A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Assessing new blood cells growth after transplant using cord blood units that do not meet FDA guidelines but meet NMDP guidelines

a study on Hematologic Malignancy Inherited Disorders of Metabolism Inherited Abnormalities of Platelets Histiocytic Disorders Acute Myeloid Leukemia Leukemia Acute Lymphoblastic Leukemia Chronic Myeloid Leukemia Hodgkin's Lymphoma Lymphoma Non-Hodgkin Lymphoma Multiple Myeloma Disorders of the Immune System Autoimmune Disease Anemia Neoplasms Hematologic Neoplasms

Summary

Location
at UCLA UCSD UCSF
Dates
study started
estimated completion
Principal Investigator
by Edward Ball, MD (ucsd)Gary Schiller, MD (ucla)Mark Walters, MD (ucsf)Thomas Martin, MD (ucsf)Sandhya Kharbanda, MD (ucsf)
Headshot of Edward Ball
Edward Ball
Headshot of Mark Walters
Mark Walters
Headshot of Thomas Martin
Thomas Martin
Headshot of Sandhya Kharbanda
Sandhya Kharbanda

Description

Summary

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Official Title

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications

Details

Principal Investigators:

The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers.

Study Design:

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Primary Objective:

The primary objective of this study is to examine the incidence of neutrophil recovery of ≥500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed.

Secondary Objectives:

In patients receiving a non-licensed CBU:

  • Assess incidence of transmission of infection
  • Assess incidence of serious infusion reaction
  • Determine 1 year overall survival after cord blood transplantation
  • Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV
  • Assess cumulative incidence of chronic GVHD
  • Determine platelet engraftment of >20,000 mcL and >50,000 mcL

Keywords

Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia, Lymphoma, Leukemia, Multiple Myeloma, Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myeloid Leukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Hematologic Neoplasms, Leukemia, Myeloid, Acute, Aplastic Anemia, Congenital Abnormalities, Disease, Unlicensed CBU

Eligibility

You can join if…

  • Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment
  • Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment
  • Pediatric and adult patients of any age

You CAN'T join if...

  • Patients who are receiving only licensed CBUs
  • Cord blood transplant recipients at international transplant centers
  • Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
  • Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

Locations

  • University of California San Diego accepting new patients
    La Jolla California 92093 United States
  • UCLA accepting new patients
    Los Angeles California 90095 United States
  • Children's Hospital and Research Center of Oakland accepting new patients
    Oakland California 94609 United States
  • UCSF (adults) accepting new patients
    San Francisco California 94143 United States
  • UCSF (peds) accepting new patients
    San Francisco California 94143 United States
  • Scripps Green Hospital accepting new patients
    La Jolla California 92037 United States
  • Rady Children's Hospital San Diego accepting new patients
    San Diego California 92123 United States
  • Children's Hospital of Orange County (CHOC) accepting new patients
    Orange California 92868 United States
  • Children's Hospital of Los Angeles accepting new patients
    Glendale California 91204 United States
  • Sutter Medical Center accepting new patients
    Sacramento California 95816 United States

Lead Scientists at University of California Health

  • Edward Ball, MD (ucsd)
    Dr. Ball’s research focuses on the development of monoclonal antibody (mAb)-targeted immunotherapy and its translation to the treatment of patients with malignancy. His lab produced mAbs to myeloid-associated antigens expressed on myeloid leukemia cells.
  • Gary Schiller, MD (ucla)
    Professor-in-Residence, Medicine. Authored (or co-authored) 134 research publications
  • Mark Walters, MD (ucsf)
    The over-arching goal of my research career has been to develop and expand curative therapies for hemoglobin disorders in particular and non-malignant hematopoietic disorders more broadly. I have focused on genome editing approaches most recently, which rely upon modification of autologous hematopoietic cells to elicit a curative effect.
  • Thomas Martin, MD (ucsf)
    Professor, Medicine, School of Medicine. Authored (or co-authored) 72 research publications
  • Sandhya Kharbanda, MD (ucsf)
    Professor, Pediatrics, School of Medicine. Authored (or co-authored) 37 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Center for International Blood and Marrow Transplant Research
ID
NCT01351545
Study Type
Observational
Participants
Expecting 99999 study participants
Last Updated