T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
Paul Szabolcs
Study ID
NCT03653338
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
5 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • CD3/CD19 depleted leukocytes — BIOLOGICAL
    Negative selection for CD3+/CD19+ cells will be performed on the CliniMACS® depletion device.
  • CD45RA depleted leukocytes — BIOLOGICAL
    Negative selection for CD45RA will be performed on the CliniMACS® depletion device.
  • Hydroxyurea — DRUG
    Sickle Cell Disease Conditioning
  • Rituximab — DRUG
    Sickle Cell Disease Conditioning
  • Alemtuzumab — DRUG
    Sickle Cell Disease Conditioning
  • Fludarabine — DRUG
    Sickle Cell Disease Conditioning
  • Thiotepa — DRUG
    Sickle Cell Disease Conditioning

Study Details

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.

Key Dates

Start date
Aug 2, 2018
Status verified
Aug 2025
Primary completion
Aug 1, 2026
Completion
Aug 1, 2027

Study Design

Enrollment
5 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Hematopoietic Stem Cell Transplantation
    All patients will receive a CD3+/CD19+ depleted stem cell transplant. In this study, the investigators will use HLA mismatched unrelated or haploidentical related donor peripheral blood stem cells. Prior to transplantation, the marrow (90-95%) will be negatively selected for CD3/CD19 using the ClinicMACs® depletion device. The remaining (5-10%) will undergo CD45+RA+ depletion and be frozen for future use as an immune boost. Subjects will undergo hematopoietic stem cell transplant utilizing CD3+/CD19+ depleted cells following conditioning therapy.

Primary Outcome Measure

Graft rejection [ Time Frame: Day -30 through study completion, an average of 2 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of Pittsburgh of UPMCPittsburghPennsylvania15224
Paul Pszabolcs, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Pittsburgh, PA

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

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