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Clinical Studies
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PLS Natural History Study (PLS)
Rochester, Minn.,
Jacksonville, Fla.
The purpose of this study is to develop a natural history dataset and biorepository of early Primary Lateral Sclerosis (PLS) and well-established PLS cases for future clinical trials.
Closed for Enrollment
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A Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled, First-In-Patient Study Of AJ201 To Evaluate Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics In Adults With Spinal And Bulbar Muscular Atrophy (SMBA)
Rochester, Minn.,
Jacksonville, Fla.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).
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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) (FORTITUDE-ALS)
Rochester, Minn.
The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.
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A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation
Jacksonville, Fla.,
Rochester, Minn.
The primary purpose of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adults with Amyotrophic Lateral Sclerosis (ALS). The primary objective of Part C of this study is to evaluate the clinical effectiveness of BIIB067 administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation.
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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
Rochester, Minn.,
Jacksonville, Fla.
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) profiles and effects on disease progression of BIIB067 administered to participants with ALS and confirmed SOD1 mutation.
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Compassionate Use of 3,4 Diaminopyridine in Patients with Congenital Myasthenic Syndrome
Rochester, Minn.
This study is being done to find out whether the investigational drug 3,4-DAP will help patients with Congenital Myasthenic Syndrome (CMS), inherited muscle weakness and fatigue.
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Compassionate Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome
Rochester, Minn.
This study is being done to determine the long-term response of patients with Lambert-Eaton Myasthenic Syndrome (LEMS) to the investigational drug [not yet approved by the Food and Drug Administration (FDA)] 3, 4-DAP. 3, 4-DAP increases the communication between the nerve and muscle by increasing the release of an important compound in this process, acetylcholine. This drug has been shown in studies done at Mayo and elsewhere to benefit patients with LEMS as well as some patients with congenital myasthenic syndromes.
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Development of a PLS-Specific Clinical Rating Scale, Capable of Evaluating the Clinical Functional State of Patients with PLS in Multi-Site Study
Jacksonville, Fla.,
Rochester, Minn.
The purpose of this study is to assess the internal consistency and construct validity of the scale, assess test-retest reliability, intra-rater reliability, inter-rater reliability, reliability between in-person and telephone administration of the scale, to determine the sensitivity of the scale to detect changes over time.
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Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis
Rochester, Minn.
The purpose of this study is to evaluate and rank-order a set of new outcome measures administered to patients with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than the current Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
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