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Trial record 21 of 448 for:    Expanded Access Studies

Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

Expanded access is currently available for this treatment.
Verified May 2017 by Nancy Bunin, Children's Hospital of Philadelphia
Sponsor:
ClinicalTrials.gov Identifier:
NCT03145545
First Posted: May 9, 2017
Last Update Posted: May 9, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Nancy Bunin, Children's Hospital of Philadelphia
  Purpose
The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia Institutional Review Board (IRB) approved protocols that utilize CliniMACs technology for T depletion.

Condition Intervention
Leukemia Bone Marrow Failure Syndrome Immunodeficiencies Device: Apha/beta T and CD19+ cell depletion using CliniMACS device

Study Type: Expanded Access     What is Expanded Access?
Available for Treatment IND/Protocol
Official Title: Expanded Access Protocol Using TCR Alpha/Beta T Cell/CD19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells

Resource links provided by NLM:


Further study details as provided by Nancy Bunin, Children's Hospital of Philadelphia:

Intervention Details:
    Device: Apha/beta T and CD19+ cell depletion using CliniMACS device
    Stem cells will be processed using the CliniMACS device for alpha/beta and CD19+ T cell depletion. Processing of cells using the CliniMACS will occur in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique.
Detailed Description:
Only 25-30% of patients who may benefit from HSCT have a matched related donor. An unrelated cord blood may not be available due to size or matching criteria, or if a reduced intensity regiment is recommended. The risk of severe graft vs. host disease (GVHD) and other complications is higher with unrelated donors, or partially matched related donors. At the Children's Hospital of Philadelphia (CHOP) there is extensive experience using mismatched unrelated donors or partially matched related donors with complete or partial T depletion to reduce the risk of severe GVHD.
  Eligibility

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Ages Eligible for Study:   1 Month and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Criteria

PATIENT AND DONOR ELIGIBILITY

Patients who lack an HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for β T/CD19+ depletion.

Patients with the following transplantable diseases:

Non-malignant diseases:

  • Metabolic storage diseases correctable by HSCT
  • Bone marrow failure syndromes
  • Immunodeficiencies/immune dysregulation syndromes
  • Sickle cell disease with severe central nervous system (CNS) vasculopathy
  • Other hemoglobinopathies requiring HSCT

Malignant diseases:

  • Acute leukemias
  • Chronic leukemias
  • Lymphomas
  • Myelodyplastic syndrome

Organ function criteria:

It is important to note that the conditioning prescribed to the patient will be determined based on the disease and organ status and will be regimens considered standard. Appropriate combinations of chemotherapy, immunotherapy and/or radiation will be determined on an individual basis.

Patient eligibility will be assessed as per our institutional standard operating procedures:

  • Lansky or Karnofsky performance >60
  • Renal function: will be determined based on serum creatinine as per our Institutional SOP
  • Hepatic: Transaminases will be assessed as per current institutional SOP
  • Cardiac shortening fraction >27% as per institutional SOP
  • Bilirubin <2.5x normal (unless elevation due to Gilberts disease) as per Institutional SOP
  • No active untreated infection
  • Signed informed consent
  • No fully HLA matched sibling donor available.
  • Females of childbearing potential must have negative pregnancy test.

Donor Eligibility Patients must have an identified living donor

  • Donor selection will comply with 21 Code of Federal Regulations (CFR) 1271*
  • Unrelated donor that meets the matching criteria of the NMDP: Unrelated donors that may be up to a one antigen mismatch at A, B or DRB1. donor
  • Related donor mismatched at one to five antigens (haploidentical)
  • Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, Hepatitis B (HepB), Hepatitis C (HepC) polymerase chain reaction (PCR) negative.
  • CHOP bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is Foundation for the Accreditation of Cellular Therapy (FACT) accredited.
  • Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis
  • The donors selected for this investigational new drug (IND) will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors; NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases.

Exclusion criteria:

  • Uncontrolled bacterial, viral or fungal infections
  • Fully HLA matched sibling donor
  • Donor unable to donate peripheral stem cells
  • Pregnant Females
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03145545


Contacts
Contact: Barbara McGlynn, BSN, RN 215-590-1303 MCGLYNN@email.chop.edu
Contact: Patricia Hankins, RN 215-590-5168 hankinsp@email.chop.edu

Locations
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contact: Barbara McGlynn, BSN, RN       MCGLYNN@email.chop.edu   
Sponsors and Collaborators
Children's Hospital of Philadelphia
  More Information

Responsible Party: Nancy Bunin, Director, Blood and Marrow Transplant, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT03145545     History of Changes
Other Study ID Numbers: 16-013527
First Submitted: May 5, 2017
First Posted: May 9, 2017
Last Update Posted: May 9, 2017
Last Verified: May 2017

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Pancytopenia
Anemia, Aplastic
Hemoglobinuria, Paroxysmal
Immune System Diseases
Hematologic Diseases
Anemia
Bone Marrow Diseases
Anemia, Hemolytic
Myelodysplastic Syndromes