Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide

April 22, 2021 updated by: Hagen, Patrick A, Loyola University

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

40

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Mary Lee, RN
  • Phone Number: 708-327-2241
  • Email: mlee@luc.edu

Study Locations

  • United States
    • Illinois
      • Maywood, Illinois, United States, 60153
        • Recruiting
        • Loyola University Medical Center
        • Contact:
          • Mary Lee, RN
          • Phone Number: 708-327-2241
          • Email: mlee@luc.edu
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 90 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: All patients will receive Haploidentical

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.
Drug: Cyclophosphamide
IV medication given for prevention of graft versus host disease.
Other Names:
  • Cytoxan
Drug: Tacrolimus
IV medication given for prevention of graft versus host disease.
Other Names:
  • Prograf
Drug: Mycophenolate mofetil
IV medication given for prevention of graft versus host disease.
Other Names:
  • Cellcept
Other: Haploidentical Stem Cell Transplantation
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.
Other Names:
  • Haploidentical hematopoietic stem cell transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Chimerism
Time Frame: 100 days
Blood test that measures amount of donor's cells
100 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Neutrophil engraftment
Time Frame: Day 28
Blood test that measures the white cell count
Day 28
Platelet engraftment
Time Frame: Day 60
Blood test that measures the platelet count
Day 60
Grade 3 to 4 acute graft-verus-host disease (GVHD)
Time Frame: 100 days
National Institutes of Health Acute Graft-Versus-Host Disease Grading and Form
100 days
Frequency and severity of chronic GVHD
Time Frame: 1 year
National Institutes of Health Chronic Graft-Versus-Host Disease Grading and Form
1 year
Disease status with blast counts (immature blood cell count) above 5%
Time Frame: 3 years
Blood work and/or bone marrow biopsy will be used
3 years
Survival status by patient contact
Time Frame: 3 years
Contact with patient by phone or doctor's visit
3 years
Immune reconstitution
Time Frame: 3 years
Blood work will be used to evaluate recovery of T and B cell count subset that assess cells which make antibodies to fight infections
3 years
Grade 3 through 5 Adverse Events
Time Frame: 2 years
Toxicities that are possibly, probably, and definitely related to study treatment according to NCI CTCAE Version 4
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Loyola University

Investigators

  • Principal Investigator: Zeina Al-Mansour, MD, Cardinal Bernardin Cancer Center, Loyola University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 18, 2017

Primary Completion (Anticipated)

January 31, 2022

Study Completion (Anticipated)

January 31, 2022

Study Registration Dates

First Submitted

February 22, 2017

First Submitted That Met QC Criteria

March 17, 2017

First Posted (Actual)

March 23, 2017

Study Record Updates

Last Update Posted (Actual)

April 23, 2021

Last Update Submitted That Met QC Criteria

April 22, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 208941

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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