Clinical Trials in the Spotlight



NINDS Clinical Trials features descriptions of a selected group of actively-recruiting NINDS-sponsored trials organized by neurological disorder.

Acute Ischemic Stroke

Stroke Hyperglycemia Insulin Network Effort (SHINE) Trial

The Stroke Hyperglycemia Insulin Network Effort (SHINE) Trial is a multicenter, randomized, controlled clinical trial of 1400 patients that will include approximately 60 enrolling sites. The study hypotheses are that treatment of hyperglycemic acute ischemic stroke patients with targeted glucose concentration (80mg/dL - 130 mg/dL) will be safe and result in improved 3 month outcome after stroke. Eligible subjects must be within 12 hours of stroke symptom onset and have diabetes and glucose concentrations of over 110 mg/dL on initial evaluation.

ALS

Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation

Background: - Some people have a mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). The mutation causes a small piece of DNA to repeat itself thousands of times. The C9ORF gene mutation mostly occurs in families. In those families, some persons have ALS and others have FTD. Occasionally the C9ORF gene mutation occurs in persons without a family history. Researchers want to understand how this gene causes different diseases. They will study how symptoms caused by the C9ORF gene develop and change over time. They will measure...

Autism

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

The investigators are enrolling 3-12 month old infants with a diagnosis of tuberous sclerosis complex (TSC) for a new study on early markers of autism. The study is looking for early signs for autism in a population (TSC) where autism is common. The goal of this project is to use behavioral testing, MRI and EEG techniques to identify children at risk for developing autism starting at 3 months of age and continuing until 36 months of age. Throughout the study, the investigators will recommend Early Intervention services for any child who shows early signs of autism.

Benzodiazepine Refractory Status Epilepticus

Established Status Epilepticus Treatment Trial

The primary objective is to determine the most effective and/or the least effective treatment of benzodiazepine-refractory status epilepticus (SE) among patients older than 2 years. There are three active treatment arms being compared: fosphenytoin (FOS),levetiracetam (LEV), and valproic acid (VPA). The second objective is comparison of three drugs with respect to secondary outcomes. The final objective is to ensure that the trial is informative for treatment of established SE in children by describing the effectiveness, safety, and rate of adverse reactions of these drugs in children.

Brain and Spinal Tumors

Tumor Related Epilepsy

Background: Some people with brain tumors have seizures related to the tumor. This is called tumor-related epilepsy. Usually brain tumors are treated by removing as much of the brain tumor as possible without causing problems. Researchers think this may improve the outcome for people with brain tumors. It may completely relieve or greatly reduce the number of seizures they have. Objectives: To evaluate people with brain tumors that are associated with seizures and to offer surgical treatment. Also, to study how surgery affects seizures. Eligibility: People age 8 and older who have a brain...

Evaluation and Treatment of Neurosurgical Disorders

This study offers evaluation and treatment of patients with a variety of neurological disorders that may require surgery. The protocol is not designed to test new treatments; rather, patients will receive standard care. The study is designed to: 1) allow NINDS s Surgical Neurology Branch staff to learn more about changes that cause nervous system disorders, 2) train physicians in the evaluation and treatment of these disorders; and 3) establish a pool of patients who may be eligible for other NINDS protocols. (Participants in this protocol will not be required to join another study; the...

Carotid Stenosis

Carotid Revascularization and Medical Management for Asymptomatic Carotid Stenosis Trial

Carotid revascularization for primary prevention of stroke (CREST-2) is two independent multicenter, randomized controlled trials of carotid revascularization and intensive medical management versus medical management alone in patients with asymptomatic high-grade carotid stenosis. One trial will randomize patients in a 1:1 ratio to endarterectomy versus no endarterectomy and another will randomize patients in a 1:1 ratio to carotid stenting with embolic protection versus no stenting. Medical management will be uniform for all randomized treatment groups and will be centrally directed.

Cryptococcal Meningitis

Adjunctive Sertraline for the Treatment of HIV-Associated Cryptococcal Meningitis

This is a phase III trial to determine whether adjunctive sertraline will lead to improved survival 18-week survival. There was an initial phase I/II unmasked dose finding pharmacokinetic study of CSF concentrations in 172 persons conducted from August 2013 to August 2014..

