Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.

Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03088709
Recruitment Status : Recruiting
First Posted : March 23, 2017
Last Update Posted : February 28, 2019
Sponsor:
Information provided by (Responsible Party):
Patrick Hagen, Loyola University

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.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Acute Lymphocytic Leukemia Myelodysplastic Syndrome Non-hodgkin Lymphoma Chronic Lymphocytic Leukemia Drug: Cyclophosphamide Drug: Tacrolimus Drug: Mycophenolate mofetil Other: Haploidentical Stem Cell Transplantation Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
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
Actual Study Start Date : January 18, 2017
Estimated Primary Completion Date : January 31, 2020
Estimated Study Completion Date : January 31, 2021


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 Name: Cytoxan

Drug: Tacrolimus
IV medication given for prevention of graft versus host disease.
Other Name: Prograf

Drug: Mycophenolate mofetil
IV medication given for prevention of graft versus host disease.
Other Name: 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 Name: Haploidentical hematopoietic stem cell transplantation




Primary Outcome Measures :
  1. Chimerism [ Time Frame: 100 days ]
    Blood test that measures amount of donor's cells


Secondary Outcome Measures :
  1. Neutrophil engraftment [ Time Frame: Day 28 ]
    Blood test that measures the white cell count

  2. Platelet engraftment [ Time Frame: Day 60 ]
    Blood test that measures the platelet count

  3. 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

  4. Frequency and severity of chronic GVHD [ Time Frame: 1 year ]
    National Institutes of Health Chronic Graft-Versus-Host Disease Grading and Form

  5. 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

  6. Survival status by patient contact [ Time Frame: 3 years ]
    Contact with patient by phone or doctor's visit

  7. 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

  8. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   16 Years to 90 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03088709


Contacts
Layout table for location contacts
Contact: Zeina Al-Mansour, MD 708-327-2336 Zeina.Al-Mansour@lumc.edu
Contact: Mary Lee, RN 708-327-2241 mlee@luc.edu

Locations
Layout table for location information
United States, Illinois
Loyola University Medical Center Recruiting
Maywood, Illinois, United States, 60153
Contact: Zeina Al-Mansour, MD    708-327-2336    Zeina.Al-Mansour@lumc.edu   
Contact: Mary Lee, RN    708-327-2241    mlee@luc.edu   
Principal Investigator: Patrick Stiff, MD         
Sponsors and Collaborators
Loyola University
Investigators
Layout table for investigator information
Principal Investigator: Zeina Al-Mansour, MD Cardinal Bernardin Cancer Center, Loyola University

Layout table for additonal information
Responsible Party: Patrick Hagen, Assistant Professor, Loyola University
ClinicalTrials.gov Identifier: NCT03088709    
Other Study ID Numbers: 208941
First Posted: March 23, 2017    Key Record Dates
Last Update Posted: February 28, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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
Keywords provided by Patrick Hagen, Loyola University:
NHL
Non-Hodgkin Lymphoma
Haploidentical
Bone Marrow Transplant
Stem Cell Transplant
Acute Myeloid Leukemia
Acute Lymphocytic Leukemia
Myelodysplastic Syndrome
Chronic Lymphocytic Leukemia
GVHD
Additional relevant MeSH terms:
Layout table for MeSH terms
Leukemia
Leukemia, Myeloid, Acute
Preleukemia
Lymphoma, Non-Hodgkin
Leukemia, Lymphoid
Leukemia, Lymphocytic, Chronic, B-Cell
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Myelodysplastic Syndromes
Lymphoma
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Leukemia, Myeloid
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Leukemia, B-Cell
Mycophenolic Acid
Cyclophosphamide
Tacrolimus
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action