Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
NCT03047746 · Status: ACTIVE_NOT_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 50
Last updated 2025-07-16
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.
Conditions
- Acquired Aplastic Anemia
- Paroxysmal Nocturnal Hemoglobinuria
- Inherited Bone Marrow Failure Syndromes
Interventions
- DEVICE
-
CliniMACs
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
Sponsors & Collaborators
-
Children's Hospital of Philadelphia
lead OTHER
Principal Investigators
-
Timothy Olson, MD, PhD · Children's Hospital of Philadelphia
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- PARALLEL
Eligibility
- Max Age
- 25 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2017-02-01
- Primary Completion
- 2028-05-31
- Completion
- 2029-05-31
- FDA Device
- Yes
Countries
- United States
Study Locations
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