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

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.
 
ClinicalTrials.gov Identifier: NCT02354417
Recruitment Status : Terminated
First Posted : February 3, 2015
Results First Posted : October 10, 2018
Last Update Posted : October 10, 2018
Sponsor:
Information provided by (Responsible Party):
Fate Therapeutics

Tracking Information
First Submitted Date  ICMJE September 25, 2014
First Posted Date  ICMJE February 3, 2015
Results First Submitted Date  ICMJE February 28, 2018
Results First Posted Date  ICMJE October 10, 2018
Last Update Posted Date October 10, 2018
Study Start Date  ICMJE December 2014
Actual Primary Completion Date December 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 28, 2018)
Safety Profile, Primarily Assessed by Neutrophil Engraftment [ Time Frame: Neutrophil engraftment by Day 42 ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 29, 2015)
Safety Profile, Primarily Assessed by Neutrophil Engraftment [ Time Frame: Up to 2 months ]
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.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
Official Title  ICMJE 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
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hematologic Malignancies
Intervention  ICMJE Biological: Biological: ProHema-CB
Each subject will receive one administration of ProHema-CB unit transplant.
Study Arms  ICMJE Experimental: ProHema-CB

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: Biological: Biological: ProHema-CB
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 11, 2016)
3
Original Estimated Enrollment  ICMJE
 (submitted: January 29, 2015)
18
Actual Study Completion Date  ICMJE February 2017
Actual Primary Completion Date December 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02354417
Other Study ID Numbers  ICMJE FT1050-04
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Fate Therapeutics
Study Sponsor  ICMJE Fate Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Chris Storgard, MD Fate Therapeutics
PRS Account Fate Therapeutics
Verification Date February 2018

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