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ACTIVE NOT RECRUITING

NCT02990819

PHASE2

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

Sponsor: Children's Hospital of Philadelphia

View on ClinicalTrials.gov

Summary

This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with alpha/beta T and CD19+ depleted peripheral stem cell grafts from unrelated or partially matched related donors. There are two conditioning regimens depending upon patient diagnosis and age.

Official title: Phase II Study for Patients With Primary Immunodeficiencies Using and Cd19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells

Key Details

Gender

All

Age Range

Any - 25 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2016-12

Completion Date

2027-12

Last Updated

2025-11-10

Healthy Volunteers

Conditions

Immunodeficiencies Immune Dysregulation Syndromes

Interventions

DEVICE

Apha/beta T and CD19+ cell depletion using CliniMACS device

Stem cells will be processed using the CliniMACS device for alpha/beta and CD19+ T cell depletion. Processing of cells using the CliniMACS will occur in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique.

Inclusion Criteria: 1. Ages 0-25 years at time of enrollment 2. 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. 3. Clinical status * Lansky or Karnofsky performance \>=60 * Organ Function: 1. Serum creatinine \<1.5 x upper limit of normal for age Hepatic: ALT \<=250; AST \<=350 2. Cardiac shortening fraction \>=27% 3. Bilirubin \<2.5x normal (unless elevation due to Gilberts disease). 4. No active untreated infection 4. Signed informed consent 5. No HLA matched related donor available. 6. Females of childbearing potential must have negative pregnancy test. Exclusion Criteria: * Uncontrolled bacterial, viral or fungal infections * HLA matched related or unrelated donor able to donate mobilized peripheral stem cells. * Pregnant Females * Matched related donor available for bone marrow donation Donors Selection Criteria: * Donor selection will comply with 21 CFR 1271 * Unrelated donor matched or up to one antigen mismatch as per National Marrow Donor Program (NMDP). * Haploidentical parent or sibling able to undergo mobilization for peripheral stem cell collection. Maternal donor preferred over paternal donor if both equally haploidentical. * Children's Hospital of Philadelphia (CHOP) Blood and Marrow Transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. * Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis

Locations (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States