Dementia

Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation

Background: - Some people have a mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). The mutation causes a small piece of DNA to repeat itself thousands of times. The C9ORF gene mutation mostly occurs in families. In those families, some persons have ALS and others have FTD. Occasionally the C9ORF gene mutation occurs in persons without a family history. Researchers want to understand how this gene causes different diseases. They will study how symptoms caused by the C9ORF gene develop and change over time. They will measure...

Duchenne Muscular Dystrophy

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.

Dystonia

Comparison of Electrophysiologic and Ultrasound Guidance for Onabotulinum Toxin A Injections in Focal Upper Extremity Dystonia and Spasticity

Background: - It is hard for people with arm spasticity and focal hand dystonia to control their arm and hand muscles. They are often treated with botulinum toxin (BoNT) injections. Electromyography with electrical stimulation (e-stim) and ultrasound are used to find muscles for BoNT injection. Researchers want to learn which method is faster and more comfortable. Objective: - To compare 2 ways of finding muscles for BoNT injection for the treatment of focal hand dystonia and upper limb spasticity. Eligibility: - Adults 18 and older with focal hand dystonia or arm spasticity who have been...

Deep Brain Stimulation Surgery for Movement Disorders

Background: Deep brain stimulation (DBS) is an approved surgery for certain movement disorders, like Parkinson's disease, that do not respond well to other treatments. DBS uses a battery-powered device called a neurostimulator (like a pacemaker) that is placed under the skin in the chest. It is used to stimulate the areas of the brain that affect movement. Stimulating these areas helps to block the nerve signals that cause abnormal movements. Researchers also want to record the brain function of people with movement disorders during the surgery. Objectives: To study how DBS surgery affects...

Epilepsy

Surgery as a Treatment for Medically Intractable Epilepsy

Background: Medically intractable epilepsy is the term used to describe epilepsy that cannot be controlled by medication. Many people whose seizures do not respond to medication will respond to surgical treatment, relieving seizures completely or almost completely in one-half to two-thirds of patients who qualify for surgery. The tests and surgery performed as part of this treatment are not experimental, but researchers are interested in training more neurologists and neurosurgeons in epilepsy surgery and care in order to better understand epilepsy and its treatment. Objectives: To use...

PRX-00023 Therapy in Localization-Related Epilepsy

Background: - The brain chemical serotonin helps nerve cells communicate. Previous research suggests that serotonin activity may be lower in brain areas where seizures start, and that increasing activity at the serotonin receptor site on nerve cells may help prevent seizures. Researchers are interested in determining whether the experimental medication PRX-00023, which increases the activity of serotonin receptors, can reduce seizure frequency in people whose seizures are not well-controlled on antiseizure medication. PRX-00023 has not previously been studied in people with epilepsy and...

Tumor Related Epilepsy

Background: Some people with brain tumors have seizures related to the tumor. This is called tumor-related epilepsy. Usually brain tumors are treated by removing as much of the brain tumor as possible without causing problems. Researchers think this may improve the outcome for people with brain tumors. It may completely relieve or greatly reduce the number of seizures they have. Objectives: To evaluate people with brain tumors that are associated with seizures and to offer surgical treatment. Also, to study how surgery affects seizures. Eligibility: People age 8 and older who have a brain...

Focal Dystonia

Comparison of Electrophysiologic and Ultrasound Guidance for Onabotulinum Toxin A Injections in Focal Upper Extremity Dystonia and Spasticity

Background: - It is hard for people with arm spasticity and focal hand dystonia to control their arm and hand muscles. They are often treated with botulinum toxin (BoNT) injections. Electromyography with electrical stimulation (e-stim) and ultrasound are used to find muscles for BoNT injection. Researchers want to learn which method is faster and more comfortable. Objective: - To compare 2 ways of finding muscles for BoNT injection for the treatment of focal hand dystonia and upper limb spasticity. Eligibility: - Adults 18 and older with focal hand dystonia or arm spasticity who have been...

