Neurology &
Neurological Sciences
Neurology & Neurological Sciences Clinical Trials

Neurology & Neurological Sciences Clinical Trials


Double-Blind, Randomized, Placebo-Controlled, Phase II Safety and Efficacy Trial of MultiStem® in Adults with Ischemic Stroke
The primary objectives of this stem cell trial for acute stroke are to determine the highest well-tolerated and safest single dose of MultiStem up to a maximum of 1200 million total cells in subjects with ischemic stroke and determine the efficacy of MultiStem on functional outcome in subjects with ischemic stroke.
PI: Neil Schwartz, MD, PhD (NCT01436487)

Biobank For MS And Other Demyelinating Diseases
To establish a large, longitudinal collection of high quality samples and data from subjects
with MS, selected other demyelinating diseases as a shared resource to scientists researching
the causes, sub-types, and biomarkers of MS and related demyelinating diseases.
PI: Jeffrey Dunn, MD (NCT00445367)

A Phase I/II Study of Local Field Irradiation and Temozolomide Followed by Continuous Infusion Plerixafor as an Upfront Therapy for Newly Diagnosed Glioblastoma GBM 
PI: Lawrence Recht, MD (NCT01977677)

ExAblate Transcranial MR Guided Focused Ultrasound for the Treatment of Essential Tremors
A prospective, randomized, double-blind crossover, multi-site, two-arm study will test the efficacy of treatment using the ExAblate Transcranial System and demonstrate safety in medication-refractory tremor in subjects with essential tremor.
PI: Pejman Ghanouni, MD, PhD (NCT01827904)

A Feasibility Study to Evaluate the Safety and Initial Effectiveness of MR Guided High Intensity Focused Ultrasound (MRgHIFU) in the Treatment of Facetogenic Lumbar Back Pain
To evaluate the safety and initial effectiveness of using MR guided focused ultrasound technology to treat low back pain caused by facet joint arthritis.
PI: Pejman Ghanouni, MD, PhD

Alzheimer's Disease Neuroimaging Initiative 2 (ADNI2)
This study will build upon the Alzheimer's Disease Neuroimaging Initiative (ADNI1) and ADNI-GO to measure the progression of mild cognitive impairment (MCI) and early Alzheimer's disease (AD) with neuroimaging.
PI: Jerome Yesavage, MD (NCT01231971)

A Study to Explore the Safety and Tolerability of Acthar in Patients with Amyotrophic Lateral Sclerosis
An 8-week randomized, open-label evaluation to examine the acute safety and tolerability of 4 Acthar dosing regimens to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis.
PI: Yuen So, MD, PhD (NCT01906658)

Compassion Training and Pain
The purpose of this study is to determine whether compassion training will improve the physical and psychological well-being of patients with chronic pain.
PI: Sean Mackey, MD, PhD (NCT01776645)

Epilepsy Impact Scale
The investigators are developing a questionnaire to quickly measure the impact of epilepsy on a person's life and whether it increases, decreases or stays the same over time.
PI: Robert Fisher, MD, PhD (NCT01833234)

Maternal and Neurodevelopmental Outcomes of in Utero Antiepileptic Drug (AED) Exposure (MONEAD)
To establish the relationship between antiepileptic drug exposure and outcomes in the mother and child as well as describe and explain the variability in antiepileptic drug exposure and response
PI: Kimford J. Meador, MD (NCT01730170)

VNS Therapy Automatic Magnet Mode Outcomes Study in Epilepsy Patients Exhibiting Ictal Tachycardia (E-37)
Obtain baseline clinical outcome data (Stage 1) upon which to base a subsequent study (Stage 2) of the Model 106 VNS implantable pulse generator.
PI: Robert Fisher, MD, PhD (NCT01846741)

Lactic Acidosis During and After Seizures
This project looks at the time course of lactic acid rise (if any) after seizures. Salivary and capillary lactic acids are tested.
PI: Robert Fisher, MD, PhD (NCT01833247)

Vitamin D Supplementation in Multiple Sclerosis
Patients with relapsing-remitting MS will receive high-dose or low-dose oral vitamin D and glatiramer acetate. The rate of MS attacks, number of new lesions and change in brain volume on MRI will be determined.
PI: Alexandra Goodyear, MD (NCT01490502)

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of BIIB033 in Subjects with Relapsing Forms of Multiple Sclerosis when used Concurrently with Avonex
PI: Alexandra Goodyear, MD (NCT01864148)

