Neurology Clinical Trials

Washington University neurologists are leading groundbreaking clinical trials aimed at improving the neurological health of people throughout the world.

By participating in a clinical trial, our patients are shaping the future of medicine. Clinical trials allow researchers to study new treatment options, drugs or combinations of treatments to determine if they can be integrated into a standard practice of care. A list of active clinical trials is provided below.

Aging & Dementia

Principal Investigator Title
Randall Bateman, MD Dominantly Inherited Alzheimer Network (DIAN) - Coordinating Center Application (No subjects are enrolled under this submission.)
Eric McDade Dominantly Inherited Alzheimer Network (DIAN) Expanded Registry
John Morris, MD Protocol I7X-MC-LLCF Effect of LY3202626 on Alzheimer’s Disease Progression as Measured by Cerebral 18F-AV-1451 Tau-PET in Mild Alzheimer’s Disease Dementia
Anne Niven Analysis of Human CSF Research Samples for Biomarkers of Novel (non-A-B or Tau) Analytes for Alzheimer’s Disease
John Morris, MD A Phase 2b/3 Randomized, Double-blind, Placebo-Controlled, Parallel Group, Multicenter Study Investigating the Efficacy and Safety of JNJ-54861911 in Subjects who are Asymptomatic At Risk for Developing Alzheimer’s Dementia
Barbara Snider, MD, PhD A Phase 3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Aducanumab (BIIB037) in Subjects with Early Alzheimer's Disease
Beau Ances, MD, PhD Alzheimer’s Disease Neuroimaging Initiative 3

Epilepsy & Seizure Disorders

Principal Investigator Title
Brian Day, MD, PhD Prospective Florbetapir F 18 PET/CT Neuroimaging to Evaluate Brain Amyloid Deposition in Chronic Temporal Lobe Epilepsy
Kwee Thio, MD, PhD Human Epilepsy Project
Robert Hogan, MD A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF LACOSAMIDE AS ADJUNCTIVE THERAPY FOR UNCONTROLLED PRIMARY GENERALIZED TONIC-CLONIC SEIZURES IN SUBJECTS WITH IDIOPATHIC GENERALIZED EPILEPSY
Robert Hogan, MD AN OPEN-LABEL, MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF LACOSAMIDE AS ADJUNCTIVE THERAPY FOR UNCONTROLLED PRIMARY GENERALIZED TONIC-CLONIC SEIZURES IN SUBJECTS WITH IDIOPATHIC GENERALIZED EPILEPSY
Robert Hogan, MD AN OPEN-LABEL, REPEAT-DOSE PHARMACOKINETICS STUDY OF NRL-1 IN EPILEPSY SUBJECTS UNDER SEIZURE AND NORMAL CONDITIONS
Robert Hogan, MD A 12-Month Open-Label, Repeat-Dose Safety Study of NRL-1 in Epilepsy Subjects (DIAZ.001.05)
Robert Hogan, MD An Open Label, Multicenter, Safety and Pharmacokinetic Study of YKP3089 as Adjunctive Therapy in Subjects with Partial Onset Seizures
Robert Hogan, MD A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SAGE-547 Injection in the Treatment of Subjects with Super-Refractory Status Epilepticus
Michael Diringer, MD An Open Label, Expanded Access Protocol to Evaluate the Efficacy and Safety of SAGE-547 Injection in the Treatment of Subjects with Super-Refractory Status Epilepticus