Frontotemporal Dementia

Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation

Background: - Some people have a mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). The mutation causes a small piece of DNA to repeat itself thousands of times. The C9ORF gene mutation mostly occurs in families. In those families, some persons have ALS and others have FTD. Occasionally the C9ORF gene mutation occurs in persons without a family history. Researchers want to understand how this gene causes different diseases. They will study how symptoms caused by the C9ORF gene develop and change over time. They will measure...

Fungal Meningitis

Adjunctive Sertraline for the Treatment of HIV-Associated Cryptococcal Meningitis

This is a phase III trial to determine whether adjunctive sertraline will lead to improved survival 18-week survival. There was an initial phase I/II unmasked dose finding pharmacokinetic study of CSF concentrations in 172 persons conducted from August 2013 to August 2014..

Intracerebral Hemorrhage

Minimally Invasive Surgery Plus Rt-PA for ICH Evacuation Phase III

A phase III, randomized, case-controlled, open-label, 500-subject clinical trial of minimally invasive surgery plus rt-PA in the treatment of intracerebral hemorrhage (ICH).

Intracerebral Hemorrhage Deferoxamine Trial - iDEF Ttrial

The investigators hypothesize that treatment with the iron chelator, Deferoxamine Mesylate, improves the outcome of patients with brain hemorrhage. The purpose of this study is to determine whether treatment with Deferoxamine Mesylate is of sufficient promise to improve outcome before pursuing a larger clinical trial to examine its effectiveness as a treatment for intracerebral hemorrhage.

Ischemic Stroke

Safety Evaluation of 3K3A-APC in Ischemic Stroke

The purpose of this study is to evaluate the safety, pharmacokinetics (PK) and preliminary efficacy of multiple ascending intravenous doses of 3K3A-APC, a Recombinant Variant of Human activated protein C (APC), in in the treatment of acute ischemic stroke following treatment with recombinant tissue plasminogen activator (tPA), mechanical thrombectomy or both.

Muscle Spasticity

Comparison of Electrophysiologic and Ultrasound Guidance for Onabotulinum Toxin A Injections in Focal Upper Extremity Dystonia and Spasticity

Background: - It is hard for people with arm spasticity and focal hand dystonia to control their arm and hand muscles. They are often treated with botulinum toxin (BoNT) injections. Electromyography with electrical stimulation (e-stim) and ultrasound are used to find muscles for BoNT injection. Researchers want to learn which method is faster and more comfortable. Objective: - To compare 2 ways of finding muscles for BoNT injection for the treatment of focal hand dystonia and upper limb spasticity. Eligibility: - Adults 18 and older with focal hand dystonia or arm spasticity who have been...

Musician's Dystonia

Comparison of Electrophysiologic and Ultrasound Guidance for Onabotulinum Toxin A Injections in Focal Upper Extremity Dystonia and Spasticity

Background: - It is hard for people with arm spasticity and focal hand dystonia to control their arm and hand muscles. They are often treated with botulinum toxin (BoNT) injections. Electromyography with electrical stimulation (e-stim) and ultrasound are used to find muscles for BoNT injection. Researchers want to learn which method is faster and more comfortable. Objective: - To compare 2 ways of finding muscles for BoNT injection for the treatment of focal hand dystonia and upper limb spasticity. Eligibility: - Adults 18 and older with focal hand dystonia or arm spasticity who have been...

Myasthenia Gravis

Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy

The purpose of this trial is to determine if thymectomy combined with prednisone therapy is more beneficial in treating non-thymomatous myasthenia gravis than prednisone therapy alone.

Parkinson's Disease

Study of Urate Elevation in Parkinson's Disease, Phase 3

A multicenter, randomized, double-blind, placebo-controlled, phase 3 trial to determine whether oral inosine dosed to moderately elevate serum urate (from ≤5.7 mg/dL to 7.1-8.0 mg/dL) over 2 years slows clinical decline in early PD. Clinical decline will be assessed as change in the primary outcome variable of the Movement Disorders Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS), a composite scale comprising patient- and clinician-reported outcomes.

