The National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, is looking for individuals to participate in clinical studies. Participating in clinical trials allows you to play an active role in research on the nature and causes of many disorders of the brain and nervous system, and to possibly help physician-scientists develop future treatments. The information below is designed to help you quickly learn about actively recruiting research studies for which you or someone you know may be eligible.

Description:

The purpose of this study is to determine if bone marrow mononuclear (or white blood) cells (BMMNCs) can improve brain structure and neurocognitive or functional outcomes in children with severe traumatic brain injury (TBI). BMMNCs are made up of a mixture of cells, including stem cells. Stem cells are cells that have the potential to develop into some or many different cell types in the body.

Severe pediatric TBI is the leading cause of death and disability in children ages 1 to 14 years old.  Children who survive severe TBI usually have problems with memory and coordination that interfere with school and social interactions.  Currently there are no treatments to repair the brain after a severe TBI or to correct the memory or coordination problems that typically follow severe TBI.

Fifty children, ages 5 to 15 years old, will be enrolled over 5 years in this prospective, randomized study.  In addition to standard TBI management and treatment, each participant will have 1 of 2 procedures:  a bone marrow harvest and infusion of his/her own BMMNC, or a placebo or “sham” harvest and infusion of normal saline. Participants also will undergo a spleen ultrasound, magnetic resonance imaging (MRI), and blood and cerebrospinal fluid collections.

Follow-up will occur at 1-month, 6-month, and 1-year post TBI.  During each follow-up session, participants will undergo physical and neurological exams, laboratory tests, blood collection, a spleen ultrasound, a chest x-ray, and neuropsychological testing.

Knowledge gained from this study may provide scientists with information on how to better treat TBI in children.

Eligibility Criteria:

INCLUSION:

  • Between 5 and 15 years of age on the day of injury
  • Hospital admission Glasgow Coma Score between 3 and 8
  • Initial injury occurring less than 24 hours prior to consent

EXCLUSION:

  • Known history of: previous brain injury, psychiatric disorder, neurologic impairment and/or deficit, seizure disorder requiring anti-convulsant therapy, recently treated infection, renal disease or altered renal function, hepatic disease or altered liver function, cancer, immunosuppression, HIV positive, chemical and/or alcohol (ETOH) dependency, history of child abuse, or premature birth.
  • Initial hospital intracranial pressure (ICP)>40 mm/Hg
  • Hemodynamic instability at the time of consent (24 hours post-injury)
  • Uncorrected coagulopathy at the time of bone marrow harvest
  • Unstable pelvic fractures
  • Pulmonary contusions
  • Greater than American Association for the Surgery of Trauma (AAST) Grade 3 solid or hollow visceral injury of the abdomen and/or pelvis as diagnosed by CT or other imaging
  • Spinal cord injury diagnosed by CT or MRI or by clinical findings
  • Persistent hypoxia defined as SaO2<94% for > 30 minutes occurring at any time from hospital admission to time of consent
  • Participation in a concurrent interventional study
  • Contraindications to MRI
  • Penetrating brain injury

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Outcomes Assessor), Primary Purpose: Treatment

Study Location:

International Locations