Movement Disorders

Principal Investigator Title
Susan Criswell, MD AN OPEN-LABEL, LONG TERM SAFETY STUDY OF SD-809 ER IN SUBJECTS WITH CHOREA ASSOCIATED WITH HUNTINGTON DISEASE
Brad Racette, MD A Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled,Parallel-Group Study to Evaluate the Efficacy and Safety of Laquinimod (0.5,1.0 and 1.5 mg/day) as Treatment in Patients with Huntington's Disease / Phase II Study
Susan Criswell, MD A 12 Month, Dose-Level Blinded Study Investigating the Safety and Efficacy of CVT 301 (Levodopa Inhalation Powder) in Parkinson’s Disease Patients With Motor Response Fluctuations (OFF Phenomena)
Susan Criswell, MD A Phase 3, Multicenter, Randomized, Double-Blind, Placebo- Controlled Study with an Open-Label Phase to Determine the Efficacy and Safety of Tozadenant as Adjunctive Therapy in Levodopa-Treated Patients with Parkinson’s Disease Experiencing End-of-Dose “Wearing-Off”(TOZ-PD)
Brad Racette, MD A Phase 2 multi-center, randomized, double-blind, placebo controlled study in subjects with late prodromal and early manifest Huntington disease (HD) to assess the safety, tolerability, pharmacokinetics, and efficacy of VX15/2503 (SIGNAL)
Paul Kotzbauer, MD, PhD DUOdopa/Duopa in Patients with Advanced Parkinson's Disease (PD) – a GLobal OBservational Study Evaluating Long-Term Effectiveness
Susan Criswell, MD A multicenter, randomized, double-blind, placebo controlled, parallel group clinical study investigating the efficacy, tolerability, and safety of continuous subcutaneous ND0612 infusion Given as adjunct treatment to oral levOdopa in patients with Parkinson’s Disease with motor fluctuations
Susan Criswell, MD A multi-center, North American, open-label extension study of pridopidine (ACR16) in the symptomatic treatment of Huntington's Disease
Susan Criswell, MD A Phase 2a, Double-Blind, Placebo-Controlled, Two-Part Study To Investigate the Safety And Efficacy Of Increasing Doses Of DNS-7801 In Parkinson’s Disease (PD) Subjects With Motor Fluctuations
Susan Criswell, MD OPEN-LABEL SAFETY STUDY OF ADS-5102 (AMANTADINE HCL) EXTENDED RELEASE CAPSULES FOR THE TREATMENT OF LEVODOPA INDUCED DYSKINESIA

Multiple Sclerosis (MS)

Principal Investigator Title
Dorothy Cross, MD Protocol Number: WA25046, A Phase III, multicentre, randomized, parallel-group, double blinded, placebo controlled study to evaluate the efficacy and safety of ocrelizumab in adults with Primary Progressive Multiple Sclerosis
Robert Naismith, MD Protocol Number: WA21092, A Randomized, Double-Blind, Double-Dummy, Parallel-Group Study To Evaluate The Efficacy And Safety Of Ocrelizumab In Comparison To Interferon Beta-1a (Rebif®) In Patients With Relapsing Multiple Sclerosis
Robert Naismith, MD A RANDOMIZED, DOUBLE-BLIND,PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND ACTIVITY OF IBUDILAST (MN-1660 IN SUBJECTS WITH PROGRESSIVE MULTIPLE SCLEROSIS
Robert Naismith, MD A Mutlicenter, Global, Observational Study to Collect Information on Saftey and to Document the Drug Ulitization of Tecfidera (TM) (Dimethyl Fumarate) When Used in Routine Medical Practice in the Treatment of Multiple Sclerosis
Robert Naismith, MD A Multinational, Multicenter, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Once Daily Oral Administration of Laquinimod (0.6 or 1.5 mg) in Patients with Primary Progressive Multiple Sclerosis (PPMS)
Robert Naismith, MD A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Trial to Evaluate the Safety and Efficacy of Eculizumab in Patients with Relapsing Neuromyelitis Optica (NMO)
Robert Naismith, MD A Phase III, Open-Label, Extension Trial of ECU-NMO-301 to Evaluate the Safety and Efficacy of Eculizumab in Patients with Relapsing Neuromyelitis Optica (NMO)
Timothy Miller, MD, PhD Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis
Alan Pestronk, MD A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis
Robert Naismith, MD An Open-Label Study to Evaluate the Effectiveness and Safety of Ocrelizumab in Patients with Relapsing Remitting Multiple Sclerosis Who Have Had a Suboptimal Response to an Adequate Course of Disease-Modifying Treatment
Dorothy Cross AN OPEN-LABEL, MULTICENTER, BIOMARKER STUDY TO EXPLORE THE MECHANISM OF ACTION OF OCRELIZUMAB AND B-CELL BIOLOGY IN PATIENTS WITH RELAPSING MULTIPLE SCLEROSIS OR PRIMARY PROGRESSIVE MULTIPLE SCLEROSIS
Robert Naismith, MD A Phase 3 Open Label Study to Evaluate the Long-term Safety and Tolerability of ALKS 8700 in Adults with Relapsing Remitting Multiple Sclerosis
Robert Naismith, MD A randomized, double-blind, double-dummy, parallel-group study comparing the efficacy and safety of ofatumumab versus teriflunomide in patients with relapsing multiple sclerosi
Robert Naismith, MD Multi-center, randomized, double-blinded assessment of Tecfidera (R) in extending the time to a first attack in radiologically isolated syndrome
Robert Naismith, MD AN OPEN-LABEL, MULTICENTER, EXPANDED ACCESS PROGRAM FOR OCRELIZUMAB IN PATIENTS WITH PRIMARY PROGRESSIVE MULTIPLE SCLEROSIS
Robert Naismith, MD A Multi-Site, Open-Label Extension Trial of Oral RPC1063 in Relapsing Multiple Sclerosis
Robert Naismith, MD Effect of MD1003 in progressive multiple sclerosis: a randomized double blind placebo controlled study
Robert Naismith, MD A Phase 3 Study in Subjects with Relapsing Remitting Multiple Sclerosis to Evaluate the Tolerability of ALKS 8700 and Dimethyl Fumarate
Robert Naismith, MD Multiple Sclerosis Partners Advancing Technology and Health Solutions
Gregory Wu, MD, PhD Plegridy (peginterferon B-1a) Real World Effectiveness and Safety Observational Program

