):
Fate Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Non-Hodgkin's Lymphoma (NHL) Hodgkin's Disease Chronic Lymphocytic Leukemia (CLL) Acute Myelogenous Leukemia (AML) Acute Lymphoblastic Leukemia (ALL) Biological: Single FT1050 treated UCB unit Phase 1

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  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : January 2012
Actual Primary Completion Date : October 2013
Actual Study Completion Date : November 2013

Arm Intervention/treatment
Experimental: Single FT1050 treated UCB Unit
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood
Biological: Single FT1050 treated UCB unit
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. Immune reconstitution [ Time Frame: 2 years ]
    To assess immune reconstitution (B-, T-, and NK-cells).

  3. Donor search [ Time Frame: Day 0 ]
    To determine time from the initiation of donor search to transplantation.

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01527838

United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02214
Dana Farber Cancer Institute-Hematopoietic Stem Cell Transplant Program
Boston, Massachusetts, United States, 02215
United States, Ohio
Ohio State Univeristy Comprehensive Cancer Center
Columbus, Ohio, United States, 43210
Sponsors and Collaborators
Fate Therapeutics
Study Director: Samuel Dychter, MD Fate Therapeutics

Responsible Party: Fate Therapeutics Identifier: NCT01527838     History of Changes
Other Study ID Numbers: FT1050-02
First Posted: February 7, 2012    Key Record Dates
Last Update Posted: September 12, 2016
Last Verified: January 2015

Keywords provided by Fate Therapeutics:
Hematologic malignancies

Additional relevant MeSH terms:
Lymphoma, Non-Hodgkin
Leukemia, Lymphoid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Hodgkin Disease
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Leukemia, B-Cell