A Study of Haploidentical Bone Marrow Transplant for Patients With Hematologic Malignancies

A Phase 2 Study of Nonmyeloablative Conditioning With Transplantation of Partially Human Leukocyte Antigen (HLA)-Mismatched Bone Marrow and Post-transplant Cyclophosphamide for Patients With Hematologic Malignancies

The primary objective is to determine overall survival 180 days after transplantation involving HLA-haploidentical stem cell/bone marrow graft, and post-transplant Cy.

Study Overview

• Status: Suspended
• Conditions: Hematologic Malignancies
• Intervention / Treatment: Drug: Cyclophosphamide 50 mg/kg IV Days 3 and 4 post transplant

Detailed Description

Study Design: This is a single center Phase 2 study of myeloablative (MA) and nonmyeloablative (NMA) conditioning, transplantation of partially HLA-mismatched bone marrow or peripheral blood stem cells and post-transplantation cyclophosphamide (Cy) in patients with hematologic malignancies including:

1. Acute lymphoblastic leukemia/lymphoma, acute myelogenous leukemia, and Burkitt's lymphoma in remission.
2. Relapsed lymphoma, including marginal zone B cell lymphoma, follicular lymphoma, and chemotherapy-sensitive large-cell or Hodgkin lymphoma.
3. Myelodysplastic Syndrome (MDS)
4. Blastic plasmacytoid dendritic cell neoplasm

Primary Objective:

The primary objective is to determine overall survival 180 days after transplantation involving Myeloablative and Non myeloablative conditioning, HLA-haploidentical marrow or peripheral blood stem cell grafts, and post-transplant Cyclophosphamide as GVHD prophylaxis.

Secondary Objectives:

Secondary objectives include estimating overall and progression-free survival at 100 days, 180 days, and one year after transplantation, treatment-related mortality, incidence of neutrophil and platelet recovery or engraftment, incidence of graft failure, cumulative incidence of acute and chronic Graft versus Host Disease (GVHD), incidence of infections, and cumulative incidence of relapse/progression. The investigators will also examine the amount of time to transplant (day of unrelated search initiation to day 0).

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • La Jolla, California, United States, 92093
      • UCSD Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Age: Subjects 18-70 years old.
2. Donor must be 18 years of age.
3. HLA typing will be performed at high resolution. A minimum match of 5 out of 10 is required.
4. Patients must have received multi-agent chemotherapy within 3 months of consent date (measured from the start date of chemotherapy).

5. Acute Leukemias.

   • Acute Lymphoblastic Leukemia in high risk 1st complete response (CR1)
   • Acute Myelogenous Leukemia in high risk 1st complete response (CR1)
   • Acute Leukemias in 2nd or subsequent Complete Response (CR)
   • Biphenotypic/Undifferentiated Leukemias in 1st or subsequent Complete Response (CR).
6. Burkitt's lymphoma: second or subsequent Complete Response (CR).
7. Lymphoma
8. Patients with adequate physical function
9. Performance status: Karnofsky score 70-100%.

Exclusion Criteria:

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: cyclophosphamide post BMT; Cyclophosphamide 50 mg/kg IV Days 3 and 4 post transplant	Drug: Cyclophosphamide 50 mg/kg IV Days 3 and 4 post transplant; Cyclophosphamide 50 mg/kg IV Days 3 and 4 post transplant

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The primary endpoint is overall survival at 180 days from the time of transplantation.	180 days after transplantation	Day 180

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Neutrophil Engraftment	incidence of neutrophil engraftment at Day 180	180 days post transplant day 0

Collaborators and Investigators

• Sponsor: University of California, San Diego
• Investigators: Principal Investigator: Grace Ku, MD, UCSD

Study record dates

Study Major Dates

• Study Start: July 1, 2012
• Primary Completion (Anticipated): July 1, 2022
• Study Completion (Anticipated): July 1, 2022

Study Registration Dates

• First Submitted: August 29, 2013
• First Submitted That Met QC Criteria: December 4, 2015
• First Posted (Estimate): December 7, 2015

Study Record Updates

• Last Update Posted (Actual): July 21, 2022
• Last Update Submitted That Met QC Criteria: July 20, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Keywords: hematologic malignancies
• Additional Relevant MeSH Terms: Neoplasms by Site; Hematologic Diseases; Neoplasms; Hematologic Neoplasms; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antirheumatic Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Cyclophosphamide
• Other Study ID Numbers: 120931

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No