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 range
5 Years – 45 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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%.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. 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.
. Females who are pregnant or who are lactating.
. 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.