Primary ImmunodeficiencyPhase 1/2BOLT-BMTNCT03330795
Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
A Phase 1/2 study testing CD3/CD19 neg allogeneic BMT in people with Primary Immunodeficiency and PID, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 10 to 45 years
- Conditions the study is for
- Primary Immunodeficiency, PID
- What is being tested?
- CD3/CD19 neg allogeneic BMT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2017to 1 Mar 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
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- 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 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; and
- 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.
- Eligibility for Bone Marrow Transplant:
- GFR >50 mL/min/1.73 m^2;
- AST, ALT <4x upper limit of normal, Total bilirubin < 2.5 mg/dL;
- Cardiac ejection fraction ≥40% or shortening fraction of at least 26%;
- Human Immunodeficiency Virus (HIV) negative by serology and PCR;
- Human T-lymphotropic virus (HTLV) serology negative;
- Forced vital capacity (FVC) and Forced expiratory volume (FEV1) ≥ 40% predicted for age and SpO2 of >90% at rest on room air AND with clearance by the lung transplant team;
- Absence of uncontrolled infection as determined by blood cultures and radiographic results of previously affected sites, in particular, pulmonary densities during the past 2 weeks prior to chemotherapy;
- Absence of Acute Cellular Rejection (ACR); and
- Bone marrow processing has been completed, and an appropriate stem cell product is available for administration.
- Note: The decision to proceed with the BMT will be at the discretion of the lung transplant team following clearance by the bone marrow team based on the criteria below. The conditioning for the BMT will begin no less than 8 weeks following the lung transplant.
Who cannot join
- Inability or unwillingness of a participant to give written informed consent or comply with study protocol;
- Subjects who have underlying malignant conditions;
- Subjects who have non-malignant conditions that do not require hematopoietic stem cell transplantation;
- Human Immunodeficiency Virus (HIV) positive by serology or polymerase chain reaction (PCR), human T-lymphotropic virus (HTLV) positive by serology;
- Females who are pregnant or who are lactating;
- Allergy to dimethyl sulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product;
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- Uncontrolled pulmonary infection, as determined by radiographic findings and/or significant clinical deterioration.
- -- 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.;
- Recent recipient of any licensed or investigational live attenuated vaccine(s), within 4 weeks of transplant; or
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose:
- additional risks from participation in the study,
- may interfere with the participant's ability to comply with study requirements,
- or that may impact the quality or interpretation of the data obtained from the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
About this study
Official title: Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA Matched Cadaveric Donors (RTB-003)
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.
Read the detailed description
The primary purpose of this study is to evaluate the safety and efficacy of performing bilateral orthotopic lung transplantation (BOLT) followed by cadaveric, partially HLA-matched CD3+/CD19+-depleted hematopoietic stem cell transplantation (HSCT) from the same donor for participants with primary immunodeficiency diseases (PID) and end-stage lung disease. For many patients with primary immunodeficiencies, HSCT, which we refer to as Bone Marrow Transplant (BMT) is a curative, life-saving therapy, resulting in restoration of function in the immune system. Patients with primary immunodeficiencies often develop pulmonary complications as a result of chronic or recurrent infections, making them ineligible for BMT due to the high risk of mortality and pulmonary complications. Lung transplant prior to BMT would allow for restoration of pulmonary function prior to BMT, allowing PID patients to proceed to BMT , which would be curative for the patient's underlying immunodeficiency. As a secondary aim, after successful engraftment with donor bone marrow, the feasibility of participants tolerating planned withdrawal of immunosuppression and achieving eventual freedom from all immunosuppressive drugs and attaining a tolerant state will be assessed.
This is a single center study in which participants receive a cadaveric, partially Human Leukocyte Antigen (HLA)-matched lung transplant followed by a CD3+/CD19+ depleted bone marrow transplant (BMT) from the same donor. In this study, the investigators will use a ≥ 2/6 HLA-matched T cell depleted bone marrow transplant from a cadaveric organ donor with an identical ABO blood type as the recipient.
Participants will undergo:
- Bilateral orthotopic lung transplant (BOLT) utilizing basiliximab induction or an alternate induction therapy based on their underlying disease. Rituximab may be initiated prior to the lung transplant, with tacrolimus as the ongoing maintenance immunosuppression.
- BMT utilizing CD3+/CD19+-depleted bone marrow with bone marrow conditioning beginning no less than 8 weeks after BOLT.
The duration of participant involvement in the trial is up to 2 years post-BMT.