An Intravenous Infusion Study of rHIgM22 in Patients with Multiple Sclerosis
This Phase I, multi-center, double-blind, randomized, placebo-controlled, dose-escalation study will evaluate safety, tolerability, pharmacokinetics, and immunogenicity of single intravenous (IV) administrations of rHIgM22 in patients with MS.
PI: Alexandra Goodyear, MD (NCT01803867)

Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses (A1AT for NMO)
PI: Alexandra Goodyear, MD (NCT02087813)

Understanding Participation of Racial and Ethnic Groups in Multiple Sclerosis Clinical Trials
PI: Jeffrey Dunn, MD

Antihypertensive Treatment of Acute Cerebral Hemorrhage (ATACH-II)
To 1) determine the therapeutic benefit of intensive treatment compared to standard treatment in patients with death and disability (mRS 4-6) at 3 months among subjects with ICH who are treated within 4.5 hours of symptom onset; 2) compare quality of life at 3 months;  3) compare hematoma expansion;  and 4) compare treatment-related serious adverse events within 72hrs.
PI: Chitra Venkatasubramanian, MBBS, MD (NCT01176565)

Development of Multimodal Imaging Biomarkers for Cognitive Dysfunction in Parkinson's Disease
To develop multimodal imaging biomarkers to characterize the etiology of Parkinson's disease associated cognitive impairment and dementia.
PI: Kathleen Poston, MD, MS

Early Differential Diagnosis of Parkinsonism with Metabolic Imaging and Pattern Analysis
PI: Kathleen Poston, MD, MS

A Phase II, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AMG 334 in Chronic Migraine Prevention
To evaluate the effect of AMG 334 compared to placebo on the change from baseline in monthly migraine days, in subjects with chronic migraine.
PI: Sheena K. Aurora, MD (NCT02066415)

CHAMP: The Childhood and Adolescent Migraine Prevention Study
To evaluate if amitriptyline (AMI) and topiramate (TPM) are superior to placebo in reducing migraine frequency in children and adolescents, ages 8 to 17 years old, inclusive, and to conduct a comparative effectiveness study of the two therapies.
PI: Sheena K. Aurora, MD (NCT01581281)

Non-Invasive Neurostimulation of the Vagus Nerve with the Gammacore Device for the Treatment of Cluster Headache
To collect clinical data related to the safety and effectiveness of non-invasive vagus nerve stimulation with the GammaCore® device for the acute treatment of cluster headache.
PI: Sheena K. Aurora, MD (NCT01792817)

Assessment of LBR-101 in Chronic Migraine
To evaluate the efficacy of two distinct doses of subcutaneous LBR-101 in the preventive treatment of chronic migraine, measured by mean change from baseline in number of monthly cumulative headache hours of any severity on headache days, captured from the daily headache diary, relative to the 28-day post treatment period ending with week 12.
PI: Sheena K. Aurora, MD (NCT02021773)

A Multicenter Assessment of LBR-101 in High Frequency Episodic Migraine (HFEM)
To evaluate the efficacy of two distinct doses of subcutaneous LBR-101 in the preventive treatment of HFEM, measured by mean change from baseline in the monthly migraine days during the 28-day post treatment period ending with week 12.
PI: Sheena K. Aurora, MD (NCT02025556)

Migraine Prophylaxis with BOTOX in Adults
To study the long-term efficacy, safety and tolerability of BOTOX (onabotulinumtoxinA) for the prophylaxis of headaches in adult patients with chronic migraine
PI: Meredith Barad, MD

Migraine Prophylaxis with BOTOX in Children
To study the long-term efficacy, safety and tolerability of BOTOX (onabotulinumtoxinA) for the prophylaxis of headaches in adolescents patients (children 12 to < 18 years of age) with chronic migraine.
PI: Meredith Barad, MD

Screening Tool Validation for Chronic Migraine
PI: Nada Hindiyeh, MD

Population-based Studies of the Prevalence and Predisposing Factors of Peripheral Neuropathy
In collaboration with epidemiologists at UC Berkeley, the study investigates the potential environmental or occupational risk factors for peripheral neuropathy.
PI: Yuen So, MD, PhD

HDE Post-Approval Study (PAS) of NeuRx DPS™ for ALS 
Further investigation on the benefits and risks of diaphragmatic pacing to preserve respiratory function in patients with amyotrophic lateral sclerosis (ALS).
PI: Yuen So, MD, PhD (NCT01605006)

A Phase IV Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
PI: John Day, MD, PhD (NCT01526785)

Brain Networks in Neurodegenerative Diseases
To prospectively evaluate the application of FDG PET to aid in the diagnosis of Parkinson’s disease and other atypical Parkinsonian syndromes. 
PI: Kathleen Poston, MD, MS

