Clinical Trials Logo
  1. Home
  2. Severe Chronic Neutropenia
  3. NCT01852370

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases — BOLT+BMT  

Wiskott-Aldrich Syndrome Clinical Trial

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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01852370
Study type Interventional
Source University of Pittsburgh
Contact
Status Enrolling by invitation
Phase Phase 1/Phase 2
Start date June 20, 2013
Completion date November 2026
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT01652092 - Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies N/A
Completed NCT01953016 - Participation in a Research Registry for Immune Disorders
Active, not recruiting NCT02333760 - Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome Phase 1/Phase 2
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT04371939 - Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome Phase 2
Completed NCT01347346 - Gene Therapy for WAS Phase 1/Phase 2
Completed NCT00160355 - Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome Phase 1
Recruiting NCT01821781 - Immune Disorder HSCT Protocol Phase 2
Completed NCT01289847 - A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency Phase 4
Completed NCT01347242 - Gene Therapy for Wiskott-Aldrich Syndrome (WAS) Phase 1/Phase 2
Terminated NCT00006054 - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies N/A
Enrolling by invitation NCT03198195 - Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome N/A
Recruiting NCT03019809 - A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients Phase 2
Terminated NCT00909363 - Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients Phase 2
Completed NCT03513328 - Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Phase 1/Phase 2
Active, not recruiting NCT01410825 - Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome Phase 1/Phase 2
Active, not recruiting NCT00004341 - Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders N/A
Completed NCT03399461 - Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
Active, not recruiting NCT03837483 - A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome Phase 3