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Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

NCT01852370 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2025-12-15

No results posted yet for this study

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.

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)

Interventions

BIOLOGICAL

CD3/CD19 negative allogeneic hematopoietic stem cells

Negative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection and given at time no less than 8 weeks post lung transplant.

Sponsors & Collaborators

  • Paul Szabolcs

    lead OTHER

Principal Investigators

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-06-20
Primary Completion
2026-11-30
Completion
2027-11-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01852370 on ClinicalTrials.gov