Transient Ischemic Attack (TIA) Triage and Evaluation of Stroke Risk
PI: Gregory Albers, MD (NCT01423201)

An Exploratory Study to Assess Two Doses of GSK2402968 in the Treatment of Ambulant Boys with Duchenne Muscular Dystrophy
PI: Yuen So, MD, PhD (NCT01462292)

The PLasma for Alzheimer's SymptoM Amelioration (PLASMA) Study
A Randomized, Double-Blind, Placebo-Controlled, Cross-Over Trial of Intravenously Administered Plasma from Young Donors for Treatment of Mild-to-Moderate Alzheimer's Disease
PI: Sharon J. Sha, MD MS (NCT02256306)
Efficacy and Safety Trial of MK-8931 in Participants with Prodromal Alzheimer's Disease
A Phase III, Randomized, Placebo-Controlled, Parallel-Group, Double-Blind Clinical Trial to Study the Efficacy and Safety of MK-8931 in Subjects with Amnestic Mild Cognitive Impairment Due to Alzheimer’s Disease.
PI: Sharon J. Sha, MD MS (NCT01953601)

The Aging Brain: Risk for Dementia
This study will enroll older individuals with cognitive problems with the goal of determining which factors are most predictive developing dementia. 
PI: Sharon J. Sha, MD MS

High Resolution Imaging of Hippocampal Mechanisms in Age-Related Memory Decline
This study will enroll healthy older adults without memory complaints to better understand the processes and mechanisms of memory that change as we age.
PI:  Anthony Wagner, PhD

Prognosis of Critically Ill Neurological Patients
To determine how well health care providers can predict future neurological outcomes, if they differ in the prediction of outcome, and to assess outcomes of this patient population. 
PI: Anna Finley-Caulfield, MD

Tissue Banking of Blood, Spinal Fluid or Skin Biopsy for the Research of Neurological Diseases
PI: Yuen So, MD, PhD

Glyburide Advantage in Malignant Edema and Stroke
A randomized, multi-center, prospective, double blind, two-stage, adaptive Phase II trial of RP-1127 (Glyburide for Injection) in patients with a severe anterior circulation ischemic stroke who are likely to develop malignant edema.
PI: Gregory Albers, MD (NCT01794182)
Platelet-Oriented Inhibition in New TIA and Minor Ischemic Stroke (POINT) Trial
A phase III study to determine if aspirin and clopidogrel together reduces the risk of stroke, heart attacks and other complications compared to aspirin alone. 
PI: Gregory Albers, MD, James Quinn, MD (NCT00991029)

Stroke Hyperglycemia Insulin Network Effort (SHINE) Trial
To determine safety and therapeutic benefit of treating hyperglycemic acute ischemic stroke
patients with targeted glucose concentration (80mg/dL - 130 mg/dL).
PIs: Karen Hirsch, MD, James Quinn, MD (NCT01369069)

Clot Lysis: Evaluating Accelerated Resolution of Intraventricular Hemorrhage Phase III (CLEAR III) 
To determine if EVD placement with low-dose rt-PA improves modified Rankin Scale scores at 6 months compared to subjects treated with EVD placement with placebo. 
PI: Chitra Venkatasubramanian, MBBS, MD (NCT00784134)

Clinical and Genetic Characterization of Myotonic Dystrophy
PI: John W. Day, MD, PhD

Safety and Efficacy Evaluation of Threshold Sound Conditioning by Conditioning-enhanced
Hearing Aid
PI: Jaime Lopez, MD

CRISP : A Multi-Center Cohort Study of Acute Stroke Patients who are Treated with Endovascular Therapy
The study is designed to determine optimal CT perfusion criteria to select patients for endovascular stroke treatment.
PI: Maarten Lansberg, MD, PhD (NCT01622517)

A Phase III, Randomized, Open-Label, 500-Subject Clinical Trial of Minimally Invasive Surgery Plus rt-PA in the Treatment of Intracerebral Hemorrhage (MISTIE III)   
PI: Chitra Venkatasubramanian, MBBS, MD (NCT01827046)

Subject Database and Specimen Repository for Neuromuscular and Neurodegenerative Disorders
PI:  John W. Day, MD, PhD

Inherited Neuropathies Consortium
PI: John W. Day, MD, PhD

Development and Validation of a Disability Severity Index for Charcot-Marie-Tooth Disease
PI: John W. Day, MD, PhD (NCT01455623)

A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients with Infantile-Onset Spinal Muscular Atrophy
PI: John W. Day, MD, PhD (NCT01839656)

A Phase III Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
PI: John W. Day, MD, PhD (NCT01826487)

