Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide

NCT03088709 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: 208941
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Chimerism

  Blood test that measures amount of donor's cells

  100 days

Secondary Outcomes (8)

• Neutrophil engraftment

  Day 28

• Platelet engraftment

  Day 60

• Grade 3 to 4 acute graft-verus-host disease (GVHD)

  100 days

• Frequency and severity of chronic GVHD

  1 year

• Disease status with blast counts (immature blood cell count) above 5%

  3 years

Study Arms (1)

All patients will receive Haploidentical

EXPERIMENTAL

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; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Other: Haploidentical Stem Cell Transplantation

Interventions

Cyclophosphamide DRUG

IV medication given for prevention of graft versus host disease.

Also known as: Cytoxan

All patients will receive Haploidentical

Tacrolimus DRUG

IV medication given for prevention of graft versus host disease.

Also known as: Prograf

All patients will receive Haploidentical

Mycophenolate mofetil DRUG

IV medication given for prevention of graft versus host disease.

Also known as: Cellcept

All patients will receive Haploidentical

Haploidentical Stem Cell Transplantation OTHER

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.

Also known as: Haploidentical hematopoietic stem cell transplantation

All patients will receive Haploidentical

Eligibility Criteria

• Age: 16 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Loyola University: lead

Study Sites (1)

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myelodysplastic Syndromes; Lymphoma, Non-Hodgkin; Leukemia, Lymphocytic, Chronic, B-Cell

Interventions

Cyclophosphamide; Tacrolimus; Mycophenolic Acid

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Bone Marrow Diseases; Lymphoma; Leukemia, B-Cell; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids

Study Officials

• Zeina Al-Mansour, MD

  Cardinal Bernardin Cancer Center, Loyola University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: February 22, 2017
• First Posted: March 23, 2017
• Study Start: January 18, 2017
• Primary Completion: January 31, 2022
• Study Completion: January 31, 2022
• Last Updated: July 10, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)