Clinical Trials /

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

NCT01351545

Description:

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.

Recruiting Status:

Recruiting

URL:

https://clinicaltrials.gov/show/NCT01351545

Trial Eligibility

Document

Title

  • Brief Title: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
  • 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

Clinical Trial IDs

  • ORG STUDY ID: 10-CBA
  • NCT ID: NCT01351545

Conditions

  • 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

Interventions

DrugSynonymsArms
A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)Unlicensed CBU

Purpose

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.

Detailed Description

      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

Trial Arms

NameTypeDescriptionInterventions
Unlicensed CBUThe cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.
  • A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)

Eligibility Criteria

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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
Maximum Eligible Age:N/A
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Neutrophil recovery of ≥500/mm3 after cord blood transplantation
Time Frame:Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 60 days post-transplant.
Safety Issue:
Description:The primary objective of this access and distribution protocol 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 FDA licensed.

Details

Phase:
Primary Purpose:Observational
Overall Status:Recruiting
Lead Sponsor:Center for International Blood and Marrow Transplant Research

Last Updated

July 6, 2021