Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
United States5 participantsStarted 2017-12-01
Plain-language summary
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched CD3+/CD19+ depleted bone marrow transplant (BMT) is safe and effective for individuals aged 10 through 45 years with the diagnosis of primary immunodeficiency (PID) and end-stage lung disease.
The enrollment goal: 8 participants who receive both BOLT and BMT.
Who can participate
Age range10 Years – 45 Years
SexALL
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Inclusion Criteria:
* Subject and/or parent guardian must be able to understand and provide informed consent;
* Subject fulfills criteria for United Network of Organ Sharing (UNOS) listing;
* Subject must have evidence of an underlying primary immunodeficiency for which Bone Marrow Transplant (BMT) is clinically indicated. Examples of such diseases include, but are not limited to:
* Severe Combined Immunodeficiency (SCID)
* Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome
* Severe Chronic Neutropenia
* Chronic Granulomatous Disease (CGD)
* Hyper Immunoglobulin E (IgE) Syndrome or Job's Syndrome
* CD40 or CD40L deficiency
* Wiskott-Aldrich Syndrome
* Mendelian Susceptibility to Mycobacterial Disease
* GATA2-associated Immunodeficiency.
* Subjects must have evidence of end-stage lung disease and be candidates for bilateral orthotopic lung transplant as determined by the lung transplant team;
* Glomerular filtration rate (GFR) ≥ 50 mL/min/1.73 m\^2;
* Aspartate aminotransferase (AST), Alanine aminotransaminase (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 Food and Drug Administration (FDA) approved method of…
What they're measuring
1
Safety: Death
Timeframe: Average of approximately 31 months for those who received an initial transplant
2
Safety: Engraftment Syndrome
Timeframe: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
3
Engraftment Failure
Timeframe: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
4
Grade 4 or 5 Events Potentially Attributable to Rituximab
Timeframe: Average of ~25 months.
5
BOS at 1 Year Post BOLT
Timeframe: throughout the first year post BOLT
6
Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support
Timeframe: at 1 Year Post Lung Transplant (BOLT)
7
Efficacy: Count of Participants With T-cell Chimerism
Timeframe: 1 Year Post Bone Marrow Transplant (BMT)
8
Efficacy: Count of Participants With Myeloid Chimerism
Trial details
NCT IDNCT03330795
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)