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Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

Primary Purpose

Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes

Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
CliniMACs
Sponsored by
Children's Hospital of Philadelphia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acquired Aplastic Anemia

Eligibility Criteria

undefined - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Acquired and Inherited Bone Marrow Failure Conditions Associated with Trilinear Bone Marrow Failure

    • Acquired Aplastic Anemia

      1. Must meet criteria for severe or very severe aplastic anemia (AA), defined by:

        i. Bone marrow biopsy demonstrating cellularity of <25% overall or bone marrow biopsy that is overall hypocellular for age by pathology report with reductions in any two hematopoietic lineages (myeloid, erythroid, or megakaryocyte)

        ii. In addition, 2 of the following must be met:

        1. absolute neutrophil count (ANC) < 500/µL (severe) or < 200/µL (very severe). Current ANC or last ANC prior to start of Granulocyte-colony stimulating factor (G-CSF) may be used.
        2. platelets < 30,000/µL or transfusion dependence
        3. absolute reticulocyte count < 40,000/µL

        iii. Negative evaluation for inherited bone marrow failure conditions (see below)

        iv. Must not have accompanying diagnosis of myelodysplastic syndrome

      2. Patients whose best available donor is a ≤7/10 related donor allele level HLA match will only be eligible if they have failed prior immune suppressive therapy. All others may receive study therapy as the initial treatment approach
      3. Patients who have received prior immune suppression therapy will be eligible if they have refractory or relapsed disease, defined by persistent transfusion dependence and/or ANC < 500/µL at least 12 weeks after initiation of immune suppression therapy.
    • Paroxysmal Nocturnal Hemoglobinuria

      1. Patients must have testing (eg. Flow Cytometry demonstrating cells with absent cluster of differentiation 55 (CD55) or cluster of differentiation 59 (CD59) expression demonstrating a PNH clone in greater than 10% of peripheral blood red blood cells and/or granulocytes, along with clinical or laboratory evidence of intravascular hemolysis, such as:

        i. Elevated Lactate dehydrogenase (LDH)

        ii. Low to absent serum haptoglobin

        iii. Hemoglobinuria

        iv. Reticulocytosis

        v. Studies demonstrating aberrant complement activation

      2. Patients who have small paroxysmal nocturnal hemoglobinuria (PNH) clones, no evidence of hemolysis, and meet criteria for severe or very severe AA as defined above, will be classified as acquired AA for treatment stratification.
    • Fanconi Anemia

      1. To be eligible, patients must meet the following criteria:

        i. Must have evidence of BM failure, defined as a bone marrow biopsy demonstrating cellularity of <25% in addition to peripheral blood cytopenias

        ii. Must have chromosomal breakage (stress) testing performed demonstrating increased sensitivity to DNA damage caused by mitomycin C (MMC) or diepoxybutane (DEB)

        iii. Specific testing to define the subtype of Fanconi Anemia through genetic sequencing for causative mutations or complementation group studies is strongly recommended, though not required.

    • Dyskeratosis Congenita and related telomere disorders

      1. To be eligible, patients must meet the following criteria:

        i. Must have evidence of BM failure, defined as a bone marrow biopsy demonstrating cellularity of <25% in addition to peripheral blood cytopenias

        ii. Must have lymphocyte telomere length analysis performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified facility, demonstrating telomeres <1%ile for age

        iii. Specific gene sequencing testing to define the causative genetic mutation is strongly recommended, though not required.

    • Shwachman-Diamond Syndrome

      1. To be eligible, patients must meet the following criteria:

        i. Must have genetic testing confirming a mutation in the Shwachman-Bodian-Diamond syndrome (SBDS) gene, and/or classic clinical features of Shwachman-Diamond Syndrome, including pancreatic insufficiency, musculoskeletal anomalies, and endocrinopathies

        ii. Must have developed trilineage BM failure, including BM cellularity < 25%.

      2. Patients meeting the above criteria for Shwachman-Diamond syndrome will be eligible for conditioning regimen #1 with dosing of Total Body Irradiation (TBI) and cyclophosphamide according to the dyskeratosis congenita regimen
  • Inherited Bone Marrow Failure Conditions Associated with Predominant Single Lineage Failure

    • Severe Congenital Neutropenia

      1. To be eligible, patients must meet the following criteria:

      i. Have a baseline ANC < 500/µL prior to G-CSF therapy

ii. Require chronic G-CSF therapy greater than 3 doses per week in order to maintain an ANC > 1000/µL

iii. Have genetic testing demonstrating mutation(s) in a gene known to cause severe congenital neutropenia and/or have negative testing for autoimmune causes of neutropenia or BM biopsy demonstrating myeloid lineage arrest at neutrophil precursor stage.

iv. History of severe bacterial or fungal infection associated with neutropenia, including, but not limited to, pneumonia, osteomyelitis, mastoiditis, or bacteremia. If no infection history, must otherwise have evidence of toxicity due to chronic G-CSF therapy, including osteopenia, splenomegaly or isolated cytogenetic abnormalities.

