Aging and dementia
Principal investigatorTitle
Randall Bateman, MDDominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals With a Type of Early Onset Alzheimer’s Disease Caused by a Genetic Mutation (DIAN-TU) (DIAN-TU), ClinicalTrials.gov Identifier: NCT05269394
Randall Bateman, MDDominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer’s Disease Caused by a Genetic Mutation. Master Protocol DIAN-TU001 (DIAN-TU), ClinicalTrials.gov Identifier: NCT01760005
Eric M. McDade, DOA Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer’s Disease Caused by a Genetic Mutation, ClinicalTrials.gov Identifier: Not yet available
Randall Bateman, MDDominantly Inherited Alzheimer Network (DIAN) – Coordinating Center Application (No subjects are enrolled under this submission.)
Eric M. McDade, DODominantly Inherited Alzheimer Network (DIAN) Expanded Registry
John Morris, MDProtocol 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 NivenAnalysis of Human CSF Research Samples for Biomarkers of Novel (non-A-B or Tau) Analytes for Alzheimer’s Disease
John Morris, MDA 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, PhDA 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, PhDAlzheimer’s Disease Neuroimaging Initiative 3

For a more comprehensive list of our Alzheimer’s disease clinical trials, please visit the Knight ADRC website, the Knight ADRC Clinical Trials Unit website and DIAN website.

Epilepsy and seizure disorders
Principal investigatorTitle
Brian Day, MD, PhDProspective Florbetapir F 18 PET/CT Neuroimaging to Evaluate Brain Amyloid Deposition in Chronic Temporal Lobe Epilepsy
Kwee Thio, MD, PhDHuman Epilepsy Project
Robert Hogan, MDA 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, MDAn 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, MDAn Open-Label, Repeat-Dose Pharmacokinetics Study of NRL-1 in Epilepsy Subjects Under Seizure and Normal Conditions
Robert Hogan, MDA 12-Month Open-Label, Repeat-Dose Safety Study of NRL-1 in Epilepsy Subjects (DIAZ.001.05)
Robert Hogan, MDAn Open Label, Multicenter, Safety and Pharmacokinetic Study of YKP3089 as Adjunctive Therapy in Subjects with Partial Onset Seizures
Robert Hogan, MDA 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, MDAn 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 investigatorTitle
Kevin Black, MDPeripheral Induction of Inhibitory Brain Circuits to Treat Tourette’s: Pilot
Susan Criswell, MDAn Open-Label, Long Term Safety Study of SD-809 ER in Subjects With Chorea Associated With Huntington Disease
Susan Criswell, MDA 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, MDA 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, MDA 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, PhDDUOdopa/Duopa in Patients with Advanced Parkinson’s Disease (PD) a GLobal OBservational Study Evaluating Long-Term Effectiveness
Susan Criswell, MDA 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, MDA multi-center, North American, open-label extension study of pridopidine (ACR16) in the symptomatic treatment of Huntington’s Disease
Susan Criswell, MDA 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, MDOpen-Label Safety Study of ADS-5102 (Amantadine HCl) Extended Release Capsules for the Treatment of Levodopa Induced Dyskinesia (LID)
Multiple sclerosis (MS)
Principal investigatorTitle

