Primary Immunodeficiency 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 (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.
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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04561115 -
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral Immunodeficiency
|
Phase 3 | |
| Recruiting |
NCT06355323 -
Bronchiectasis Prevalence in Patients With Primary Humoral Immunodefiency in Champagne-Ardenne Region, France
|
N/A | |
| Completed |
NCT04566692 -
A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency
|
Phase 4 | |
| Completed |
NCT01465958 -
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
|
Phase 4 | |
| Completed |
NCT01581593 -
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
|
Phase 3 | |
| Completed |
NCT02881437 -
IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQvia
|
Phase 4 | |
| Recruiting |
NCT06092528 -
Investigation of the Effects of Pulmonary Rehabilitation in Children With Primary Immunodeficiency
|
N/A | |
| Active, not recruiting |
NCT03576742 -
Severe Immune Cytopenia Registry Www.Sic-reg.Org
|
||
| Completed |
NCT03907241 -
CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL
|
Phase 3 | |
| Not yet recruiting |
NCT04902807 -
Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation
|
||
| Completed |
NCT02806986 -
Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
|
Phase 3 | |
| Completed |
NCT03339778 -
The Benefit of 5% IVIG for Patients With Primary Immunodeficiency Disorders Who Experience Adverse Events on 10% IVIG Preparations
|
N/A | |
| Enrolling by invitation |
NCT00895271 -
Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders
|
||
| Recruiting |
NCT03206099 -
NIAID Centralized Sequencing Protocol
|
||
| Completed |
NCT02503293 -
A Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL Administered With Two Different Delivery Devices: Injections Using Pump or Rapid Push
|
Phase 4 | |
| Recruiting |
NCT03610802 -
Send-In Sample Collection to Achieve Genetic and Immunologic Characterization of Primary Immunodeficiencies
|
||
| Completed |
NCT03815357 -
What is the Incidence of an Immune Disorder in Children With Invasive Pneumococcal Disease (IPD)?
|
||
| Completed |
NCT02269163 -
Study of ProMetic BioTherapeutics Immune Globulin Intravenous (Human) 10%
|
Phase 3 | |
| Terminated |
NCT03733249 -
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
|
Phase 1/Phase 2 | |
| Completed |
NCT02604810 -
Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency
|
Phase 3 |