| Principal investigator | Title |
|---|---|
| Randall Bateman, MD | Dominantly 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, MD | Dominantly 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, DO | 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, ClinicalTrials.gov Identifier: Not yet available |
| Randall Bateman, MD | Dominantly Inherited Alzheimer Network (DIAN) – Coordinating Center Application (No subjects are enrolled under this submission.) |
| Eric M. McDade, DO | Dominantly Inherited Alzheimer Network (DIAN) Expanded Registry |
| John Morris, MD | Protocol I7X-MC-LLCF Effect of LY3202626 on Alzheimers Disease Progression as Measured by Cerebral 18F-AV-1451 Tau-PET in Mild Alzheimers Disease Dementia |
| Anne Niven | Analysis of Human CSF Research Samples for Biomarkers of Novel (non-A-B or Tau) Analytes for Alzheimers 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 Alzheimers 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 |
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.
| 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 |
| Principal investigator | Title |
|---|---|
| Kevin Black, MD | Peripheral Induction of Inhibitory Brain Circuits to Treat Tourette’s: Pilot |
| Susan Criswell, MD | An Open-Label, Long Term Safety Study of SD-809 ER in Subjects With Chorea Associated With Huntington Disease |
| Susan Criswell, MD | A 12 Month, Dose-Level Blinded Study Investigating the Safety and Efficacy of CVT 301 (Levodopa Inhalation Powder) in Parkinsons 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 Parkinsons 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 (LID) |
| Principal investigator | Title |
|---|---|
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, 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 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, 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) |
| 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 |
| Principal investigator | Title |
|---|---|
| Robert Bucelli, MD, PhD | 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 Bucelli, MD, PhD | 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 |
| Robert Bucelli, MD, PhD | A 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, MD | An Open-Label, Multicenter Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy |
| 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 |
| 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) |
| 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) |
| 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 |
| 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 |
| Glenn Lopate, MD | Expanded Access Protocol of Patisiran for Patients with Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy |
| Timothy Miller, MD, PhD | Genomic Translation for ALS Care |
| Timothy Miller, MD, PhD | Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients with ALS |
| Timothy Miller, MD, PhD | Understanding SOD1 Kinetics in Amyotrophic Lateral Sclerosis |
| Timothy Miller, MD, PhD | Dominant Inherited ALS (DIALS) Network |
| Timothy Miller, MD, PhD | Answer ALS: Individualized Initiative for ALS Discovery |
| Timothy Miller, MD, PhD | Evaluation of Biomarkers in Blood of Patients with Neuromuscular Diseases |
| Timothy Miller, MD, PhD | Neurologic and Neuromuscular Biorepository – Cerebrospinal Fluid |
| Timothy Miller, MD, PhD | Tissue Donation for Research |
| 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 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, 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 |
| Alan Pestronk, MD | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy |
| 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) |
| 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 |
| 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) |
| 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 |
| 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 |
| 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) |
| Principal investigator | Title |
|---|---|
| Anne Connolly, MD | An Open-Label, Safety Study for 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 Patisiran Clinical Study |
| 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 |
| 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 |
| Principal investigator | Title |
|---|---|
| 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 |
None available at this time.
| 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, 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 |