Mesenchymal Stem Cell Therapy for Lung Rejection
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02181712|
Recruitment Status : Completed
First Posted : July 4, 2014
Last Update Posted : August 11, 2017
|Condition or disease||Intervention/treatment||Phase|
|Lung Transplant Reject Bronchiolitis Obliterans||Biological: Mesenchymal stem cell||Phase 1|
The overall clinical development strategy of this project is to conduct this Phase I dose escalation study entitled "A pilot Study to Evaluate the Safety and Feasibility of Mesenchymal Stem Cells to Induce Remission in Lung Transplant Patients Experiencing Treatment-Refractory Moderate Lung Rejection". This study will be performed under this current IND application and will be limited to the proposed 9 subjects (3 subjects in each of dose escalating subject cohorts).
In this application we are proposing to evaluate safety and feasibility of allogeneic, bone marrow (BM) derived mesenchymal stem cells (MSC) to induce remission of moderate treatment refractory bronchiolitis obliterans (BO/BOS). Lung transplant patients with treatment refractory BO/BOS (grade 3) who do not qualify for a second transplant have near 100% mortality or extreme disability. MSCs are multi-potent cells that have the capacity to induce tissue repair and modulate immune response. They have been used successfully in preclinical and clinical studies to treat graft versus host disease (GVHD) and inflammatory bowel disease. Patients that progressed to moderate BO despite adequate medical treatments and who do not qualify for a second transplant will receive intravenous treatments of allogeneic MSC. The safety measure will include tolerance of MSC infusion and absence of significant cardiopulmonary compromise. The feasibility assessment will include ease of recruitment, practical issue of transporting, preparing and infusing the MSCs.
The clinical assessment from this initial safety study is intended to demonstrate safety of human MSC in subjects with BO and to evaluate the cell dose that demonstrates signs of efficacy. Pending favorable safety results and pharmacokinetic and pharamcodynamic data obtained in the current proposed study we will likely conduct additional human studies with a more directed focus on the evaluation of efficacy while collecting additional safety information.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study to Evaluate the Safety and Feasibility of Mesenchymal Stem Cells to Induce Remission in Lung Transplant Patients Experiencing Treatment-refractory Moderate Lung Rejection|
|Study Start Date :||July 2014|
|Primary Completion Date :||September 2016|
|Study Completion Date :||December 2016|
Experimental: Mesenchymal stem cells
2- 4 million MSC per Kg will be infused intravenously. Product will be infused at the rate of 2 - 3 ml/minute during the first 15 minutes and may be adjusted up to 5ml/minutes if tolerable. It is anticipated that the infusion will be completed within approximately 3 hours.
|Biological: Mesenchymal stem cell|
- Number of Participants with serious and non-serious adverse events [ Time Frame: Up to 2 weeks ]Patients will be assessed for their capacity to tolerate IV infusion of MSC without acute clinical or physiological deterioration.
- Changes in pulmonary function tests [ Time Frame: Up to 2 weeks ]Vital signs, pulmonary function tests (FEV1 and FCV) and Borg Dyspnea Index will be evaluated. Chest Radiograph, CBC and serum chemistry will be performed.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02181712
|United States, Florida|
|Mayo Clinic in Florida|
|Jacksonville, Florida, United States, 32224|
|Principal Investigator:||Cesar Keller, MD||Mayo Clinic|
|Principal Investigator:||Abba C Zubair, MD., PhD||Mayo Clinic|