Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
Paul Szabolcs
Study ID
NCT01852370
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • Chronic Granulomatous Disease (CGD)
  • Common Variable Immune Deficiency (CVID)
  • Hyper IgE Syndromes
  • Hyper IgM Deficiencies
  • Immunodeficiency With Predominant T-cell Defect, Unspecified
  • Mendelian Susceptibility to Mycobacterial Disease
  • Severe Chronic Neutropenia
  • Severe Combined Immunodeficiency (SCID)
  • Wiskott-Aldrich Syndrome

Eligibility Criteria

Sex
ALL
Age
5 Years - 45 Years
Healthy Volunteers
Not accepted

Interventions

  • CD3/CD19 negative allogeneic hematopoietic stem cells — BIOLOGICAL
    Negative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection and given at time no less than 8 weeks post lung transplant.

Study Details

The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged 5-45 years with primary immunodeficiency (PID) and end-stage lung disease.

Key Dates

Start date
Jun 20, 2013
Status verified
Dec 2025
Primary completion
Nov 30, 2026
Completion
Nov 30, 2027

Study Design

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

Arms

  • Experimental: BOLT+BMT
    All patients will receive a double lung transplant followed by a hematopoietic stem cell transplant. The lungs and stem cells are from the same partially HLA-matched cadaveric donor. Prior to transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device.

Primary Outcome Measure

Safety: Death [ Time Frame: Up to 2 years post stem cell transplant ]

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of Pittsburgh of UPMCPittsburghPennsylvania15224-

Find similar trials in Pittsburgh, PA

By research site
Children's Hospital of Pittsburgh of UPMC· Pittsburgh, PA

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