Bjorn E. Oskarsson, M.D.

The purpose of this study is to establish a robust clinical trial-like infrastructure to coordinate recruitment, enrollment, and longitudinal follow up of a cohort of people with ALS and associate control participants.

Amyotrophic lateral sclerosis (ALS) is a fatal, rare neurodegenerative disease affecting 30,000 people in the United States. The gold standard of care for people with ALS is multidisciplinary clinics (MDC). In these multidisciplinary clinics, which occur every 3 to 4 months, people with ALS see up to 12 different healthcare providers (e.g., speech therapy, physical therapy, the ALS doctor). These clinics can last from three to five hours, and across these three to five hours people with ALS and their caregivers receive a lot of information that is critical to the care and quality of life for people with ALS. However, this information can be difficult to remember given the large amount of information that is conveyed. The current standard for providing take-home information about the visit is to provide patients with a written after-visit summary and access to their doctor's notes about the visit, typically through the patient portal. This study tests whether providing participants with video recordings of their MDC visits improves their quality of life and the quality of life of their caregivers. The study will enroll 400 pairs of people with ALS and their caregivers from eight different sites in the United States. Half of the participants in the study will receive their after-visit summary notes (the NOTES condition) and the other half of the participants will receive both their summary notes, but will also receive video recordings of their MDC visits that they can watch on their own at home (the VIDEO condition). The study will last for 12 months, with participants receiving NOTES or VIDEO at each of their regularly-scheduled MDCs during the 12 months. The study will test whether caregiver and patient participants in the VIDEO condition experience better quality of life than those in the NOTES condition at 1 month, 6 months, and 12 months from study enrollment. The results of this study will help determine what is the most effective approach to communicating MDC information to people with ALS and their caregivers.

The purpose of this study is to obtain natural history data and perform longitudinal biofluid collection in individuals genetically at risk for ALS/FTD to characterize pre-symptomatic ALS/FTD disease states.

The primary objective of the proposed study is to examine the feasibility of using digital technology to remotely collect data from people living with amyotrophic lateral sclerosis (PALS) for the purpose of

1. Detecting the development of comorbid conditions, for possible earlier targeted intervention, and

2. Quantifying functional decline for novel clinical trial outcome measure development and guiding clinical care.

The purpose of this study is to test the clinical effectiveness of ibudilast in patients with ALS measured by NfL and change in ALSFRS-R slope.

The purpose of this study is to generate a biorepository of longitudinal blood (plasma and serum), cerebral spinal fluid (CSF) and urine linked to genetics and longitudinal clinical information that are made available to the research community using a speech recording application.

The objectives of this study are development of longitudinal bio-fluid repository in conjunction with collection of clinical data such as ALSFRS-R and ALS-cognitive behavioral screening (ALS-CBS) assessments, assessment of speech over time by collecting speech from participants during their study clinic visits and at home on a weekly basis, and assessment of vital capacity at home for participants who choose to enroll in the optional spirometer assessments for this study.

The primary objective of the proposed study is to examine the feasibility of using digital technology to remotely collect data from people living with amyotrophic lateral sclerosis (PALS) for the purpose of

1. Detecting the development of comorbid conditions, for possible earlier targeted intervention, and

2. Quantifying functional decline for novel clinical trial outcome measure development and guiding clinical care.

The purpose of this study is to collect biofluid samples for the banking and usage in ALS research. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies.

The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-ALS. The secondary objective of this study is to evaluate the pharmacokinetic profile of BIIB078.

The purpose of this study is to evaluate the safety, pharmacokinetics and biodistribution of an imaging agent, 18F-OP-801 (18F Hydroxyl Dendrimer), after intravenous administration to patients with Amyotrophic Lateral Sclerosis (ALS) and Healthy Volunteers (HV)

The primary purpose of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100.

The primary objective is to evaluate the safety and tolerability of BIIB105 in participants with amyotrophic lateral sclerosis (ALS) or poly-CAG expansion (polyQ)-ALS. The secondary objective is to assess the pharmacokinetic (PK) profile of BIIB105 in serum of participants with ALS or poly-CAG expansion (polyQ)-ALS.

This is a Phase 1/2 open-label, multicenter study to evaluate the safety and tolerability and to explore the efficacy of gene therapy AMT-162 in Participants with ALS with pathogenic or likely pathogenic variants in the SOD1 gene (SOD1-ALS). Approximately 12-20 Participants with SOD1-ALS will be treated in this study.

The purpose of this study is to demonstrate the safety and potential effectiveness of TJ-68 for improving muscle cramps in participants with Amyotrophic Lateral Sclerosis (ALS) based on a two-site, randomized, placebo-controlled double-blind multi-period crossover (N-of-1) study design.

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).

