Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Timothy Olson
Study ID
NCT04523376
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • CliniMACS — DEVICE
    Peripheral blood stem cells from closely matched unrelated 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

Study Details

This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).

Key Dates

Start date
May 14, 2020
Status verified
Mar 2026
Primary completion
Jun 23, 2025
Completion
Oct 1, 2025

Study Design

Enrollment
8 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Sickle Cell Disease
    Patients with Sickle Cell Disease (SCD) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors.
  • Experimental: Beta Thalassemias Major
    Patients with Beta Thalassemias Major (BTM) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors.

Primary Outcome Measure

Rate of Graft Failure [ Time Frame: Up to 1 year post-transplantation ]

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

Find similar trials in Philadelphia, PA

By condition
Sickle cell disease
By research site
Children's Hospital of Philadelphia· Philadelphia, PA

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