  • Isolated disorders of erythropoiesis:

    1. Includes, but not limited to: i. Diamond-Blackfan Anemia (DBA) ii. Congenital Dyserythropoietic Anemia (CDA) iii. Congenital Sideroblastic Anemia (CSA) 2. Eligibility criteria include: i. Chronic red blood cell (RBC) Transfusion Dependence, with minimum frequency of every 8 weeks ii. BM aspirate and biopsy demonstrating selective erythroid hypoplasia or dyserythropoiesis iii. For patients with DBA, must have failed at least one therapeutic trial with corticosteroids iv. Acquired viral and autoimmune causes of hypo-productive anemia have been excluded v. Specific genetic testing attempting to define the causative mutation is recommended but not required

    o Congenital Thrombocytopenia Syndromes

    1. Includes, but is not limited to, patients with Congenital Amegakaryocytic Thrombocytopenia caused by mutations in the MPL gene 2. Eligibility criteria include: i. Platelet transfusion dependence with a minimum transfusion frequency of every 8 weeks ii. Infectious, autoimmune, and other causes of secondary thrombocytopenia have been excluded iii. Genetic sequencing of the MPL gene is required iv. Additional genetic testing for causes of familial thrombocytopenia is recommended but not required

  • Organ function status

    • Renal: Serum creatinine <1.5x upper limit of normal for age
    • Hepatic: Transaminases <= 5x upper limit of normal. Bilirubin <2.0 mg/dL, (unless elevation due to Gilberts disease or known hemolytic anemia).
    • Cardiac: shortening fraction >= 27%
    • Pulmonary: Diffusing Capacity (DLCO) >= 50% predicted in patients old enough to comply with pulmonary function testing (PFTs) or no baseline oxygen requirement for younger patients.
    • Lansky or Karnofsky performance >= 60
  • Infectious disease criteria

    • No active, untreated infections
    • Patients with likely bacterial infections must be receiving appropriate antibacterial therapy and demonstrating therapy response
    • Patients with likely fungal infections must have had at least 2 weeks of appropriate anti-fungal antibiotics and be asymptomatic.
    • Patients with symptoms consistent with active viral infection will be deferred until viral symptoms resolve. Patients with evidence of cytomegalovirus (CMV), Epstein-Barr virus (EBV) or other known viremia must receive appropriate therapy to clear viremia prior to initiating study therapy.
  • Signed consent by parent/guardian or able to give consent if >= 18 years

Exclusion Criteria:

  • Patients who do not meet disease, organ or infectious criteria.
  • Patients with a clinical diagnosis of myelodysplastic syndrome (MDS) defined by combination of bone marrow dysplasia and classic cytogenetic lesion (Monosomy 7, Trisomy 8 eg.), with or without excess blasts.
  • Patients with no suitable closely Human leukocyte antigen (HLA)-matched unrelated or related haploidentical matched donor available. Patients with suitable fully matched related donor are also not eligible.
  • Pregnant females. All females of childbearing potential must have negative pregnancy test.

Donor selection and eligibility:

• Donor selection will comply with 21 Code of Federal Regulations (CFR 1271) of the U.S. Food and Drug Administration's Code of Federal Regulations

Donor testing:

  • Unrelated donor meets National Marrow Donor Program criteria for donation
  • For partially matched related donors, Children's Hospital of Philadelphia (CHOP) bone marrow transplant (BMT) standard procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. The donor collection program accredited.
  • For partially matched related donors, if subject has genetically confirmed iBMF syndrome, related donor must be evaluated for this disorder and testing must be negative
  • Infectious disease testing of donor will be per current Blood and Marrow Transplant Program Standards of Practice as per 21 CFR Part 1271. Donor medical records and history are reviewed to confirm that the donor is free of infectious risk factors and meets donor eligibility criteria as defined by 21 CFR 127.