Neuromuscular Disorders

Principal Investigator Title
Timothy Miller, MD, PhD Defining Clinical Characteristics in C9ORF72 ALS Patients
Anne Connolly, MD An Open-Label, Multicenter Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Alan Pestronk, MD An open-label, multicenter, multinational, extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
Anne Connolly, MD A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY
Anne Connolly, MD An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients with Advanced Stage Duchenne Muscular Dystrophy
Anne Connolly, MD An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy (4658-203)
Anne Connolly, MD A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne Muscular Dystrophy
Alan Pestronk, MD A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)
Anne Connolly, MD Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects with Duchenne Muscular Dystrophy
Anne Connolly, MD A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy
Anne Connolly, MD An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
Anne Connolly, MD A PHASE 2, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, MULTIPLE DOSE STUDY OF CK-2127107 IN TWO ASCENDING DOSE COHORTS OF PATIENTS WITH SPINAL MUSCULAR ATROPHY (SMA)
Timothy Miller, MD, PhD Genomic Translation for ALS Care
Alan Pestronk, MD A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy
Anne Connolly, MD A Phase II, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)
Alan Pestronk, MD A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients with Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY -ALS (CY 4031)
Timothy Miller, MD, PhD A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects with Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation
Alan Pestronk, MD An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation
Anne Connolly, MD Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy
Anne Connolly, MD A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy
Anne Connolly, MD A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)
Anne Connolly, MD A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY
Timothy Miller, MD, PhD Evaluation of the safety, tolerability, efficacy and activity of AMX0035, a fixed combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for treatment of amyotrophic lateral sclerosis (ALS)
Alan Pestronk, MD A PHASE 2, MULTI-CENTER, DOUBLE-BLIND, RANDOMIZED, DOSE-RANGING, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF CK-2127107 IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS (ALS)
Anne Connolly, MD Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy
Anne Connolly, MD Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Anne Connolly, MD A PHASE 3 EXTENSION STUDY OF ATALUREN (PTC124) IN PATIENTS WITH NONSENSE MUTATION DYSTROPHINOPATHY
Alan Pestronk, MD A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial of IMO-8400 in Patients with Dermatomyositis
Alan Pestronk, MD A Phase 2 Open-label study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (also known asor Hereditary Inclusion Body Myopathy (HIBM)) patients with Severe Ambulatory Impairment.
Alan Pestronk, MD A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Alan Pestronk, MD A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease
Glenn Lopate, MD Expanded Access Protocol of Patisiran for Patients with Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy

Neuropsychiatric Disorders

Principal Investigator Title
Anne Connolly, MD Open Label, Multiple-Dose Efficacy, Safety, and Tolerability Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy who Participated in Study 4658-US-201

Neurorehabilitation

Principal Investigator Title
Glenn Lopate, MD APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)

