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CAR T-cell Therapy

Cell Depletion using CliniMACS for Primary Immunodeficiencies

Phase 2
Recruiting
Led By Nancy Bunin, MD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diseases: Immunodeficiencies for which allogeneic hematopoietic stem cell transplant is indicated, including severe combined immunodeficiencies, immunodeficiency polyendocrinopathy X-linked syndrome (IPEX), X-linked lymphoproliferative disease, chronic granulomatous disease, Wiskott-Aldrich syndrome (WAS), hyperIgM, and other life-threatening immunodeficiencies
Immune dysregulation syndromes, including refractory or recurrent hemophagocytic lymphohistiocytosis, hemophagocytic lymphohistiocytosis (HLH) with genetic mutations, refractory multisystemic Langerhans cell histiocytosis, other macrophage activating syndrome (MAS) refractory to standard therapy
Must not have
Matched related donor available for bone marrow donation
HLA matched related or unrelated donor able to donate mobilized peripheral stem cells
Timeline
Screening 3 weeks
Treatment Varies
Follow Up one year
Awards & highlights

Summary

This trial is testing a new way to do a stem cell transplant using cells from either an unrelated or partially matched related donor. There are two different ways to do the transplant depending on the patient's diagnosis and age.

Who is the study for?
This trial is for young people (ages 0-25) with severe immune system conditions like SCID, IPEX, XLP, CGD, WAS, and others. Participants need to be in a stable condition without untreated infections or available matched donors. They should have normal organ function and females of childbearing age must not be pregnant.
What is being tested?
The study tests if a less intense treatment before transplanting stem cells that had alpha/beta T-cells and CD19+ cells removed can help the body accept these new cells. The process uses CliniMACS device on stem cell grafts from unrelated or half-matched related donors.
What are the potential side effects?
Potential side effects may include reactions to the cell depletion process such as fever or chills. There's also a risk of complications from the transplant like infection due to weakened immunity during engraftment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a severe immune system disorder that requires a stem cell transplant.
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I have a disorder where my immune system attacks my body and standard treatments haven't worked.
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I can do most activities but may need help.
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I am 25 years old or younger.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a family member who can donate bone marrow to me.
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I have a donor match for a stem cell transplant.
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I do not have any untreated serious infections.
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I am currently pregnant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~one year
This trial's timeline: 3 weeks for screening, Varies for treatment, and one year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Event free survival
Stable engraftment
Secondary study objectives
Incidence of graft vs. host disease (GVHD)
Severity of graft vs. host disease (GVHD)

Trial Design

3Treatment groups
Experimental Treatment
Group I: Reduced intensity regimenExperimental Treatment1 Intervention
Conditioning regimen is dependent on patient diagnosis and age. Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete alpha/beta T and CD19+ peripheral stem cells. Standard of care reduced intensity conditioning will include Busulfan, Fludarabine, Thiotepa followed by stem cell infusion.
Group II: Myeloablative regimenExperimental Treatment1 Intervention
Conditioning regimen is dependent on patient diagnosis and age. Patients with chronic granulomatous disease or Wiskott-Aldrich syndrome will receive cyclophosphamide in lieu of thiotepa to ensure engraftment. Myeloablative regimen with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete alpha/beta T and CD19+ peripheral stem cells. Standard of care myeloablative regimen will include Busulfan, Fludarabine, Thiotepa, or Cyclophosphamide followed by stem cell infusion.
Group III: ImmunotherapyExperimental Treatment1 Intervention
Conditioning regimen is dependent on patient diagnosis and age. Severe combined immunodeficiency (SCID) patients will be conditioned with immunotherapy only followed by stem cell transplant using the CliniMACs device to deplete alpha/beta T and CD19+ peripheral stem cells. Immunotherapy regimen will include anti-thymocyte globulin followed by stem cell infusion.

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor
721 Previous Clinical Trials
8,604,267 Total Patients Enrolled
University of California, San FranciscoOTHER
2,551 Previous Clinical Trials
15,257,826 Total Patients Enrolled
Nancy Bunin, MDPrincipal Investigator - Children's Hospital of Philadelphia
Children's Hospital of Philadelphia, Children's Seashore House of the Childrens Hospital of Phila, Children's Surgical Associates of New Jersey Inc
University Of Cincinnati College Of Medicine (Medical School)
University Of Mi Hosps (Residency)
3 Previous Clinical Trials
95 Total Patients Enrolled

Media Library

Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02990819 — Phase 2
Immune Dysregulation Syndrome Research Study Groups: Immunotherapy, Reduced intensity regimen, Myeloablative regimen
Immune Dysregulation Syndrome Clinical Trial 2023: Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells Highlights & Side Effects. Trial Name: NCT02990819 — Phase 2
Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02990819 — Phase 2
~2 spots leftby Feb 2025
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