- Conditions
- Primary Immunodeficiency, PID
- Being tested
- CD3/CD19 neg allogeneic BMT
- Keywords
- end-stage lung disease, Bilateral Orthotopic Lung Transplant (BOLT) candidate, CD3/CD19 negative allogeneic hematopoietic stem cells (HSCT), CD3+/CD19+ depleted Bone Marrow Transplant (BMT), Cadaveric (Deceased) Donor, Unrelated (Deceased) Donor
- Medical subject headings
- Primary Immunodeficiency Diseases, Death
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD3/CD19 neg allogeneic BMTBiological
Also called CD3+/CD19+ depleted HSCT
Negative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection, and cryopreserved for later marrow transplantation. BMT conditioning and transplantation will start at least 8 weeks or more post lung transplant - once the participant is clinically judged suitable to undergo BMT conditioning and transplantion.
| Study group | Role | What they receive |
|---|---|---|
| CD3/CD19 neg allogeneic BMT | Experimental | Participants may receive a double lung transplant followed by a bone marrow (hematopoietic stem cells) transplant, if a partially HLA-matched organ offer is accepted. The lungs and allogeneic hematopoietic stem cells will be from the same partially HLA-matched cadaveric donor. Prior to marrow transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device. Receives: Biological: CD3/CD19 neg allogeneic BMT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Death
How many, if any, participants die during study participation.
Measured: Average of approximately 31 months for those who received an initial transplant
Safety: Engraftment Syndrome
How many, if any, participants develop engraftment syndrome.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Engraftment Failure
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Grade 4 or 5 Events Potentially Attributable to Rituximab
Measured: Average of ~25 months.
BOS at 1 Year Post BOLT
Diagnosis of BOS (Bronchiolitis Obliterans Syndrome) at any time up to 1 year post BOLT. BOS is one of the undesirable complications of lung transplantation.
Measured: throughout the first year post BOLT
Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support
The number of participant(s) who need either supplemental oxygen and/or ventilator support (noninvasive/invasive) will be assessed using the Bronchiolitis Obliterans Syndrome (BOS) Classification Score for pulmonary function (e.g., the Forced Expiratory Volume in 1 Second (FEV1). FEV1 is air volume exhaled in 1 second during spirometry, a lung function test. (Continuing dependence on supplemental oxygen or ventilatory support is an undesirable outcome of lung transplantation).
Measured: at 1 Year Post Lung Transplant (BOLT)
Efficacy: Count of Participants With T-cell Chimerism
The number of participants who have ≥ 25% donor T-cell chimerism.
Measured: 1 Year Post Bone Marrow Transplant (BMT)
Efficacy: Count of Participants With Myeloid Chimerism
The number of participants with myeloid disorders (e.g. Chronic Granulomatous Disease [CGD]) who attain ≥ 10% myeloid chimerism.
Measured: 1 Year Post Bone Marrow Transplant (BMT)
Efficacy: Count of Participants B-cell Chimerism
The number of participants with B-cell disorders who attain ≥ 10% B-cell chimerism.
Measured: 1 Year Post Bone Marrow Transplant (BMT)
Secondary outcomes (12)
Count of Participants Able to Proceed to BMT
The number of participants for which it is feasible to proceed to BMT within 6 months following lung transplant.
Measured: 6 Months Post Lung Transplant (BOLT)
Count of Participants Who Achieve Tolerance
The number of participants who develop tolerance to both the host and pulmonary graft.
Definition of tolerance: A participant's successful withdrawal from systemic immunosuppression for 6 weeks with no increase cGVHD score and stable or improving PFTs.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Long Term Complications of Combined Solid Organ and Bone Marrow Transplant
Summary of long-term complications of combined solid organ and bone marrow transplant (BMT).
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants Who Develop Acute Cellular Rejection and Graft Failure
The number of participants who develop acute cellular rejection and graft failure post BMT.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants Able to Initiate Withdrawal of Immunosuppression
The number of participants who are able to start immunosuppression withdrawal.
Measured: 1 year following BMT
Time to Withdrawal of Immunosuppression
Time from BMT to withdrawal of immunosuppression.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Time to Independence From Treatment Dose Antimicrobial Drug
A measure of pathogen-specific immunity.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Post-BMT Achievement of Normal Lymphocyte Count for T-cell Lymphopenias
For participants with immune deficiencies with T cell lymphopenias, number of participants achieving age adjusted, low limit normal range lymphocyte count by 1-year post-BMT.