The purpose of this study is to evaluate the effect of retigabine dosages on motor neuron activity in people with Amyotrophic Lateral Sclerosis (ALS).

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.

The purpose of this study is to assess the effectiveness, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820, compared with placebo, in male and female participants,18 to 80 years of age with ALS followed by an openlabel, longterm extension period. Study ACT16970 consists of 2 parts (A and B) as follows: Part A is a 24 week, double blind, placebo controlled part, preceded by a screening period of up to 4 weeks before Day 1. On Day 1 of Part A, participants will be randomized in a 2:1 ratio to the SAR443820 treatment arm or matching placebo arm as listed below: Treatment arm: SAR443820, BID Placebo arm: Placebo, BID Randomization will be stratified by the geographic region of the study site, region of ALS onset (bulbar vs other areas), use of riluzole (yes vs no), and use of edaravone (yes vs no). Participants will attend inclinic study assessments at baseline (Day 1), Week 2, Week 4, Week 8, Week 16, and Week 24, and will receive a phone call at Week 12 and Week 20. After successful completion of Part A, all participants will rollover to Part B. The Week 24 Visit is the end of Part A and the beginning of Part B. Part B is an open label, longterm extension period that starts from the end of Part A (Week 24) and continues up to Week 106. The objectives of Part B are to further determine the safety and efficacy of longterm SAR443820 treatment. The treatment assignment of participants in Part A will remain blinded to Investigators, participants, and site personnel until the end of Part B. Every participant will receive BID oral tablets of SAR443820 in Part B.

The purpose of this study is to evaluate the effectiveness, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.

The purpose of this study is to assess the effect of Reldesemtiv versus placebo on functional outcomes in Amyotrophic Lateral Sclerosis (ALS).

This study is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.

The purpose of this study is to assess the long-term safety and tolerability of Reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)

The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS.

The purpose of this study is to evaluate and compare the effectiveness of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS), based on the time from the randomization date in Study MT-1186-A02 to at least a 12-point decrease in Revised ALS Functional Rating Score (ALSFRS-R) or death, whichever happens first, over the course of the study or until oral edaravone is commercially available in that country.

This study aims to evaluate and compare the effectiveness of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale. 

This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include two components: a Single-Ascending-Dose (SAD) stage and a Multiple-Ascending-Dose (MAD) stage.

The purpose of this study is to evaluate the safety and effectiveness of NP001 for treating patients who have amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation.

The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.

The purposes of this study are to measure the impact of two doses of ALZT-OP1a (cromolyn) on neuro-inflammation by measuring plasma neuroinflammatory biomarkers over a 12-week treatment period, to evaluate the effects of ALZT-OP1a (cromolyn) on functional changes in subjects with mild-moderate stage ALS by using the ALS Functional Rating Scale-Revised (ALSFRS-R) to measure changes in function over a 12-week treatment period, to evaluate the safety of the ALZT-OP1a (cromolyn), and to determine the optimal dose selection of ALZT-OP1a (cromolyn) out of the two doses used in the study.

The purpose of this study is to assess safety, tolerability, and pharmacokinetics (PK) following multiple doses of ABBV-CLS-7262 in subjects with amyotrophic lateral sclerosis (ALS).

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.

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.

The primary objective of this study is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS). The secondary objective is to evaluate the pharmacokinectics or movement of drug throughout the body (PK) of BIIB078 in participants with C9ORF72-ALS.

The purpose of this study is to evaluate the clinical efficacy of nL-CHCHD-001 in motor function and survival in a single ALS participant., and to evaluate further the effects of nL-CHCHD-001 in slowing, halting, or reversing the rate of decline of clinical functioning and improvement in biomarkers of disease severity in a single ALS participant.

The purpose of this study is to collect CSF and blood samples that can be used in future research studies to identify potential biomarkers in blood and CSF collected in ALS patients. Biomarkers are non-genetic elements in your blood and CSF that may help us diagnose and monitor ALS more easily. There are no readily available sources of longitudinal CSF, plasma or serum samples from people with ALS, ALS-FTD or similar neurodegenerative disorders or their family members for use in the identification of potential ALS biomarkers. Future research will examine potential biomarkers in blood and CSF collected over time to see if they change over time and can be used to diagnose and monitor people with ALS.

The purpose of this study is to collect and study blood samples that can be used in current and future research studies to identify genetic risk factors in ALS and identify potential biomarkers in blood collected in ALS patients. Biomarkers are non-genetic elements in your blood that may help us diagnose and monitor ALS more easily. There are no readily available sources of longitudinal DNA, RNA, monocytes, serum or plasma from people with ALS, ALS-FTD or similar neurodegenerative disorders or their family members for use in the identification of potential changes in gene structure over time or biomarkers in ALS. Future research will examine genes associated with the risk of ALS and potential biomarkers in blood collected over time to see if they change over time and can be used to diagnose and monitor people with ALS. This information is important for development of new medications for the treatment of ALS.

