Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric Donors

Trial Information
Who is this study for? Child to adult patients with Severe Combined Immunodeficiency
What treatments are being studied? CD3/CD19-Negative Allogeneic Hematopoietic Stem Cell Transplant
Status: Enrolling by invitation
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
Summary

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.

Am I eligible for this trial?
Participation Requirements
Sex:
All
Minimum Age:
5
Maximum Age:
45
Healthy Volunteers:
No

• Subject and/or parent guardian must be able to understand and provide informed consent.

• Male or female, 5 through 45 years old, inclusive, at the time of informed consent.

• Patients must have evidence of an underlying primary immunodeficiency for which BMT is clinically indicated.

• Examples of such diseases include, but are not limited to:

• Severe Combined Immunodeficiency

• Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome

• Severe Chronic Neutropenia

• Chronic Granulomatous Disease

• Hyper IgE Syndrome or Job Syndrome

• CD40 or CD40L deficiency

• Wiskott-Aldrich Syndrome

• Mendelian Susceptibility to Mycobacterial Disease [6]

• GATA2 Associated Immunodeficiency NOTE: A genetic diagnosis is recommended, but not required.

• Patients must have evidence of end-stage lung disease and be candidates for bilateral orthotopic lung transplant as determined by the lung transplant team.

• GFR ≥ 50 mL/min/1.73 m2.

• AST, ALT ≤ 4x upper limit of normal, total bilirubin ≤ 2.5 mg/dL, normal INR.

• Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%.

• Negative pregnancy test for females >10 years old or who have reached menarche, unless surgically sterilized.

• All females of childbearing potential and sexually active males must agree to use a FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defect.

• Subject and/or parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting.

Where is this trial taking place?
United States
Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh
When is this trial taking place?
Start Date: June 20, 2013
Estimated Completion Date: November 2024
How many participants will be in this trial?
Target number of participants: 16
What treatment is being studied in this trial?
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.
Who are the authors of this trial?

This content was sourced from clinicaltrials.gov