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

Primary Purpose

Non-Hodgkin's Lymphoma (NHL), Hodgkin's Disease, Chronic Lymphocytic Leukemia (CLL)

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Single FT1050 treated UCB unit

About this trial

This is an interventional treatment trial for Non-Hodgkin's Lymphoma (NHL) focused on measuring Hematologic malignancies

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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)
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.
Age 18-65 years.
Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
Signed IRB approved Informed Consent Form (ICF).

Exclusion Criteria:

The following hematologic malignancies are excluded:
- Myelofibrosis (Agnogenic Myeloid Metaplasia)
- Aplastic anemia.
Previous treatment that included an allogeneic transplant
Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular
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.
Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected for hemoglobin.
Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40 mL/min
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.
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.
Participation in a concurrent clinical trial with a novel, unapproved investigational agent < 30 days prior to Day 0.

Sites / Locations

Massachusetts General Hospital
Dana Farber Cancer Institute-Hematopoietic Stem Cell Transplant Program
Ohio State Univeristy Comprehensive Cancer Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single FT1050 treated UCB Unit

Arm Description

Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

Outcomes

Primary Outcome Measures

Neutrophil engraftment/chimerism

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

Safety

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

To assess immune reconstitution (B-, T-, and NK-cells).

Donor search

To determine time from the initiation of donor search to transplantation.

Full Information

NCT ID

NCT01527838

First Posted

February 1, 2012

Last Updated

September 9, 2016

Sponsor

Fate Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT01527838

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

January 2015

Overall Recruitment Status

Completed

Study Start Date

January 2012 (undefined)

Primary Completion Date

October 2013 (Actual)

Study Completion Date

November 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Fate Therapeutics

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Non-Hodgkin's Lymphoma (NHL), Hodgkin's Disease, Chronic Lymphocytic Leukemia (CLL), Acute Myelogenous Leukemia (AML), Acute Lymphoblastic Leukemia (ALL)

Keywords

Hematologic malignancies

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Single FT1050 treated UCB Unit

Arm Type

Experimental

Arm Description

Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

Intervention Type

Biological

Intervention Name(s)

Single FT1050 treated UCB unit

Intervention Description

Ex-vivo CXCR4 upregulated hematopoietic progenitor cells, cord blood

Primary Outcome Measure Information:

Title

Neutrophil engraftment/chimerism

Description

Time Frame

Day 42

Secondary Outcome Measure Information:

Title

Safety

Description

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.

Time Frame

Day 100

Title

Immune reconstitution

Description

To assess immune reconstitution (B-, T-, and NK-cells).

Time Frame

2 years

Title

Donor search

Description

To determine time from the initiation of donor search to transplantation.

Time Frame

Day 0

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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) 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. Age 18-65 years. Eastern Cooperative Oncology Group (ECOG) performance status 0-2. Signed IRB approved Informed Consent Form (ICF). Exclusion Criteria: The following hematologic malignancies are excluded: Myelofibrosis (Agnogenic Myeloid Metaplasia) Aplastic anemia. Previous treatment that included an allogeneic transplant Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular 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. Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected for hemoglobin. Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40 mL/min 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. 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. Participation in a concurrent clinical trial with a novel, unapproved investigational agent < 30 days prior to Day 0.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Samuel Dychter, MD

Organizational Affiliation

Fate Therapeutics

Official's Role

Study Director

Facility Information:

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02214

Country

United States

Facility Name

Dana Farber Cancer Institute-Hematopoietic Stem Cell Transplant Program

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02215

Country

United States

Facility Name

Ohio State Univeristy Comprehensive Cancer Center

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43210

Country

United States

12. IPD Sharing Statement

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

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