Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases (NCT01852370) | Clinical Trial Compass
By InvitationPhase 1/2
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
United States16 participantsStarted 2013-06-20
Plain-language 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.
Who can participate
Age range5 Years – 45 Years
SexALL
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Inclusion criteria
✓. 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.
✓. 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.
Exclusion criteria
✕. Inability or unwillingness of a participant to give written informed consent or comply with study protocol.
✕. Patients who have underlying malignant conditions.
✕. Patients who have non-malignant conditions not requiring hematopoietic stem cell transplantation.
✕. HIV positive by serology or PCR, HTLV positive by serology.
What they're measuring
1
Safety: Death
Timeframe: Up to 2 years post stem cell transplant
2
Safety: Engraftment syndrome
Timeframe: Up to 2 years post stem cell transplant
3
Safety: Engraftment failure
Timeframe: Up to 2 years post stem cell transplant
4
Safety: Rituximab
Timeframe: Up to 2 years post stem cell transplant
✕. Allergy to DMSO or any other ingredient used in the manufacturing of the stem cell product.
✕. Uncontrolled pulmonary infection, as determined by radiographic findings and/or significant clinical deterioration. NOTE: Pulmonary colonization with multiple organisms is common, and will not be considered an exclusion criterion.
✕. Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, PCR testing, etc.