Measured: 1 year post BMT
Count of Participants Who Develop Acute Graft-Versus-Host Disease (GVHD)
The number of participants who develop acute graft-versus-host disease (GVHD) following tandem BOLT and BMT.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants With Chronic Graft-Versus-Host Disease (GVHD)
The number of participants who develop chronic graft-versus-host disease (GVHD) following tandem BOLT and BMT.
Measured: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants Who Develop Chronic Lung Allograft Dysfunction or Allograft Failure
A significant development in chronic lung allograft dysfunction (as evidenced by a change in BOS stage) or allograft failure at 1 year and up to 2 year post lung transplant (for lung transplant alone and BOLT-BMT subjects.
Measured: up to 2 years post BOLT
Count of Pre-BMT Conditioning Rituximab Related Adverse Events
The number of Grade 4 or 5 adverse events possibly related to the use of rituximab prior to the start of BMT conditioning.
Measured: Average of approximately 9 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2017
- Study start1 Dec 2017
- Primary completion1 Mar 2024
- Study completion1 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary ImmunodeficiencyStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- University of Pittsburgh, PPD Development, LP
- Responsible party
- Sponsor
- Investigators
- Paul Szabolcs, MD, Study chair, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03330795
- Sponsor's protocol ID
- DAIT BOLT-BMT
- Other IDs
- DAIT RTB-003, U01AI125050, NIAID DAIT CRMS ID#: 32935
- Acronym
- BOLT-BMT
- Status last verified
- Jul 2025
- First submitted
- 30 Oct 2017
- Last updated
- 22 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA Matched Cadaveric Donors (RTB-003)
- ClinicalTrials.gov ID
- NCT03330795
- Acronym / sponsor ID
- BOLT-BMT / DAIT BOLT-BMT
- Conditions
- Primary Immunodeficiency, PID
- Keywords
- end-stage lung disease, Bilateral Orthotopic Lung Transplant (BOLT) candidate, CD3/CD19 negative allogeneic hematopoietic stem cells (HSCT), CD3+/CD19+ depleted Bone Marrow Transplant (BMT), Cadaveric (Deceased) Donor, Unrelated (Deceased) Donor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Collaborators
- University of Pittsburgh, PPD Development, LP
- Study start
- 1 Dec 2017 (actual)
- Primary completion
- 1 Mar 2024 (actual)
- Study completion
- 1 Mar 2024 (actual)
- First posted
- 6 Nov 2017 (actual)
- Results
- Posted 22 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The primary purpose of this study is to evaluate the safety and efficacy of performing bilateral orthotopic lung transplantation (BOLT) followed by cadaveric, partially HLA-matched CD3+/CD19+-depleted hematopoietic stem cell transplantation (HSCT) from the same donor for participants with primary immunodeficiency diseases (PID) and end-stage lung disease. For many patients with primary immunodeficiencies, HSCT, which we refer to as Bone Marrow Transplant (BMT) is a curative, life-saving therapy, resulting in restoration of function in the immune system. Patients with primary immunodeficiencies often develop pulmonary complications as a result of chronic or recurrent infections, making them ineligible for BMT due to the high risk of mortality and pulmonary complications. Lung transplant prior to BMT would allow for restoration of pulmonary function prior to BMT, allowing PID patients to proceed to BMT , which would be curative for the patient's underlying immunodeficiency. As a secondary aim, after successful engraftment with donor bone marrow, the feasibility of participants tolerating planned withdrawal of immunosuppression and achieving eventual freedom from all immunosuppressive drugs and attaining a tolerant state will be assessed.
This is a single center study in which participants receive a cadaveric, partially Human Leukocyte Antigen (HLA)-matched lung transplant followed by a CD3+/CD19+ depleted bone marrow transplant (BMT) from the same donor. In this study, the investigators will use a ≥ 2/6 HLA-matched T cell depleted bone marrow transplant from a cadaveric organ donor with an identical ABO blood type as the recipient.
Participants will undergo:
- Bilateral orthotopic lung transplant (BOLT) utilizing basiliximab induction or an alternate induction therapy based on their underlying disease. Rituximab may be initiated prior to the lung transplant, with tacrolimus as the ongoing maintenance immunosuppression.
- BMT utilizing CD3+/CD19+-depleted bone marrow with bone marrow conditioning beginning no less than 8 weeks after BOLT.
The duration of participant involvement in the trial is up to 2 years post-BMT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD3/CD19 neg allogeneic BMT | Experimental | Biological: CD3/CD19 neg allogeneic BMTParticipants may receive a double lung transplant followed by a bone marrow (hematopoietic stem cells) transplant, if a partially HLA-matched organ offer is accepted. The lungs and allogeneic hematopoietic stem cells will be from the same partially HLA-matched cadaveric donor. Prior to marrow transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device. |
Who may qualify
- Ages
- 10 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (31 listed)
- 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:
- 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;
Key exclusion criteria (14 listed)
- Inability or unwillingness of a participant to give written informed consent or comply with study protocol;
- Subjects who have underlying malignant conditions;
- Subjects who have non-malignant conditions that do not require hematopoietic stem cell transplantation;
- Human Immunodeficiency Virus (HIV) positive by serology or polymerase chain reaction (PCR), human T-lymphotropic virus (HTLV) positive by serology;
- Females who are pregnant or who are lactating;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD3/CD19 neg allogeneic BMTAlso: CD3+/CD19+ depleted HSCTNegative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection, and cryopreserved for later marrow transplantation. BMT conditioning and transplantation will start at least 8 weeks or more post lung transplant - once the participant is clinically judged suitable to undergo BMT conditioning and transplantion. | Biological | CD3/CD19 neg allogeneic BMT |
Primary outcome
Safety: Death
Time frame: Average of approximately 31 months for those who received an initial transplant
How many, if any, participants die during study participation.
Safety: Engraftment Syndrome
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
How many, if any, participants develop engraftment syndrome.
Engraftment Failure
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Grade 4 or 5 Events Potentially Attributable to Rituximab
Time frame: Average of ~25 months.
BOS at 1 Year Post BOLT
Time frame: throughout the first year post BOLT
Diagnosis of BOS (Bronchiolitis Obliterans Syndrome) at any time up to 1 year post BOLT. BOS is one of the undesirable complications of lung transplantation.
Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support
Time frame: at 1 Year Post Lung Transplant (BOLT)
The number of participant(s) who need either supplemental oxygen and/or ventilator support (noninvasive/invasive) will be assessed using the Bronchiolitis Obliterans Syndrome (BOS) Classification Score for pulmonary function (e.g., the Forced Expiratory Volume in 1 Second (FEV1). FEV1 is air volume exhaled in 1 second during spirometry, a lung function test. (Continuing dependence on supplemental oxygen or ventilatory support is an undesirable outcome of lung transplantation).
Efficacy: Count of Participants With T-cell Chimerism
Time frame: 1 Year Post Bone Marrow Transplant (BMT)
The number of participants who have ≥ 25% donor T-cell chimerism.
Efficacy: Count of Participants With Myeloid Chimerism
Time frame: 1 Year Post Bone Marrow Transplant (BMT)
The number of participants with myeloid disorders (e.g. Chronic Granulomatous Disease [CGD]) who attain ≥ 10% myeloid chimerism.
Efficacy: Count of Participants B-cell Chimerism
Time frame: 1 Year Post Bone Marrow Transplant (BMT)
The number of participants with B-cell disorders who attain ≥ 10% B-cell chimerism.
12 secondary outcomes
Count of Participants Able to Proceed to BMT
Time frame: 6 Months Post Lung Transplant (BOLT)
Count of Participants Who Achieve Tolerance
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Long Term Complications of Combined Solid Organ and Bone Marrow Transplant
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants Who Develop Acute Cellular Rejection and Graft Failure
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants Able to Initiate Withdrawal of Immunosuppression
Time frame: 1 year following BMT
Time to Withdrawal of Immunosuppression
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Time to Independence From Treatment Dose Antimicrobial Drug
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Post-BMT Achievement of Normal Lymphocyte Count for T-cell Lymphopenias
Time frame: 1 year post BMT
Count of Participants Who Develop Acute Graft-Versus-Host Disease (GVHD)
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants With Chronic Graft-Versus-Host Disease (GVHD)
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Count of Participants Who Develop Chronic Lung Allograft Dysfunction or Allograft Failure
Time frame: up to 2 years post BOLT
Count of Pre-BMT Conditioning Rituximab Related Adverse Events
Time frame: Average of approximately 9 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul Szabolcs, MD study chair
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CD3/CD19 neg allogeneic BMT in people with Primary Immunodeficiency and PID, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 31 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Death (time frame: Average of approximately 31 months for those who received an initial transplant). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2025.