Anne 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, MDProtocol 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, MDA Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability and Activity of Ibudilast (MN-166) in Subjects With Progressive Multiple Sclerosis
Robert Naismith, MDA 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, MDA 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, MDA 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, MDA 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)
Alan Pestronk, MDA 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, MDAn 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 CrossAn 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, MDA 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, MDA 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, MDMulti-center, randomized, double-blinded assessment of Tecfidera (R) in extending the time to a first attack in radiologically isolated syndrome
Robert Naismith, MDAn Open Label, MultiCenter, Expanded-Access Program for Ocrelizumab in Patients With Primary Progressive Multiple Sclerosis
Robert Naismith, MDA Multi-Site, Open-Label Extension Trial of Oral RPC1063 in Relapsing Multiple Sclerosis
Robert Naismith, MDEffect of MD1003 in progressive multiple sclerosis: a randomized double blind placebo controlled study
Robert Naismith, MDA Phase 3 Study in Subjects with Relapsing Remitting Multiple Sclerosis to Evaluate the Tolerability of ALKS 8700 and Dimethyl Fumarate
Robert Naismith, MDMultiple Sclerosis Partners Advancing Technology and Health Solutions
Gregory Wu, MD, PhDPlegridy (peginterferon B-1a) Real World Effectiveness and Safety Observational Program
Neuromuscular disorders
Principal investigatorTitle
Robert Bucelli, MD, PhDA 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 Bucelli, MD, PhDAn 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
Robert Bucelli, MD, PhDA Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis
Anne Connolly, MDAn Open-Label, Multicenter Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Anne Connolly, MDA 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, MDAn Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients with Advanced Stage Duchenne Muscular Dystrophy
Anne Connolly, MDAn Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy (4658-203)
Anne Connolly, MDA 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
Anne Connolly, MDTrial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects with Duchenne Muscular Dystrophy
Anne Connolly, MDA 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, MDAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
Anne Connolly, MDA 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)
Anne Connolly, MDA 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)
Anne Connolly, MDRandomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy
Anne Connolly, MDA 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, MDA 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, MDA multicenter, open-label extension study to evaluate the long term safety of PF-06252616 in boys with duchenne muscular dystrophy
Anne Connolly, MDPhase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy
Anne Connolly, MDPhase 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, MDA Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
Glenn Lopate, MDExpanded Access Protocol of Patisiran for Patients with Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
Timothy Miller, MD, PhDGenomic Translation for ALS Care
Timothy Miller, MD, PhDEffects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients with ALS
Timothy Miller, MD, PhDUnderstanding SOD1 Kinetics in Amyotrophic Lateral Sclerosis
Timothy Miller, MD, PhDDominant Inherited ALS (DIALS) Network
Timothy Miller, MD, PhDAnswer ALS: Individualized Initiative for ALS Discovery
Timothy Miller, MD, PhDEvaluation of Biomarkers in Blood of Patients with Neuromuscular Diseases
Timothy Miller, MD, PhDNeurologic and Neuromuscular Biorepository – Cerebrospinal Fluid
Timothy Miller, MD, PhDTissue Donation for Research
Timothy Miller, MD, PhDEvaluation 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, MDA 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)
Alan Pestronk, MDAn open-label, multicenter, multinational, extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
Alan Pestronk, MDA Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy
Alan Pestronk, MDA Phase 3, Open-Label Extension Study of Tirasemtiv for Patients with Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY -ALS (CY 4031)
Alan Pestronk, MDAn 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
Alan Pestronk, MDA 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)
Alan Pestronk, MDA Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial of IMO-8400 in Patients with Dermatomyositis
Alan Pestronk, MDA 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, MDA 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, MDA 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
Neuropsychiatric disorders
Principal investigatorTitle
Anne Connolly, MDOpen 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 investigatorTitle
Glenn Lopate, MDAPOLLO: 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 investigatorTitle
Anne Connolly, MDAn Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy
Alan Pestronk, MDAn 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, PhDDistorted Reaching in Virtual Reality for the Treatment of Hemispatial Neglect
Glenn Lopate, MDA 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 Patisiran Clinical Study
Luqi Chi, MDRegistry to Examine the Effect of CPAP on Cardiac Function in Hospitalized Patients with OSA and Heart Failure
Anne NivenElecsys evaluation in a Knight ADRC Cohort
Anne NivenLumipulse Evaluation in a Knight ADRC Cohort
David BrodyAdvanced brain imaging and biochemical approaches to traumatic brain injury
Susan Criswell, MDA Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of CX-8998 for Essential Tremor
Alan Pestronk, MDA 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, MDA 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 investigatorTitle
Michael Wong, MD, PhDAfinitor/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, PhDA 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

None available at this time.

Stroke and cerebrovascular disease
Principal investigatorTitle
Renee Van Stavern, MDRandomized, 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, PhDA 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, PhDAlpha-Synuclein Kinetics
Andria Ford, MD, MSCIA Phase 2, Multicenter, Randomized, Double-blind, 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, PhDIpsiHand Device Use In Hemiparetic and Hemiplegic Stroke Patients to Assess Motor Recovery of the Upper Extremity and Functional Outcomes
Michael Diringer, MDMinimally Invasive Surgery plus rt-PA in the treatment of Intracerebral Hemorrhage