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A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies

Primary Purpose

Hematologic Malignancies

Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Biological: ProHema-CB
Sponsored by
Fate Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hematologic Malignancies

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male and female subjects aged 1 to 18 years, inclusive.

  2. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.

    1. Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
    2. Acute Lymphoblastic Leukemia (ALL) in CR
    3. NK cell lymphoblastic leukemia in any CR
    4. Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
    5. Myelodysplastic Syndrome (MDS) at any stage.
    6. Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial.
  3. 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, as determined by the transplant physician.
  4. Availability of suitable primary and secondary umbilical cord blood (UCB) units.

  5. Adequate performance status, defined as:

    1. Subjects ≥ 16 years: Karnofsky score ≥ 70%.
    2. Subjects < 16 years: Lansky score ≥ 70%.
  6. Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%.

  7. Pulmonary:

    1. Subjects > 10 years: DLCO (diffusion capacity) > 50% of predicted (corrected for hemoglobin)
    2. FEV1, FVC > 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation > 92% on room air.
  8. Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70mL/min/1.73m2.
  9. Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN.
  10. Signed IRB approved Informed Consent Form (ICF).

Exclusion Criteria:

  1. Female subjects that are pregnant or breastfeeding.
  2. Evidence of HIV infection or HIV positive serology.
  3. Current uncontrolled bacterial, viral or fungal infection.
  4. Prior allogeneic hematopoietic stem cell transplant.
  5. Autologous transplant < 12 months prior to enrollment.
  6. Prior autologous transplant for the disease for which the UCB transplant is being performed.
  7. Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment.
  8. Inability to receive TBI.
  9. Requirement of supplemental oxygen.
  10. HLA-matched related donor able to donate.
  11. Use of an investigational drug within 30 days prior to screening.
  12. Subject is unlikely to comply with the protocol requirements, instructions and study-related restrictions

Sites / Locations

  • City of Hope
  • Boston Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ProHema-CB

Arm Description

All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour.

Outcomes

Primary Outcome Measures

Safety Profile, Primarily Assessed by Neutrophil Engraftment
To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.

Secondary Outcome Measures

Full Information

First Posted
September 25, 2014
Last Updated
February 28, 2018
Sponsor
Fate Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT02354417
Brief Title
A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
Official Title
A Phase 1 Trial of a Single ProHema® CB Product (Ex Vivo Modulated Human Cord Blood Cells) Following Myeloablative Conditioning for Pediatric Patients With Hematologic Malignancies
Study Type
Interventional

2. Study Status

Record Verification Date
February 2018
Overall Recruitment Status
Terminated
Study Start Date
December 2014 (undefined)
Primary Completion Date
December 2016 (Actual)
Study Completion Date
February 2017 (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 is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
Detailed Description
A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18 subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1 to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled simultaneously. All subjects will be admitted to the hospital, per institutional practice, and will receive a myeloablative conditioning regimen, after which they will receive an HLA-matched or partially matched ProHema-CB unit on study Day 0.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
3 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ProHema-CB
Arm Type
Experimental
Arm Description
All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour.
Intervention Type
Biological
Intervention Name(s)
Biological: ProHema-CB
Intervention Description
Each subject will receive one administration of ProHema-CB unit transplant.
Primary Outcome Measure Information:
Title
Safety Profile, Primarily Assessed by Neutrophil Engraftment
Description
To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.
Time Frame
Neutrophil engraftment by Day 42

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male and female subjects aged 1 to 18 years, inclusive. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate. Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR Acute Lymphoblastic Leukemia (ALL) in CR NK cell lymphoblastic leukemia in any CR Biphenotypic or undifferentiated leukemia in 1st or subsequent CR Myelodysplastic Syndrome (MDS) at any stage. Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial. 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, as determined by the transplant physician. Availability of suitable primary and secondary umbilical cord blood (UCB) units. Adequate performance status, defined as: Subjects ≥ 16 years: Karnofsky score ≥ 70%. Subjects < 16 years: Lansky score ≥ 70%. Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%. Pulmonary: Subjects > 10 years: DLCO (diffusion capacity) > 50% of predicted (corrected for hemoglobin) FEV1, FVC > 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation > 92% on room air. Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70mL/min/1.73m2. Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN. Signed IRB approved Informed Consent Form (ICF). Exclusion Criteria: Female subjects that are pregnant or breastfeeding. Evidence of HIV infection or HIV positive serology. Current uncontrolled bacterial, viral or fungal infection. Prior allogeneic hematopoietic stem cell transplant. Autologous transplant < 12 months prior to enrollment. Prior autologous transplant for the disease for which the UCB transplant is being performed. Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment. Inability to receive TBI. Requirement of supplemental oxygen. HLA-matched related donor able to donate. Use of an investigational drug within 30 days prior to screening. Subject is unlikely to comply with the protocol requirements, instructions and study-related restrictions
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chris Storgard, MD
Organizational Affiliation
Fate Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
City of Hope
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115-5450
Country
United States

12. IPD Sharing Statement

Learn more about this trial

A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies

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