Bone Marrow Stromal Cell Infusions for Stem Cell Transplant Complications
|ClinicalTrials.gov Identifier: NCT01633229|
Recruitment Status : Completed
First Posted : July 4, 2012
Last Update Posted : April 13, 2018
- Bone marrow stromal cells (BMSC) from bone marrow biopsies can be used to treat disorders that cause inflammation and immune system diseases. BMSC have been used to treat graft versus host disease (GVHD), a complication that can develop after stem cell transplants. BMSC have also been used to treat other post-transplant complications, like marrow failure or tissue injury.
- The National Institutes of Health (NIH) has developed a procedure for collecting and preserving BMSC from volunteer donors. These donors have passed tests to ensure that their cells are healthy enough to be used for treatment. Researchers want to use the collected cells to treat people with GVHD, marrow failure, or tissue injury following stem cell transplants.
- To test the safety and effectiveness of NIH-collected BMSC to treat complications from stem cell transplants.
- Individuals between 18 and 75 years of age who have complications from stem cell transplants.
- Complications are acute GVHD, poor bone marrow function, or tissue or organ damage.
- Participants will be screened with a physical exam and medical history. They will also have imaging studies and blood tests.
- Participants will provide blood, skin, and bone marrow samples before the BMSC treatment. Additional samples, including tissue samples, will be collected after the start of treatment.
- Participants will have up to three BMSC infusions. There will be a week between each infusion. Participants will be monitored closely during each infusion. Any side effects will be treated.
- Treatment will be monitored with frequent blood tests and physical exams.
- After the end of the infusions, participants will have regular followup visits for up to 2 years.
|Condition or disease||Intervention/treatment||Phase|
|Graft-Versus-Host Disease||Procedure: Bone Marrow Stromal Cell Infusion||Phase 1|
Bone marrow stromal cells (BMSC) derived from bone marrow aspiration/biopsy have important anti-inflammatory and immunosuppressive properties that make them suitable candidates for cellular therapy of inflammatory and degenerative disorders. BMSC suppress lymphocyte immune responsiveness and can hone in to sites of inflammation and promote healing. BMSC were first used in man to successfully treat refractory acute graft versus host disease (aGVHD). A recent multicenter study treating refractory aGVHD reported a 66% response rate and a sustained survival in responding patients. BMSC are currently being evaluated in acute GVHD in several ongoing studies worldwide. BMSC have also been used to treat post-transplant conditions not directly due to GVHD. Responses have been reported in hemorrhagic cystitis, and pneumoperitoneum. BMSC are also under evaluation in the treatment of inflammatory and autoimmune disorders.
The Cell Processing Section of the Department of Transfusion Medicine at the Clinical Center NIH has developed a procedure for collecting, expanding and cryopreserving clinical grade BMSC under an FDA Drug Master File (DMF). Marrow will be aspirated from volunteer donors participating on protocol 10-CC-0053 who have passed the standard screening for blood and marrow donors and BMSC will be expanded in vitro. Since it is not necessary to HLA-match BMSC donors with their recipient for this study, a BMSC repository will be used as the source of BMSC for this study.
This will be the first trial of the safety of the NIH BMSC cellular product in stem cell transplant recipients. In this phase I study, open to adult NIH allogeneic stem cell transplantation (SCT) recipients, we will evaluate the safety of a fixed dose of BMSC infusions (target dose of 2 x 106 BMSCs /kg (+/- 10%)) in patients with acute GVHD, marrow failure or tissue injury following allogeneic SCT. Up to three BMSC infusions will be given every 7 3 days apart. Safety will be monitored continuously with a stopping rule for toxicity based on the treatment-related serious adverse event rate. Organ-specific clinical responses will be measured. Where possible we will also attempt to identify transfused BMSC in sites of damage in biopsy material. It is planned to accrue up to 10 subjects over a 6-month period.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study of Bone Marrow Stromal Cell Infusions to Treat Acute Graft Versus Host Disease, Marrow Failure and Tissue Injury After Allogeneic Stem Cell Transplantation|
|Study Start Date :||November 14, 2011|
|Actual Primary Completion Date :||July 1, 2013|
|Actual Study Completion Date :||March 8, 2018|
- The primary endpoint to this study is to characterize the safety of bone marrow stromal cells (BMSC).
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01633229
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Minocher M Battiwalla, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|