Unrelated And Partially Matched Related Donor PSCT w/ TCR Depletion for Patients With BMF

  • End date
    Jan 25, 2022
  • participants needed
  • sponsor
    Children's Hospital of Philadelphia
Updated on 25 January 2021
stem cell transplantation
bone marrow failure
shwachman-diamond syndrome
paroxysmal nocturnal hemoglobinuria


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.


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 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.

Condition Paroxysmal nocturnal hemoglobinuria, MYELODYSPLASTIC SYNDROME, Myelodysplastic Syndromes (MDS), Acquired Aplastic Anemia, Inherited Bone Marrow Failure Syndromes, Hemoglobinuria, Paroxysmal
Treatment CliniMACs
Clinical Study IdentifierNCT03047746
SponsorChildren's Hospital of Philadelphia
Last Modified on25 January 2021


Yes No Not Sure

Inclusion Criteria

Acquired and Inherited Bone Marrow Failure Conditions Associated with Trilinear Bone Marrow Failure
Acquired Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria
Fanconi Anemia
Dyskeratosis Congenita and related telomere disorders
Shwachman-Diamond Syndrome
Inherited Bone Marrow Failure Conditions Associated with Predominant Single Lineage Failure
Severe Congenital Neutropenia
Isolated disorders of erythropoiesis
Congenital Thrombocytopenia Syndromes
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 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
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