The purpose of this study is to collect clinical data, serial blood samples for plasma, serum, DNA and RNA extraction, and a single time blood sample for peripheral blood monocytes for the preparation of stem cell lines and lymphocytes for the preparation of lymphoblastoid cell lines. There will also be a single time skin biopsy sample for tissue examination and preparation of skin fibroblasts from patients who have familial or sporadic ALS, ALS-FTD or similar neurodegenerative motor neuron disorders, and are participating in NINDS funded research projects in the Mayo Clinic ALS Center.

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.

This study is intended to obtain clinical information and establish a repository of DNA, RNA, peripheral blood monocyte, lymphocyte and skin tissue samples from people with ALS and related neurodegenerative motor neuron diseases, people with a family history of these conditions, and healthy people with no family history of these disorders. The samples will be used in future research to learn about how these disorders affect people, what causes these conditions, and how the investigators can tell when someone has this kind of disease. Future research may also include the generation of stem cells from stored blood cell and skin cell samples. Participants will not be paid for taking part in this study.

The purpose of this study is to evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale. Subjects will be allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks.

This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale. In this randomized, double-blind, placebo-controlled, parallel-group, multicenter study, subjects are allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks. The primary endpoint is slow vital capacity (SVC) at 12 weeks, with the impact on patient function assessed through 48 weeks, adjusted for patient outcome, using ALSFRS-R (combined assessment of function and survival, CAFS). Other important efficacy measures include time to respiratory events, clinical global impression (CGI), assessment of dyspnea using the Borg scale and sleep scales (Pittsburgh sleep quality index and Epworth sleepiness scale). Patient safety is monitored using conventional methods including adverse events, safety laboratory tests, vital signs and 12-lead EKG. Following screening and baseline visits, patients attend the clinic at 2, 4, 8, 12, 24, 36 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit is performed 14-25 days after the last study treatment administration. The study will be monitored by an independent data and safety monitoring board. A long-term extension study will be available for patients completing the study.

The purpose of this study is to make Tirasemtiv available to a limited group of patients who suffer from ALS, who participated in CY 4031 and CY 4033, and, in the opinion and the clinical judgment of the treating pPhysician, would benefit from continued treatment with Tirasemtiv

This study is intended to obtain clinical information and establish a repository of DNA, RNA, peripheral blood monocyte, lymphocyte and skin tissue samples from people with ALS and related neurodegenerative motor neuron diseases, people with a family history of these conditions, and healthy people with no family history of these disorders. The samples will be used in future research to learn about how these disorders affect people, what causes these conditions, and how the investigators can tell when someone has this kind of disease. Future research may also include the generation of stem cells from stored blood cell and skin cell samples. Participants will not be paid for taking part in this study.

The purpose of this study is to evaluate the potential to use TMS as a way of functionally assessing target engagement in an efficacy study of Perampanel as a treatment for ALS. The study will also determine if there is a correlation between Perampanel dose and TMS MT.

The clinical development program has demonstrated that Edaravone is a well-tolerated treatment that slows the loss of physical function in ALS.
However, as observed in prior clinical trials, ALS presentation and progression is extremely heterogeneous among patients. There is therefore an imperious need for biomarkers, which could act as reliable indicators for Edaravone’s effect on disease progression and help better elucidate Edaravone’s mechanism of action. Biomarkers can help explain empirical results of clinical outcomes by helping us understand the mechanistic molecular and cellular pathways to clinical response. Future applications of biomarker identification will include determining the feasibility and face validity of these biomarkers in a patient registry study conducted in patients going on Edaravone as part of their clinical treatment plan, incorporating identified biomarkers in a large controlled post-marketing commitment study required by FDA studying higher and more frequent dosing of intravenous Edaravone in ALS compared to usual dosing, and optimizing development and dosing scheme for non-intravenous Edaravone formulation.
The present protocol is designed to investigate a selected panel of biomarkers in patients with ALS, treated with Edaravone. Biomarkers of oxidative stress, inflammation and neurodegeneration will be explored. Epigenetic and protein biomarkers will also be investigated.

The purpose of this study is to test the clinical effectiveness of ibudilast in patients with ALS measured by NfL and change in ALSFRS-R slope.

The purpose of this study is to support and extend the results of the FORTITUDE-ALS clinical trial, a clinical trial of an investigational drug (reldesemtiv) for the treatment of amyotrophic lateral sclerosis (ALS).

The purpose of this study is to assess the functional mobility and self-reported satisfaction with the Xavier electromyography hands-free wheelchair control system in comparison with a standard joystick.