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

NCT01200017 | Unknown

• 2 other identifiers: 10082NCI-2020-02478
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Chronic Myeloid Leukemia; Myelodysplastic Syndrome; Lymphomas; Bone Marrow Failure; Hemoglobinopathy; Immune Deficiency; Osteopetrosis; Cytopenias; Leukocyte Disorders; Anemia Due to Intrinsic Red Cell Abnormality

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

Interventions

CD34+ enriched, T Cell Depleted donor stem cell product BIOLOGICAL

stem cell transplant

Also known as: bone marrow transplant

Eligibility Criteria

• Age: 2 Months - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• \>2 months - 30 years
• Patient must have a malignant or non-malignant disease that can benefit from alternative stem cell transplantation according to standard practice guidelines for including patients for transplant as outlined in UCSF Pediatric Bone Marrow Transplant (BMT) Standard Operating Procedure (SOP) #206.04. Examples include acute and chronic leukemias, myelodysplastic syndrome, lymphoma, severe acquired and congenital cytopenias, white and red blood cell abnormalities, inborn errors of metabolism and immunodeficiencies. Patient with Fanconi's Anemia will be eligible regardless of match with donor.
• Patients with acute leukemia (AML excepted) or lymphoma 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) +/- Plerixafor 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Christopher Dvorak: lead

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Myelodysplastic Syndromes; Lymphoma; Bone Marrow Failure Disorders; Hemoglobinopathies; Immunologic Deficiency Syndromes; Osteopetrosis; Cytopenia; Leukocyte Disorders; Graft vs Host Disease; Leukemia; Leukemia, Lymphoid; Leukemia, Myeloid; Preleukemia; 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

Interventions

Bone Marrow Transplantation

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Tissue Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Christopher Dvorak, MD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 9, 2010
• First Posted: September 13, 2010
• Last Updated: May 27, 2021

Record last verified: 2021-05

Locations

US (1)