Sites / Locations

  • Children's Hospital of PhiladelphiaRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Other

Other

Other

Arm Label

TCRalpha/beta Tcell Depletion for BMF with trilineage aplasia

TCRalpha/beta Tcell Depletion for BMF w/o trilineage aplasia

TCRalpha/beta Tcell Depletion for BMF w/ Fanconi Anemia

Arm Description

Patients with acquired or inherited bone marrow failure (iBMF) with trilineage aplasia excluding Fanconi Anemia will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.

Patients with acquired or inherited bone marrow failure (iBMF) without trilineage aplasia will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.

Patients with acquired or inherited bone marrow failure (iBMF) with Fanconi Anemia and related DNA Repair Disorders will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.

Outcomes

Primary Outcome Measures

Rate of graft failure
Time to neutrophil engraftment
Incidence of acute graft vs. host disease (GVHD)
Incidence of chronic graft vs. host disease (GVHD)

Secondary Outcome Measures

Treatment-related Mortality (TRM)
Probability of event-free survival (EFS)
Probability of overall survival (OS)
Reactivation/Infection from CMV, EBV, adenovirus

Full Information

First Posted
February 7, 2017
Last Updated
July 12, 2023
Sponsor
Children's Hospital of Philadelphia
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1. Study Identification

Unique Protocol Identification Number
NCT03047746
Brief Title
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
Official Title
Unrelated And Partially Matched Related Donor Peripheral Blood Stem Cell Transplantation (PSCT) With TCR αβ + T Cell And B Cell Depletion For Patients With Acquired And Inherited Bone Marrow Failure
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 1, 2017 (Actual)
Primary Completion Date
February 2024 (Anticipated)
Study Completion Date
February 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital of Philadelphia

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
Yes
Device Product Not Approved or Cleared by U.S. FDA
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.
Detailed Description
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes. Previously established, disease-specific transplant preparative regimens will be administered based on the specific underlying BMF condition. Mobilized PBSC will be processed using the CliniMACS system for TCR alpha/beta+ T cell depletion plus cluster of differentiation 19+ (CD19+) B cell depletion. The study will determine efficacy of this strategy in terms of engraftment, rates of acute and chronic Graft versus Host Disease (GvHD), and one year overall and event-free survival.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
TCRalpha/beta Tcell Depletion for BMF with trilineage aplasia
Arm Type
Other
Arm Description
Patients with acquired or inherited bone marrow failure (iBMF) with trilineage aplasia excluding Fanconi Anemia will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.
Arm Title
TCRalpha/beta Tcell Depletion for BMF w/o trilineage aplasia
Arm Type
Other
Arm Description
Patients with acquired or inherited bone marrow failure (iBMF) without trilineage aplasia will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.
Arm Title
TCRalpha/beta Tcell Depletion for BMF w/ Fanconi Anemia
Arm Type
Other
Arm Description
Patients with acquired or inherited bone marrow failure (iBMF) with Fanconi Anemia and related DNA Repair Disorders will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.
Intervention Type
Device
Intervention Name(s)
CliniMACs
Intervention Description
Peripheral blood stem cells from closely matched unrelated or partially matched related donors will be processed using the CliniMACS device to remove TCRalpha/beta T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
Primary Outcome Measure Information:
Title
Rate of graft failure
Time Frame
Up to three years post-transplantation
Title
Time to neutrophil engraftment
Time Frame
Up to 60 days post-transplantation
Title
Incidence of acute graft vs. host disease (GVHD)
Time Frame
Up to 100 days post-transplantation
Title
Incidence of chronic graft vs. host disease (GVHD)
Time Frame
Up to three years post-transplantation
Secondary Outcome Measure Information:
Title
Treatment-related Mortality (TRM)
Time Frame
Up to 100 days post-transplantation
Title
Probability of event-free survival (EFS)
Time Frame
Up to 1 year post-transplantation
Title
Probability of overall survival (OS)
Time Frame
Up to 1 year post-transplantation
Title
Reactivation/Infection from CMV, EBV, adenovirus
Time Frame
Up to 1 year post-transplantation

