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TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

NCT04356469 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2025-06-24

No results posted yet for this study

Summary

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.

Conditions

  • Hemoglobinopathy (Disorder)
  • Severe Aplastic Anemia
  • Bone Marrow Failure Syndrome

Interventions

BIOLOGICAL

Haploidentical Hematopoietic Cell Transplantation

TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantation

Sponsors & Collaborators

  • Johns Hopkins All Children's Hospital

    lead OTHER

Principal Investigators

  • Deepak Chellapandian, MD · Johns Hopkins All Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-07-22
Primary Completion
2026-06-30
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT04356469 on ClinicalTrials.gov