Deep Brain Stimulation Surgery for Movement Disorders

Background: Deep brain stimulation (DBS) is an approved surgery for certain movement disorders, like Parkinson's disease, that do not respond well to other treatments. DBS uses a battery-powered device called a neurostimulator (like a pacemaker) that is placed under the skin in the chest. It is used to stimulate the areas of the brain that affect movement. Stimulating these areas helps to block the nerve signals that cause abnormal movements. Researchers also want to record the brain function of people with movement disorders during the surgery. Objectives: To study how DBS surgery affects...

Sickle Cell Disease

Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II

The overall goal of this proposal is to conduct a partial double-blind randomized Phase III clinical trial for primary stroke prevention in children with sickle cell anemia (SCA) in sub-Saharan Africa.

Stroke

THrombolysis for Acute Wake-up and Unclear-onset Strokes With Alteplase at 0.6 mg/kg Trial (THAWS)

The purpose of this study is to clarify efficacy and safety of MRI-based intravenous thrombolysis with alteplase for patients with acute wake-up ischemic stroke and those having acute ischemic stroke with unknown time of symptom onset.

The Neurotrophic Effects of Lithium Carbonate Following Stroke: A Feasibility Study

Stroke is the leading cause of adult disability and the third leading cause of death in Canada. Most stroke survivors live with residual impairments that diminish independence and quality of life. This may include vascular cognitive impairment (loss of ability to plan, think and reason) which can lead to dementia and loss of mental and functional independence. The current treatment to reduce stroke induced brain tissue injury is limited to thrombolytics (clot busters), a therapy useful only if given in the first hours following stroke. One major new approach aims to reduce cell death after...

Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II

The overall goal of this proposal is to conduct a partial double-blind randomized Phase III clinical trial for primary stroke prevention in children with sickle cell anemia (SCA) in sub-Saharan Africa.

Traumatic Brain Injury

Pediatric Autologous Bone Marrow Mononuclear Cells for Severe Traumatic Brain Injury

Pediatric severe traumatic brain injury (TBI) is the leading cause of death and disability in children ages 1-14 years old. There are no effective therapies to treat secondary brain injury and the post-injury response of CNS apoptosis and neuroinflammation. This study is a follow-up trial from a previously performed Phase I trial that demonstrated the safety and potential CNS structural preservation effect of intravenous autologous bone marrow mononuclear cells (BMMNC) after severe TBI in children. (Cox, 2011) The study is designed as a prospective, randomized, placebo controlled, blinded...

ADAPT

The ADAPT study will evaluate the effectiveness of different medical therapies on children with severe traumatic brain injury (TBI).

Each year TBI contributes to a substantial number of deaths and cases of permanent disability.   In fact, TBI is a contributing factor to a third of all injury-related deaths in the United States. 

Transforming Research and Clinical Knowledge in Traumatic Brain Injury

The overall goal of Transforming Research and Clinical Knowledge in Traumatic Brain Injury (TRACK-TBI) study is to determine the relationships among the clinical, neuroimaging, cognitive, genetic and proteomic biomarker characteristics for the entire spectrum of TBI from concussion to coma. TRACK-TBI will validate biomarkers and outcome measures for clinical trials, advance diagnostic and prognostic models for TBI and improve clinical trial design.

Tremor

Deep Brain Stimulation Surgery for Movement Disorders

Background: Deep brain stimulation (DBS) is an approved surgery for certain movement disorders, like Parkinson's disease, that do not respond well to other treatments. DBS uses a battery-powered device called a neurostimulator (like a pacemaker) that is placed under the skin in the chest. It is used to stimulate the areas of the brain that affect movement. Stimulating these areas helps to block the nerve signals that cause abnormal movements. Researchers also want to record the brain function of people with movement disorders during the surgery. Objectives: To study how DBS surgery affects...

Tuberous Sclerosis Complex

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

The investigators are enrolling 3-12 month old infants with a diagnosis of tuberous sclerosis complex (TSC) for a new study on early markers of autism. The study is looking for early signs for autism in a population (TSC) where autism is common. The goal of this project is to use behavioral testing, MRI and EEG techniques to identify children at risk for developing autism starting at 3 months of age and continuing until 36 months of age. Throughout the study, the investigators will recommend Early Intervention services for any child who shows early signs of autism.