10. Eligibility

Sex
All
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Acquired and Inherited Bone Marrow Failure Conditions Associated with Trilinear Bone Marrow Failure Acquired Aplastic Anemia Must meet criteria for severe or very severe aplastic anemia (AA), defined by: i. Bone marrow biopsy demonstrating cellularity of <25% overall or bone marrow biopsy that is overall hypocellular for age by pathology report with reductions in any two hematopoietic lineages (myeloid, erythroid, or megakaryocyte) ii. In addition, 2 of the following must be met: absolute neutrophil count (ANC) < 500/µL (severe) or < 200/µL (very severe). Current ANC or last ANC prior to start of Granulocyte-colony stimulating factor (G-CSF) may be used. platelets < 30,000/µL or transfusion dependence absolute reticulocyte count < 40,000/µL iii. Negative evaluation for inherited bone marrow failure conditions (see below) iv. Must not have accompanying diagnosis of myelodysplastic syndrome Patients meeting other eligibility criteria may receive study therapy as the initial treatment approach provided an eligible unrelated or mismatched related ("haplo") donor is available Patients who have received prior immune suppression therapy will be eligible if they have refractory or relapsed disease defined as per treating clinician's judgement, at least 12 weeks after initiation of immune suppression therapy. Relapsed patients who previously met hematologic criteria for severe aplastic anemia do not have to meet these hematologic criteria for severe aplastic anemia at time of relapse to be eligible for transplant. Paroxysmal Nocturnal Hemoglobinuria Patients must have testing (eg. Flow Cytometry demonstrating cells with absent cluster of differentiation 55 (CD55) or cluster of differentiation 59 (CD59) expression demonstrating a PNH clone in greater than 10% of peripheral blood red blood cells and/or granulocytes, along with clinical or laboratory evidence of intravascular hemolysis, such as: i. Elevated Lactate dehydrogenase (LDH) ii. Low to absent serum haptoglobin iii. Hemoglobinuria iv. Reticulocytosis v. Studies demonstrating aberrant complement activation Patients who have small paroxysmal nocturnal hemoglobinuria (PNH) clones, no evidence of hemolysis, and meet criteria for severe or very severe AA as defined above, will be classified as acquired AA for treatment stratification. Fanconi Anemia To be eligible, patients must meet the following criteria: i. Must have evidence of BM failure, defined as a bone marrow biopsy demonstrating cellularity of <25% in addition to peripheral blood cytopenias ii. Must have chromosomal breakage (stress) testing performed demonstrating increased sensitivity to DNA damage caused by mitomycin C (MMC) or diepoxybutane (DEB) iii. Specific testing to define the subtype of Fanconi Anemia through genetic sequencing for causative mutations or complementation group studies is strongly recommended, though not required. Dyskeratosis Congenita and related telomere disorders To be eligible, patients must meet the following criteria: i. Must have evidence of BM failure, defined as a bone marrow biopsy demonstrating cellularity of <25% in addition to peripheral blood cytopenias ii. Must have lymphocyte telomere length analysis performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified facility, demonstrating telomeres <1%ile for age iii. Specific gene sequencing testing to define the causative genetic mutation is strongly recommended, though not required. Shwachman-Diamond Syndrome To be eligible, patients must meet the following criteria: i. Must have genetic testing confirming a mutation in the Shwachman-Bodian-Diamond syndrome (SBDS) gene, and/or classic clinical features of Shwachman-Diamond Syndrome, including pancreatic insufficiency, musculoskeletal anomalies, and endocrinopathies ii. Must have developed trilineage BM failure, including BM cellularity < 25%. Patients meeting the above criteria for Shwachman-Diamond syndrome will be eligible for conditioning regimen #1 with dosing of Total Body Irradiation (TBI) and cyclophosphamide according to the dyskeratosis congenita regimen Inherited Bone Marrow Failure Conditions Associated with Predominant Single Lineage Failure Severe Congenital Neutropenia 1. To be eligible, patients must meet the following criteria: i. Have a baseline ANC < 500/µL prior to G-CSF therapy ii. Require chronic G-CSF therapy greater than 3 doses per week in order to maintain an ANC > 1000/µL iii. Have genetic testing demonstrating mutation(s) in a gene known to cause severe congenital neutropenia and/or have negative testing for autoimmune causes of neutropenia or BM biopsy demonstrating myeloid lineage arrest at neutrophil precursor stage. iv. History of severe bacterial or fungal infection associated with neutropenia, including, but not limited to, pneumonia, osteomyelitis, mastoiditis, or bacteremia. If no infection history, must otherwise have evidence of toxicity due to chronic G-CSF therapy, including osteopenia, splenomegaly or isolated cytogenetic abnormalities. Isolated disorders of erythropoiesis: 