Clinical Trials /

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

NCT01200017

Description:

This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor) is available to donate. In this procedure, some of the blood forming cells (the stem cells) are collected from the blood of a partially human leukocyte antigen (HLA) matched (haploidentical) donor and are transplanted into the patient (the recipient) after administration of a "conditioning regimen". A conditioning regimen consists of chemotherapy and sometimes radiation to the entire body (total body irradiation, or TBI), which is meant to destroy the cancer cells and suppress the recipient's immune system to allow the transplanted cells to take (grow). A major problem after a transplant from an alternative donor is increased risk of Graft-versus-Host Disease (GVHD), which occurs when donor T cells (white blood cells that are involved with the body's immune response) attack other tissues or organs like the skin, liver and intestines of the transplant recipient. In this study, stem cells that are obtained from a partially-matched donor will be highly purified using the investigational CliniMACS® stem cell selection device in an effort to achieve specific T cell target values. The primary aim of the study is to help improve overall survival with haploidentical stem cell transplant in a high risk patient population by limiting the complication of GVHD.

Related Conditions:
  • Chronic Myeloid Leukemia
Recruiting Status:

Available

Trial Eligibility

Document

Expanded Access Protocol (EAP) Using the CliniMACS Device for Pediatric Haplocompatible Donor <span class="go-doc-concept go-doc-intervention">Stem Cell Transplant</span>

Title

  • Brief Title: Expanded Access Protocol (EAP) Using the CliniMACS Device for Pediatric Haplocompatible Donor Stem Cell Transplant
  • Official Title: An Expanded Access Study of the Feasibility of Using the CliniMACS Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients
  • Clinical Trial IDs

    NCT ID: NCT01200017

    ORG ID: UCSF-10082

    Trial Conditions

    Lymphomas

    White Blood Cell Abnormalities

    Red Blood Cell Abnormalities

    Acute Myeloid Leukemia

    Bone Marrow Failure

    Myelodysplastic Syndrome

    Immune Deficiency

    Osteopetrosis

    Chronic Myeloid Leukemia

    Cytopenias

    Acute Lymphoblastic Leukemia

    Hemoglobinopathy

    Trial Interventions

    Drug Synonyms Arms

    Trial Purpose

    This protocol provides expanded access to bone marrow transplants for children who lack a
    histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor
    (unrelated donor or half-matched related donor) is available to donate. In this procedure,
    some of the blood forming cells (the stem cells) are collected from the blood of a partially
    human leukocyte antigen (HLA) matched (haploidentical) donor and are transplanted into the
    patient (the recipient) after administration of a "conditioning regimen". A conditioning
    regimen consists of chemotherapy and sometimes radiation to the entire body (total body
    irradiation, or TBI), which is meant to destroy the cancer cells and suppress the
    recipient's immune system to allow the transplanted cells to take (grow). A major problem
    after a transplant from an alternative donor is increased risk of Graft-versus-Host Disease
    (GVHD), which occurs when donor T cells (white blood cells that are involved with the body's
    immune response) attack other tissues or organs like the skin, liver and intestines of the
    transplant recipient. In this study, stem cells that are obtained from a partially-matched
    donor will be highly purified using the investigational CliniMACS stem cell selection
    device in an effort to achieve specific T cell target values. The primary aim of the study
    is to help improve overall survival with haploidentical stem cell transplant in a high risk
    patient population by limiting the complication of GVHD.

    Detailed Description

    Trial Arms

    Name Type Description Interventions

    Eligibility Criteria

    Inclusion Criteria:

    - Age 0 (newborn) - 30 years

    - Patient must have a malignant or non-malignant disease that can benefit from
    alternative stem cell transplantation according to standard practice guidelines.

    - Patients with lymphoma or acute leukemia (except acute myeloid leukemia, AML) must be
    in remission at the time of transplant.

    - Patients must lack a healthy human leukocyte antigen (HLA)-identical related donor.

    - Recipient or authorized guardian must sign informed consent for this study.

    - If recipient is female and of child-bearing age, negative pregnancy test.

    - Patient must have a healthy, willing mismatched related or an unrelated donor who is:

    - Able to receive granulocyte colony-stimulating factor (G-CSF) and undergo
    apheresis either through placement of catheters in antecubital veins or a
    temporary central venous catheter,

    - For Related donor: sibling, half-sibling, parent, cousin, aunt, uncle or
    grandparent will all be considered eligible.

    - For Related donor: HLA antigen genotypic match 4/8 and 7/8
    (haplocompatible).

    - For unrelated donor: 6/8 or 7/8 HLA antigen match (if two mismatches, they must
    be at different loci).

    - Complete medical history, physical and screening for infectious diseases that
    are acceptable for donation.

    - If donor is female and of child-bearing age, negative pregnancy test.

    - Absence of anti-HLA antibodies in recipient directed against donor antigens.

    - Donor must be willing to sign informed consent for this study. If donor is < 18
    years of age, donor must be willing to give assent and parents willing to sign
    informed consent. For unrelated donors: The National Marrow Donor Program (NMDP)
    will obtain informed consent for donor's apheresis product to be used in this
    study.

    - Age 5 years

    Exclusion Criteria:

    - Patient with an anticipated life expectancy of < 1 month

    - Active infectious hepatitis or cytomegalovirus (CMV) disease (organ involvement)

    - Human immunodeficiency virus (HIV) or Human T-lymphotropic virus (HTLV-I/II)
    infection

    - Cardiac ejection fraction < 45%; can be lower if patient is not in clinical cardiac
    failure and a reduced intensity conditioning regimen is used.

    - Creatinine clearance <60 ml/min/1.72 m2; can be lower if a reduced intensity
    conditioning regimen is used.

    - Pulmonary diffusion capacity (corrected for hemoglobin), forced expiratory volume in
    one second (FEV1), or forced vital capacity (FVC) <60% of predicted or oxygen
    saturation (O2 sat) > 94% on room air if unable to perform pulmonary function tests
    (PFTs); can be lower if a reduced intensity conditioning regimen is used.

    - Serum alanine aminotransferase (ALT) > 5 x upper limit of normal (can be up to 10x
    upper limit of normal if a reduced intensity conditioning regimen is used) or
    bilirubin > 2.

    - Performance score (Lansky/Karnofsky) < 50

    - Any condition that compromises compliance with the procedures of this protocol, as
    judged by the principal investigator.

    Minimum Eligible Age: N/A

    Maximum Eligible Age: 30 Years

    Eligible Gender: Both

    Primary Outcome Measures

    Secondary Outcome Measures

    Trial Keywords

    T cell depleted

    Matched unrelated donors

    Haplocompatible donors

    Graft vs Host Disease

    Hemoglobinopathies

    Leukemia

    Leukemia, Lymphoid

    Precursor Cell Lymphoblastic Leukemia-Lymphoma

    Leukemia, Myeloid, Acute

    Leukemia, Myeloid

    Leukemia, Myelogenous, Chronic, BCR-ABL Positive

    Lymphoma

    Myelodysplastic Syndromes

    Preleukemia

    Osteopetrosis

    Pancytopenia

    Immune System Diseases

    Hematologic Diseases

    Genetic Diseases, Inborn

    Neoplasms by Histologic Type

    Neoplasms

    Lymphoproliferative Disorders

    Lymphatic Diseases

    Immunoproliferative Disorders

    Myeloproliferative Disorders

    Bone Marrow Diseases

    Precancerous Conditions

    Osteosclerosis

    Osteochondrodysplasias

    Bone Diseases, Developmental

    Bone Diseases

    Musculoskeletal Diseases

    Severe acquired and congenital cytopenias

    White and red blood cell abnormalities