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



Status:Completed
Conditions:Blood Cancer, Lymphoma, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:Any
Updated:11/18/2012
Start Date:January 2012
End Date:December 2013
Contact:Elizabeth Wieland
Email:ELIZABETH.WIELAND@FATETHERAPEUTICS.COM
Phone:805-208-5232

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A Phase I/II 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 24 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 dosing cohorts of 6 subjects each, followed
by an expansion cohort of 6 additional subjects enrolled at the minimally acceptable TNC
dose level.


The trial will be conducted in three sequential dosing cohorts of 6 subjects each, followed
by an expansion cohort of 6 additional subjects enrolled at the minimally acceptable TNC
dose level. 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.

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)

- Myelodysplastic disease

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)

- Chronic Myelogenous Leukemia (CML), unless there has been exposure to cytotoxic
chemotherapy for the treatment of blast phase or there has been demonstrated
intolerance to at least 2 tyrosine kinase inhibitors.

- Aplastic anemia, in the absence of transformation to Myelodysplastic disorder.

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.
We found this trial at
3
sites
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Columbus, OH
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
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Boston, MA
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Boston, MA
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