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Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide

Primary Purpose

Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome

Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Cyclophosphamide
Tacrolimus
Mycophenolate mofetil
Haploidentical Stem Cell Transplantation
Sponsored by
Loyola University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Acute Myeloid Leukemia focused on measuring NHL, Non-Hodgkin Lymphoma, Haploidentical, Bone Marrow Transplant, Stem Cell Transplant, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, GVHD

Eligibility Criteria

16 Years - 90 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Ages 16 years old and up
  • Performance Status 70 percent or above
  • Patients should have the following diseases:
  • Acute myelogenous leukemia (AML)
  • Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL)
  • Transfusion dependent myelodysplastic syndrome (MDS)
  • Non-Hodgkin's Lymphoma (NHL)
  • Chronic lymphocytic leukemia (CLL)
  • Pulmonary function as measured by forced expiratory volume at one second (FEV1) and/or corrected diffusing capacity of lung for carbon monoxide (DLCO) at 60 percent of predicted or above
  • Left ventricular ejection fraction at 45 percent or above
  • If the donor-specific HLA antibodies (DSA) are positive, the patient must undergo a desensitization protocol resulting in undetectable DSA prior to day of transplant

Exclusion Criteria:

  • Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea)
  • Uncontrolled bacterial, fungal or viral infections at time of study enrollment
  • Positive for HIV, human T-cell leukemia virus (HTLV-1) and/or Hepatitis C
  • Subjects with signs/symptoms of active central nervous system (CNS) disease

Sites / Locations

  • Loyola University Medical CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

All patients will receive Haploidentical

Arm Description

The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD. All patients will receive a Haplo-identical stem cell transplantation. GVHD Prevention Treatment: Cyclophosphamide 50mg/kg will be administered IV on Day 3 and Day 5 post transplant. Tacrolimus 0.03 mg/kg daily will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant. Mycophenolate mofetil 15mg/kg will be administered twice a day IV until patient can take it by mouth starting on Day 1 post transplant until 28 days.

Outcomes

Primary Outcome Measures

Chimerism
Blood test that measures amount of donor's cells

Secondary Outcome Measures

Neutrophil engraftment
Blood test that measures the white cell count
Platelet engraftment
Blood test that measures the platelet count
Grade 3 to 4 acute graft-verus-host disease (GVHD)
National Institutes of Health Acute Graft-Versus-Host Disease Grading and Form
Frequency and severity of chronic GVHD
National Institutes of Health Chronic Graft-Versus-Host Disease Grading and Form
Disease status with blast counts (immature blood cell count) above 5%
Blood work and/or bone marrow biopsy will be used
Survival status by patient contact
Contact with patient by phone or doctor's visit
Immune reconstitution
Blood work will be used to evaluate recovery of T and B cell count subset that assess cells which make antibodies to fight infections
Grade 3 through 5 Adverse Events
Toxicities that are possibly, probably, and definitely related to study treatment according to NCI CTCAE Version 4

Full Information

First Posted
February 22, 2017
Last Updated
April 22, 2021
Sponsor
Loyola University
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1. Study Identification

Unique Protocol Identification Number
NCT03088709
Brief Title
Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide
Official Title
Safety, Efficacy and Feasibility of Haploidentical Stem Cell Transplantation (Haplo-SCT) Using Post-Transplant Cyclophosphamide (PTCy) as an Alternative Donor Source for Patients Who Lack a Matched Sibling/Unrelated Donor Options
Study Type
Interventional

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Unknown status
Study Start Date
January 18, 2017 (Actual)
Primary Completion Date
January 31, 2022 (Anticipated)
Study Completion Date
January 31, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Loyola University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Historically, the best results of allogeneic SCT have been obtained when the stem cell donor is a human leukocyte antigen (HLA)-matched sibling, however, this is only available for approximately 30 percent of patients in need for SCT. Alternative donor sources include matched unrelated donor utilizing the donor registry, cord blood transplant and mismatched donor transplant. A human leukocyte antigen (HLA)-haploidentical donor is one who shares, by common inheritance, exactly one HLA haplotype with the recipient, and includes the biologic parents, biologic children and full or half siblings. There is strong body of evidence supporting the use of haplo-SCT in patient who lack a matched sibling or unrelated donor with high rates of successful engraftment, effective Graft Versus Host Disease (GVHD) control and favorable outcomes comparative to those seen using other allograft sources, including HLA-matched sibling SCT. Furthermore, it provides a cost-efficient donor option in a timely manner especially for patients who need to proceed quickly to transplant due to concern of disease relapse/progression.
Detailed Description
An open label, single-arm, single-center study to evaluate the safety, efficacy and feasibility of haplo-SCT as an alternative donor source for patients who lack a matched sibling/unrelated donor options. The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD. GVHD Prevention Treatment: Cyclophosphamide will be administered IV on Day 3 and Day 5 post transplant. Tacrolimus will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant. Mycophenolate mofetil will be administered IV until patient can take it by mouth starting on Day 1 post transplant until 28 days.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Non-hodgkin Lymphoma, Chronic Lymphocytic Leukemia
Keywords
NHL, Non-Hodgkin Lymphoma, Haploidentical, Bone Marrow Transplant, Stem Cell Transplant, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, GVHD

