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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Fate Therapeutics
ClinicalTrials.gov Identifier:
NCT01527838
First received: February 1, 2012
Last updated: October 7, 2013
Last verified: October 2013

February 1, 2012
October 7, 2013
January 2012
October 2013   (final data collection date for primary outcome measure)
Neutrophil engraftment/chimerism [ Time Frame: Day 42 ] [ Designated as safety issue: No ]
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.
Netrophil engraftment/chimerism [ Time Frame: Day 42 ] [ Designated as safety issue: No ]
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.
Complete list of historical versions of study NCT01527838 on ClinicalTrials.gov Archive Site
  • Safety [ Time Frame: Day 100 ] [ Designated as safety issue: Yes ]
    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.
  • Immune reconstitution [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To assess immune reconstitution (B-, T-, and NK-cells).
  • Donor search [ Time Frame: Day 0 ] [ Designated as safety issue: No ]
    To determine time from the initiation of donor search to transplantation.
Same as current
Not Provided
Not Provided
 
Single Treatment With FT1050 of an Ex-vivo Modulated Umbilical Cord Blood Unit
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.

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.

Interventional
Phase 1
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • 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
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood
Experimental: Single FT1050 treated UCB Unit
Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood
Intervention: Biological: Single FT1050 treated UCB unit
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
October 2013
October 2013   (final data collection date for primary outcome measure)

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.
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01527838
FT1050-02
Yes
Fate Therapeutics
Fate Therapeutics
Not Provided
Study Director: Pratik Multani, MD Fate Therapeutics
Fate Therapeutics
October 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP