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Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases | Clinical Trials | Clareo Health

ENROLLING_BY_INVITATIONPHASE1/PHASE2

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

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

NCT01852370•Paul Szabolcs

View on ClinicalTrials.gov

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.

Detailed Description

This is an original IND for an investigator initiated phase I/II study. The primary purpose of the 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 patients with primary immunodeficiency diseases (PID) and end-stage lung disease. For many patients with primary immunodeficiencies, HSCT 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 HSCT due to the high risk of mortality and pulmonary complications. Lung transplant prior to HSCT would allow for restoration of pulmonary function prior to HSCT, allowing PID patients to proceed to HSCT, which would be curative for the patient's underlying immunodeficiency. As a secondary aim after successful engraftment with donor bone marrow, there is realistic hope for tolerating planned withdrawal of immunosuppression achieving eventual freedom from all immunosuppressive drugs and attaining a tolerant state.

Eligibility

Age

5 - 45 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Subject and/or parent guardian must be able to understand and provide informed consent.
•2. Male or female, 5 through 45 years old, inclusive, at the time of informed consent.
•3. 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
+10 more criteria

Exclusion Criteria

•1. Inability or unwillingness of a participant to give written informed consent or comply with study protocol.
•2. Patients who have underlying malignant conditions.
•3. Patients who have non-malignant conditions not requiring hematopoietic stem cell transplantation.
•4. HIV positive by serology or PCR, HTLV positive by serology.
•5. Females who are pregnant or who are lactating.
•6. Allergy to DMSO or any other ingredient used in the manufacturing of the stem cell product.
•7. 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.
•8. Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, PCR testing, etc.
•9. Recent recipient of any licensed or investigational live attenuated vaccine(s) within 4 weeks of transplant.
•10. 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.
+9 more criteria

Locations (1)

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Key Dates

Start Date

June 20, 2013

Primary Completion Date

November 1, 2026

Completion Date

November 1, 2027

Last Updated

December 15, 2025

Enrollment

ESTIMATED participants

Conditions

Severe Combined Immunodeficiency (SCID)Immunodeficiency With Predominant T-cell Defect, UnspecifiedSevere Chronic NeutropeniaChronic Granulomatous Disease (CGD)Hyper IgE SyndromesHyper IgM DeficienciesWiskott-Aldrich SyndromeMendelian Susceptibility to Mycobacterial DiseaseCommon Variable Immune Deficiency (CVID)

Interventions

CD3/CD19 negative allogeneic hematopoietic stem cells

BIOLOGICAL

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

NCT04049084

Adenosine Deaminase DeficiencySevere Combined Immunodeficiency (SCID)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 15, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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