Other

Principal Investigator Title
Anne Connolly, MD AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED ATALUREN (PTC124) PATIENTS WITH NONSENSE MUTATION DYSTROPHINOPATHY
Alan Pestronk, MD An Open label Phase 2 Extension Study to Evaluate the Long Term-Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR)Capsules in Patients with GNE Myopathy or Hereditary Inclusion Body Myopathy Protocol UX001-CL202
Alexandre Carter, MD, PhD Distorted Reaching in Virtual Reality for the Treatment of Hemispatial Neglect
Glenn Lopate, MD A MULTICENTER, OPEN-LABEL, EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF PATISIRAN IN PATIENTS WITH FAMILIAL AMYLOIDOTIC POLYNEUROPATHY WHO HAVE COMPLETED A PRIOR CLINICAL STUDY WITH PATISIRAN
Luqi Chi, MD Registry to Examine the Effect of CPAP on Cardiac Function in Hospitalized Patients with OSA and Heart Failure
Anne Niven Elecsys evaluation in a Knight ADRC Cohort
Anne Niven Lumipulse Evaluation in a Knight ADRC Cohort
David Brody Advanced brain imaging and biochemical approaches to traumatic brain injury
Brad Racette, MD A PHASE IV, PROSPECTIVE, OBSERVATIONAL, MULTICENTRE STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF DYSPORT® (ABOBOTULINUMTOXINA) IN PAEDIATRIC LOWER LIMB SPASTICITY
Susan Criswell, MD A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of CX-8998 for Essential Tremor
Alan Pestronk, MD A Phase 3, Randomized, Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Abatacept SC with Standard Treatment Compared to Standard Treatment Alone in Improving Disease Activity in Adults with Active Idiopathic Inflammatory Myopathy (IIM)
Glenn Lopate, MD A Phase 2, Randomized, Double-blind, Placebo-controlled, 6-Week, Parallel-design Study of the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy

Pediatric Neurology

Principal Investigator Title
Bryan McGill, MD Effects of AFQ056 on Language Learning in Young Children with Fragile X Syndrome (FXS)
Brad Racette, MD BOTOX® Treatment of Pediatric Lower Limb Spasticity: Open-label Study
Brad Racette, MD BOTOX® Treatment in Pediatric Upper Limb Spasticity: Open-label Study
Michael Wong, MD, PhD Afinitor/Votubia (Everolimus), A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures
Michael Wong, MD, PhD A double-blind, randomized, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P, CBD) as add-on therapy in patients with tuberous sclerosis complex who experience inadequately-controlled seizures

Sleep Medicine

Principal Investigator Title
   

Stroke & Cerebrovascular Disease

Principal Investigator Title
Renee Van Stavern, MD Randomized, double-blind, Evaluation in secondary Stroke Prevention comparing the EfficaCy and safety of the oral Thrombin inhibitor dabigatran etexilate (110 mg or 150 mg, oral b.i.d.) versus acetylsalicylic acid (100 mg oral q.d.) in patients with Embolic Stroke of Undetermined Source (RESPECT ESUS)
Jin-Moo Lee, MD, PhD A Multicenter, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Ranging Study to Evaluate the Safety and Efficacy of Intravenous Natalizumab (BG00002) in Acute Ischemic Stroke
Paul Kotzbauer, MD, PhD Alpha-Synuclein Kinetics
Andria Ford, MD, MSCI A PHASE 2, MULTICENTER, RANDOMIZED, DOUBLE-BLIND (WITHIN DOSE),PLACEBO-CONTROLLED, PARALLEL-GROUP, DOSE-RANGE-FINDING STUDY TO EVALUATE THE EFFICACY AND SAFETY OF TF0023 SPRAY VERSUS PLACEBO IN FUNCTIONAL IMPROVEMENT OF PATIENTS WITH ISCHEMIC STROKES
Alexandre Carter, MD, PhD IpsiHand Device Use In Hemiparetic and Hemiplegic Stroke Patients to Assess Motor Recovery of the Upper Extremity and Functional Outcomes
Michael Diringer, MD Minimally Invasive Surgery plus rt-PA in the treatment of Intracerebral Hemorrhage
Michael Diringer, MD A PHASE 1B MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE SAFETY AND TOLERABILITY AND DETERMINE THE MAXIMUM TOLERATED DOSE OF PF-05230907 IN SUBJECTS WITH INTRACEREBRAL HEMORRHAGE (ICH)