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NCT01852370

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

Enrolling by Invitation
PHASE1Ages 5–45InterventionalTreatment
Paul Szabolcs
~16 participants
Updated 2025-12-15 on ClinicalTrials.gov
What's tested:CD3/CD19 negative allogeneic hematopoietic stem cells

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety: Death
Measured over Up to 2 years post stem cell transplant
+7 more outcomes measured

Conditions

Severe Combined Immunodeficiency (SCID)
Immunodeficiency With Predominant T-cell Defect, Unspecified
Severe Chronic Neutropenia
Chronic Granulomatous Disease (CGD)
Hyper IgE Syndromes
Hyper IgM Deficiencies
Wiskott-Aldrich Syndrome
Mendelian Susceptibility to Mycobacterial Disease
Common Variable Immune Deficiency (CVID)

Explore related trials

22q11.2 Deletion SyndromeChronic Granulomatous DiseaseCongenital Combined ImmunodeficiencyEnd-Stage Lung DiseaseGATA2 Associated ImmunodeficiencyImmunodeficiency with Hyper-IgM, Type 3Inborn Error of ImmunityMendelian Susceptibility to Mycobacterial DiseasesOmenn SyndromeSevere Chronic NeutropeniaSevere Combined ImmunodeficiencySTAT3 DeficiencyWiskott-Aldrich Syndrome

Where it's being run

1 sites across 1 states
Pennsylvania1

Study leadership

  • Paul Szabolcs, MD · PRINCIPAL_INVESTIGATOR · Division of BMT and Cellular Therapy, Children's Hospital of Pittsburgh of UPMC

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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
Wiskott-Aldrich Syndrome
Mendelian Susceptibility to Mycobacterial Disease \[6\]
GATA2 Associated Immunodeficiency NOTE: A genetic diagnosis is recommended, but not required. 4. Patients must have evidence of end-stage lung disease and be candidates for bilateral orthotopic lung transplant as determined by the lung transplant team. 5. GFR ≥ 50 mL/min/1.73 m2. 6. AST, ALT ≤ 4x upper limit of normal, total bilirubin ≤ 2.5 mg/dL, normal INR. 7. Cardiac ejection fraction ≥ 40% or shortening fraction ≥26%. 8. Negative pregnancy test for females \>10 years old or who have reached menarche, unless surgically sterilized. 9. All females of childbearing potential and sexually active males must agree to use a 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. 10. 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.

What this trial measures

  • Safety: DeathUp to 2 years post stem cell transplant

    How many, if any, patients die.

  • Safety: Engraftment syndromeUp to 2 years post stem cell transplant

    How many, if any, patients develop engraftment syndrome.

  • Safety: Engraftment failureUp to 2 years post stem cell transplant

    How many patients, if any, develop engraftment failure.

  • Safety: RituximabUp to 2 years post stem cell transplant

    The number of grade 4 and 5 events potentially related to rituximab.

  • Efficacy: BOS score1 year post stem cell transplant

    Bronchiolitis Obliterans Syndrome (BOS) score for all patients who receive both lungs and stem cell transplants.

  • Efficacy: T-cell chimerism1 year post stem cell transplant

    The number of patients who have ≥ 25% donor T-cell chimerism.

  • Efficacy: Myeloid chimerism1 year post stem cell transplant

    The number of patients with myeloid disorders (e.g. CGD) who attain ≥ 10% myeloid chimerism.

  • Efficacy: B-cell chimerism1 year post stem cell transplant

    The number of patients with B-cell disorders who attain ≥ 10% B-cell chimerism.