TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

Part of paid clinical trials in St. Petersburg, Florida.

Sponsor
Johns Hopkins All Children's Hospital
Study ID
NCT04414046
Phase
PHASE2
Status
Recruiting
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Conditions

  • Metabolic Disease
  • Primary Immune Deficiency Disorders

Eligibility Criteria

Sex
ALL
Age
N/A - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Haploidentical Hematopoietic Cell Transplantation — BIOLOGICAL
    TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantation

Study Details

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.

Key Dates

Start date
Jul 22, 2020
Status verified
Jun 2025
Primary completion
Jun 30, 2026
Completion
Jun 30, 2026

Study Design

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

Arms

  • Experimental: TCR alpha beta T cell depletion
    The leukapheresis product will undergo TCR alpha beta negative selection following a standardized protocol

Primary Outcome Measure

Incidence of successful donor engraftment [ Time Frame: Day 100 after transplantation ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Johns Hopkins All Children's HospitalSt. PetersburgFlorida33701
Deepak Chellapandian, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Johns Hopkins All Children's Hospital· St. Petersburg, FL

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