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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up one year

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Event free survival

Stable engraftment

Secondary outcome measures

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.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor

708 Previous Clinical Trials

8,582,760 Total Patients Enrolled

University of California, San FranciscoOTHER

2,506 Previous Clinical Trials

15,238,495 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are participants still being accepted for this research project?

"That is correct, the listing on clinicaltrials.gov does show that this study has an active recruitment status. The trial was originally posted on December 1st, 2016 and updated November 8th, 2022. They are looking for 50 individuals to participate at a single location."

Answered by AI

How many people are total in this testing program?

"Yes, this is an active recruitment according to the information provided on clinicaltrials.gov. The trial was first posted December 1st, 2016 and has since been updated November 8th, 2022. They are recruiting for 50 patients at a single location."

Answered by AI

Has this medication been federally approved in the United States?

"The safety of this proposed treatment was given a score of 2. This is due to the fact that, while there is some evidence supporting its safety, there is no data suggesting that it is an effective intervention."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells for Patients With Primary Immunodeficiencies

Nancy Bunin 2016. "Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells for Patients With Primary Immunodeficiencies". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02990819.

All > Primary Immunodeficiency