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
All patients will receive Haploidentical
Arm Type
Experimental
Arm Description
The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD. All patients will receive a Haplo-identical stem cell transplantation. GVHD Prevention Treatment: Cyclophosphamide 50mg/kg will be administered IV on Day 3 and Day 5 post transplant. Tacrolimus 0.03 mg/kg daily will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant. Mycophenolate mofetil 15mg/kg will be administered twice a day IV until patient can take it by mouth starting on Day 1 post transplant until 28 days.
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Other Intervention Name(s)
Cytoxan
Intervention Description
IV medication given for prevention of graft versus host disease.
Intervention Type
Drug
Intervention Name(s)
Tacrolimus
Other Intervention Name(s)
Prograf
Intervention Description
IV medication given for prevention of graft versus host disease.
Intervention Type
Drug
Intervention Name(s)
Mycophenolate mofetil
Other Intervention Name(s)
Cellcept
Intervention Description
IV medication given for prevention of graft versus host disease.
Intervention Type
Other
Intervention Name(s)
Haploidentical Stem Cell Transplantation
Other Intervention Name(s)
Haploidentical hematopoietic stem cell transplantation
Intervention Description
A stem cell transplant that involves matching a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related donor.
Primary Outcome Measure Information:
Title
Chimerism
Description
Blood test that measures amount of donor's cells
Time Frame
100 days
Secondary Outcome Measure Information:
Title
Neutrophil engraftment
Description
Blood test that measures the white cell count
Time Frame
Day 28
Title
Platelet engraftment
Description
Blood test that measures the platelet count
Time Frame
Day 60
Title
Grade 3 to 4 acute graft-verus-host disease (GVHD)
Description
National Institutes of Health Acute Graft-Versus-Host Disease Grading and Form
Time Frame
100 days
Title
Frequency and severity of chronic GVHD
Description
National Institutes of Health Chronic Graft-Versus-Host Disease Grading and Form
Time Frame
1 year
Title
Disease status with blast counts (immature blood cell count) above 5%
Description
Blood work and/or bone marrow biopsy will be used
Time Frame
3 years
Title
Survival status by patient contact
Description
Contact with patient by phone or doctor's visit
Time Frame
3 years
Title
Immune reconstitution
Description
Blood work will be used to evaluate recovery of T and B cell count subset that assess cells which make antibodies to fight infections
Time Frame
3 years
Title
Grade 3 through 5 Adverse Events
Description
Toxicities that are possibly, probably, and definitely related to study treatment according to NCI CTCAE Version 4
Time Frame
2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Ages 16 years old and up Performance Status 70 percent or above Patients should have the following diseases: Acute myelogenous leukemia (AML) Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL) Transfusion dependent myelodysplastic syndrome (MDS) Non-Hodgkin's Lymphoma (NHL) Chronic lymphocytic leukemia (CLL) Pulmonary function as measured by forced expiratory volume at one second (FEV1) and/or corrected diffusing capacity of lung for carbon monoxide (DLCO) at 60 percent of predicted or above Left ventricular ejection fraction at 45 percent or above If the donor-specific HLA antibodies (DSA) are positive, the patient must undergo a desensitization protocol resulting in undetectable DSA prior to day of transplant Exclusion Criteria: Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea) Uncontrolled bacterial, fungal or viral infections at time of study enrollment Positive for HIV, human T-cell leukemia virus (HTLV-1) and/or Hepatitis C Subjects with signs/symptoms of active central nervous system (CNS) disease
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Patrick A Hagen, MD
Phone
708-327-3156
Email
patrick.hagen@lumc.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Mary Lee, RN
Phone
708-327-2241
Email
mlee@luc.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Zeina Al-Mansour, MD
Organizational Affiliation
Cardinal Bernardin Cancer Center, Loyola University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Loyola University Medical Center
City
Maywood
State/Province
Illinois
ZIP/Postal Code
60153
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zeina Al-Mansour, MD
Phone
708-327-2336
Email
Zeina.Al-Mansour@lumc.edu
First Name & Middle Initial & Last Name & Degree
Mary Lee, RN
Phone
708-327-2241
Email
mlee@luc.edu
First Name & Middle Initial & Last Name & Degree
Patrick Stiff, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide

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