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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

No Placebo-Only Group

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ one year

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)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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.

0 / 8Eligibility criteria met