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Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

NCT04523376 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2026-04-28

Study results available
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Summary

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).

Conditions

Interventions

DEVICE

CliniMACS

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

Sponsors & Collaborators

  • Timothy Olson

    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

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-05-14
Primary Completion
2025-06-23
Completion
2025-10-01
FDA Device
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04523376 on ClinicalTrials.gov