Phase I/Ib, Multicenter, Open-label, Dose-Escalation and Expansion Study to Evaluate the Safety and Antitumor Activity of MEDI3617, a Human Monoclonal Antibody Directed Against ANG2, as a Single-Agent or in Combination Therapy in Adult Subjects with Advanced Solid Tumors.
PIs: Seema Nagpal, MD and Lawrence Recht, MD (NCT01248949)


Quantifying Collateral Perfusion in Cerebrovascular Disease
This study utilizes MRI to improve the detection and assessment of collateral blood vessels in patients with diseases of the brain, such as Moyamoya disease and stroke. 
PI:  Greg Zaharchuk, MD, PhD (NCT01419275)

Microvascular Measures of Perfusion in Stroke Recanalization
This study will lead to better understanding of mapping flow and microvascular status in patients with severe cerebrovascular disease and greatly enhance the already significant diagnostic power of MRI in acute ischemic stroke by better mapping metabolic perfusion mismatches after reperfusion.
PIs: Michael Moseley, PhD, Greg Albers, MD

Imaging Collaterals in Acute Stroke (iCAS)
The goal of this multi-center NIH study is to develop new magnetic resonance imaging methods to assess brain collaterals and to evaluate whether this information can help us better identify acute stroke patients who would benefit from endovascular therapy.
PI:  Greg Zaharchuk, MD, PhD

Advanced MR and CT Imaging for Understanding Acute Stroke Evolution and Predicting Response to Recanalization Therapy
This study aims to evaluate the diagnostic ability of MRI and CT to reliably identify irreversibly damaged tissue, at-risk tissue and tissue that is most likely to transform into hemorrhage in acute stroke patients.
PIs: Roland Bammer, PhD, Greg Albers, MD


Phase III Study Evaluating Limited Target Volume Boost Irradiation and Reduced Dose Craniospinal Radiotherapy 18.00 Gy and Chemotherapy in Children with Newly Diagnosed Standard Risk Medulloblastoma
PI: Paul Fisher, MD (NCT00085735)

Efficacy of Carboplatin Administered Concomitantly with Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other than Average Risk Medulloblastoma/PNET Patients 
PI: Paul Fisher, MD (NCT00392327)

Phase II Screening Trial of Temozolomide with Irinotecan versus Temozolomide, Irinotecan plus for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood
PI: Paul Fisher, MD (NCT01217437)

A Randomized Phase II/III Study of Vorinostat and Local Irradiation OR Temozolomide and Local Irradiation OR Bevacizumab and Local Irradiation Followed by Maintenance Bevacizumab and Temozolomide in Newly Diagnosed High Grade Glioma
PI: Paul Fisher, MD (NCT01236560)

Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Ages 1 to 21 years
PI: Paul Fisher, MD (NCT01096368)

A Phase II Study of Sunitinib in Recurrent, Refractory or Progressive High Grade Glioma and Ependymoma Brain Tumors in Pediatric and Young Adult Patients
 PI: Paul Fisher, MD (NCT01462695)

A Phase II Randomized Trial of Lenalidomide (NSC # 703813, IND # 70116) in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytoma and Optic Pathway Gliomas
 PI: Paul Fisher, MD (NCT01553149)

Phase II Trial of Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors (CNS GCT)
PI: Paul Fisher, MD (NCT01602666)

Risk-Adapted Therapy for Young Children with Embryonal Brain Tumors, High-Grade Glioma, Choroid Plexus Carcinoma Or Ependymoma (SJYC07)
PI: Paul Fisher, MD (NCT00602667)

Phase I and Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma
PI: Paul Fisher, MD (NCT01089101)

Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children with Recurrent or Refractory Medulloblastoma
PI: Paul Fisher, MD (NCT01239316)

Comprehensive Molecular Analysis of Tumor Samples Derived From Patients with Diffuse Brainstem Glioma—A Pilot Study
PI: Paul Fisher, MD (NCT00899834)

A Phase II Placebo-Controlled Trial of Modafinil to Improve Neurocognitive Deficits in Children Treated for a Primary Brain Tumor
PI: Paul Fisher, MD (NCT01381718)

A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children with Recurrent High-Grade Glioma, Ependymoma, Medulloblastoma/Primitive Neuroectodermal Tumor (PNET) and Diffuse Intrinsic Pontine Glioma (DIPG)
PI: Paul Fisher, MD (NCT01836549)

For questions about the following clinical trials please contact our clinical trials research coordinator Maria Coburn at 650-736-9551 or

For more details on all of our clinical trials visit the SOM Clinical Trial Database or visit

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