Pediatric Autologous Bone Marrow Mononuclear Cells for Severe Traumatic Brain Injury
|Traumatic Brain Injury||Biological: autologous bone marrow mononuclear cells Other: Placebo Infusion||Phase 1 Phase 2|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Phase 2 Multicenter Trial of Pediatric Autologous Bone Marrow Mononuclear Cells (BMMNCs) for Severe Traumatic Brain Injury (TBI)|
- brain white matter and gray matter structural preservation on diffusion tensor magnetic resonance imaging (DTMRI) [ Time Frame: one year post infusion ]DTMRI quantitative indices of both macro and microscopic integrity will be evaluated and compared to DTMRI of immediate post-injury treated and non-treated controls.
- CNS white matter and gray matter preservation in regions of interest and improves functional and neurocognitive deficits in children after TBI [ Time Frame: one year post infusion ]
- Infusional toxicity safety evaluations [ Time Frame: 7 days post-infusion ]Murray Score and liver function tests
|Study Start Date:||August 2013|
|Estimated Study Completion Date:||June 2018|
|Estimated Primary Completion Date:||December 2017 (Final data collection date for primary outcome measure)|
Experimental: autologous bone marrow mononuclear cells
a bone marrow harvest will be performed, followed by a single intravenous infusion of autologous bone marrow mononuclear cells within 48 hours of injury.
Biological: autologous bone marrow mononuclear cells
BMMNC infusion of either 6x10^6 cells/kg or 10x10^6 cells/kg weight.
Other Name: BMMNCs
Placebo Comparator: placebo infusion
a sham harvest will be performed, followed by a single intravenous placebo infusion within 48 hours of injury.
Other: Placebo Infusion
Placebo infusion of 0.9% Sodium Chloride
Other Name: Saline Infusion
Study Design: Multicenter, randomized, blinded, placebo controlled, Bayesian adaptive dose escalation design.
Study Intervention: Single dose administered within 48 hours from time of injury. Controls will undergo a sham harvest and receive similarly labeled/external appearance and volume of 0.9% NaCl. BMMNC's will be harvested and undergo processing under cGMP conditions to obtain 6x10^6 cells/kg or 10x10^6 cells/kg weight. The cellular product/placebo will be infused within 48 hours of injury.
Safety Monitoring & Follow-Up: Subjects will be monitored for infusion related toxicity post-infusion through hospital discharge and follow-up return study visits. Laboratory and imaging studies will be repeated at the 1, 6, and 12-month follow-up visits. A medical safety monitor (MSM) will review blinded SAE reports following post-infusion Day 14 for each subject in real time to ensure good clinical practice and to quickly identify safety concerns. The MSM will remain blinded to the treatment assignment, unless the NINDS appointed DSMB approves unblinding.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01851083
|United States, Arizona|
|Phoenix Children's Hospital I University of Arizona|
|Phoenix, Arizona, United States, 85006|
|United States, Texas|
|The University of Texas Health Science Center at Houston|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Charles S Cox, Jr., M.D.||The University of Texas Health Science Center, Houston|