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 |
Pending
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 |