Neuroscience: Clinical Trials

The NICHD conducts and supports a variety of clinical research related to neuroscience. Select a link below to learn more about these projects.

Featured NICHD Clinical Trials

  • Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
    This is a clinical trial to test the safety, tolerability, feasibility, and pharmacokinetics of 2-hydroxypropyl-beta-cyclodextrin for the treatment of Niemann-Pick disease type C, a lethal, autosomal recessive lysosomal storage disorder that leads to neurodegeneration in early childhood and death in adolescence.
  • Late Hypothermia for Hypoxic-Ischemic Encephalopathy
    This randomized, placebo-controlled trial will test the safety and efficacy of initiating hypothermia 6–24 hours after birth as treatment for hypoxic-ischemic encephalopathy (HIE). This is a rare but life-threatening condition characterized by acute or subacute brain injury due to asphyxia, usually with unknown cause or timing. Up to 80% of those infants who survive stage 3 HIE develop significant long-term neurological disabilities.
  • Optimizing (Longer, Deeper) Cooling for Neonatal Hypoxic-Ischemic Encephalopathy(HIE)
    This trial will test four cooling strategies (two different core temperatures and two durations of cooling) of induced hypothermia for treatment of HIE. Neurodevelopmental outcomes in the four groups will be compared at age 18 to 22 months.
  • Brain-Derived Neurotrophic Factor in Obesity and Brain Function
    This is a cross-sectional study to evaluate serum concentrations of brain-derived neurotrophic factor (BDNF), metabolism, body composition, and neurocognition in Prader-Willi syndrome and MC4R function–altering mutation patients, comparing the results with measurements from control subjects matched for age, sex, race, and BMI. The investigation could lead mechanism-specific therapy for the hyperphagia and obesity associated with Prader-Willi syndrome and MC4R mutations.
  • Botulinum Toxin for Pelvic Pain in Women With Endometriosis
    This study is examining if botulinum toxin injections into the pelvic floor muscles can decrease pain and spasms in women with pelvic pain.
  • Testing and Calibration of Non-Invasive Optical Imaging Technology for Functional Brain Imaging
    This study will examine the capabilities of non-invasive functional near infrared imaging techniques on healthy volunteers and compare the results with the existing outcomes of functional magnetic resonance imaging.
  • Home-Based Automated Therapy of Arm Function After Stroke Via Tele-Rehabilitation
    Constraint-Induced Movement therapy, also known as CI therapy, is an approach for rehabilitating use of the more-affected arm in individuals more than one year after stroke with mild to moderate motor impairment. The purpose of this project is to develop and test a method for automating the delivery of this efficacious treatment in a way that the therapy can be provided in stroke patients’ homes.
  • Methylphenidate (Ritalin) and Memory/Attention in Traumatic Brain Injury (TBI)
    This is a two-by-two design with medication (methylphenidate/placebo) and cognitive therapy (Memory and Attention Training [MAAT] or an Attention control intervention) as possible interventions. Using a randomized, placebo-controlled, double-blind design, 200 individuals with persistent cognitive deficits 6 to 12 months after MTBI will be randomized to receive a six week trial of either (1) MAAT and placebo, (2) MAAT and methylphenidate (0.3 mg/kg BID), (3) attention control intervention and methylphenidate (0.3 mg/kg BID), or (4) attention control intervention and placebo.
  • Interventions for Children With Attention and Reading Disorders (ICARD)
    The objective of this randomized clinical trial is to address unanswered questions about the relative effectiveness of treatments for children with both Attention Deficit Hyperactivity Disorder (ADHD) and significant reading difficulties (RD). The study evaluates attentional and word reading outcomes for students with both conditions when provided with either (a) disorder-specific ADHD treatment (carefully managed stimulant medication + parent training), (b) disorder-specific RD treatment (intensive, individualized reading instruction), or (c) the combination of ADHD and RD treatment.
  • Psychosocial Adjustment of Adolescents With Spina Bifida (CHATS)
    The purpose of this longitudinal study is to evaluate a model of psychological adjustment in young adolescents with spina bifida. The proposed study will be the first to collect observed peer interaction data (target child with close friend) in adolescents with spina bifida. The degree to which neuropsychological limitations experienced by children with spina bifida contribute to their social difficulties and other psychological problems will also be examined. Participants will include a sample of 140 children and young adolescents with spina bifida (with meningomyelocele and shunted hydrocephalus) along with their parents, a close friend, teachers, and health professionals. Questionnaire, interview, neuropsychological testing, and observational methods will be employed during home visits. Findings of this study will provide information on the relevance of family relationships, peer relationships, neuropsychological factors, and biological factors for adjustment in adolescents with spina bifida.

NICHD Clinical Trials

ClinicalTrials.gov Search Results

Information on current NIH-sponsored clinical trials related to neuroscience is available by following the link below or by calling 800-411-1222.

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