1. Includes, but not limited to: i. Diamond-Blackfan Anemia (DBA) ii. Congenital Dyserythropoietic Anemia (CDA) iii. Congenital Sideroblastic Anemia (CSA) 2. Eligibility criteria include: i. Chronic red blood cell (RBC) Transfusion Dependence, with minimum frequency of every 8 weeks ii. BM aspirate and biopsy demonstrating selective erythroid hypoplasia or dyserythropoiesis iii. For patients with DBA, must have failed at least one therapeutic trial with corticosteroids iv. Acquired viral and autoimmune causes of hypo-productive anemia have been excluded v. Specific genetic testing attempting to define the causative mutation is recommended but not required o Congenital Thrombocytopenia Syndromes 1. Includes, but is not limited to, patients with Congenital Amegakaryocytic Thrombocytopenia caused by mutations in the MPL gene 2. Eligibility criteria include: i. Platelet transfusion dependence with a minimum transfusion frequency of every 8 weeks ii. Infectious, autoimmune, and other causes of secondary thrombocytopenia have been excluded iii. Genetic sequencing of the MPL gene is required iv. Additional genetic testing for causes of familial thrombocytopenia is recommended but not required Organ function status Renal: Serum creatinine <1.5x upper limit of normal for age Hepatic: Transaminases <500 upper limit of normal. Bilirubin <2.5 mg/dL, (unless elevation due to Gilberts disease or known hemolytic anemia). Cardiac: shortening fraction >= 27% Pulmonary: Diffusing Capacity (DLCO) >= 50% predicted in patients old enough to comply with pulmonary function testing (PFTs) or no baseline oxygen requirement for younger patients. Lansky or Karnofsky performance >= 60 Infectious disease criteria No active, untreated infections Patients with likely bacterial infections must be receiving appropriate antibacterial therapy and demonstrating therapy response Patients with likely fungal infections must have had at least 2 weeks of appropriate anti-fungal antibiotics and be asymptomatic. Patients with symptoms consistent with active viral infection will be deferred until viral symptoms resolve. Patients with evidence of cytomegalovirus (CMV), Epstein-Barr virus (EBV) or other known viremia must receive appropriate therapy to clear viremia prior to initiating study therapy. Signed consent by parent/guardian or able to give consent if >= 18 years Exclusion Criteria: Patients who do not meet disease, organ or infectious criteria. Patients with a clinical diagnosis of myelodysplastic syndrome (MDS) defined by combination of bone marrow dysplasia and classic cytogenetic lesion (Monosomy 7, Trisomy 8 eg.), with or without excess blasts. Patients with no suitable closely Human leukocyte antigen (HLA)-matched unrelated or related haploidentical matched donor available. Patients with suitable fully matched related donor are also not eligible. Pregnant females. All females of childbearing potential must have negative pregnancy test. Donor selection and eligibility: • Donor selection will comply with 21 Code of Federal Regulations (CFR 1271) of the U.S. Food and Drug Administration's Code of Federal Regulations Donor testing: Unrelated donor meets National Marrow Donor Program criteria for donation For partially matched related donors, Children's Hospital of Philadelphia (CHOP) bone marrow transplant (BMT) standard procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. The donor collection program is FACT accredited. For partially matched related donors, if subject has genetically confirmed iBMF syndrome, related donor must be evaluated for this disorder and testing must be negative Infectious disease testing of donor will be per current Blood and Marrow Transplant Program Standards of Practice as per 21 CFR Part 1271. Donor medical records and history are reviewed to confirm that the donor is free of infectious risk factors and meets donor eligibility criteria as defined by 21 CFR 127. Donor matching High resolution HLA typing at HLA-A, -B, -C, DRB1, and DQB1 loci Unrelated donor Donor must be an antigen and allele match at ≥ 8/10 HLA Loci In donor with 2 mismatches, only one mismatch involving HLA-A, -B, or -DRB1 will be allowed Donor and collection center willing to undergo mobilization and apheresis Partially matched related donor Related donor must be ≥ 5/10 but < 10/10 HLA match Donor must be willing to undergo G-CSF mobilization and stem cell apheresis.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Barb McGlynn, RN, BSN
Phone
215-590-1303
Email
MCGLYNN@chop.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Timothy Olson, MD, PhD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbara McGlynn, RN, BSN
Email
MCGLYNN@chop.edu
First Name & Middle Initial & Last Name & Degree
Nancy Bunin, MD
First Name & Middle Initial & Last Name & Degree
Stephan Grupp, MD, PhD
First Name & Middle Initial & Last Name & Degree
Timothy Olson, MD, PhD

12. IPD Sharing Statement

Plan to Share IPD
No

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Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

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