Clinical Trials

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 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).

This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm Pacing System device. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed.

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 study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function.

The primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx® Diaphragm Pacing System™ (DPS) with respect to survival.

The secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.

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

This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs). The autologous NurOwn® (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells

This is a multi-center, randomized, double blind, placebo controlled study to evaluate the safety and efficacy of autologous (self) transplantation of Neurotrophic factors-secreting Mesenchymal Stromal Cells (MSC-NTF, NurOwn™) in patients with ALS .

MSC-NTF cells are a novel cell-therapeutic approach which is expected to effectively deliver Neurotrophic factors, which are potent survival factors for neurons, directly to the site of damage.

The purpose of this study is to establish a biorepository and phenotyping database to investigate longitudinal changes in ALS subjects. Blood, including DNA and RNA, cerebrospinal fluid (CSF) and electrophysiologic measures will be collected every 6 months over 1 and a half years. The database and specimen repository will be made available to ALS researchers on a merit basis.

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

This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis (ALS). The study will also investigate the mean rate of change in the ALSFRS-R total score as an exploratory endpoint to help design future studies.

This study will enroll up to 40 patients and include an optional 28-week open-label extension period plus a 3-week treatment taper and 1-week follow up period. After completion of Week 8, patients enrolled in a treatment group that is considered safe and ...

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).

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. 

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.

The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of one patient who has amyotrophic lateral sclerosis (ALS).

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 purpose of this study is to confirm that levosimendan can significantly improve respiratory function measured by supine slow vital capacity (SVC) in amyotrophic lateral sclerosis (ALS) patients.

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 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 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 is a multicenter, multiple dose study to examine the effect of H.P. Acthar® (Acthar) on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS).

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).

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.

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 identify patients with a diagnosis of Amyotrophic Lateral Disease (ALS) or ALS variants who have laboratory markers of inflammation or autoimmunity. Presently, there is no effective therapy for treatment of ALS or ALS variants. Recent evidence suggests that some patients with ALS who have laboratory markers of inflammation may respond to therapies that modulate immune system function. The aim of this study is to determine what percentage of patients with ALS and ALS variants have positive laboratory markers of inflammation or autoimmunity. This study will also determine what percentage of patients with ALS or ALS variants ...

The goals of this study are to identify biomarkers that allow improved staging or prognosis of the disease, and through the discovery of previously unrecognized immune abnormalities in ALS we aim to eventually identify immune therapies that may provide benefit in ALS.

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 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 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 ...

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 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.

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 assess the long-term safety and tolerability of tirasemtiv in patients with ALS.

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 ...

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 ...

The purpose of this study is to evaluate the safety and effectiveness of investigational products for the treatment of 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 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 so that blood (plasma and blood cells) and/or extra spinal fluid (which was taken for clinical purposes) can be obtained and stored for later research on a large cohort of patients with progressive motor neuronopathies and neuropathies, many of which will be determined to suffer from Amyotrophic Lateral Sclerosis (ALS). 

The purpose of this study is to provide up to three intrathecal injections of NurOwn® (MSC-NTF cells, autologous Mesenchymal Stem Cells [MSC] Secreting Neurotrophic Factors [NTF]) given two months apart to patients who have completed all scheduled treatments and follow-up assessments in the BCT-002-US clinical trial. A bone marrow aspiration will be required as part of this program. It will take about 30 weeks for participants to complete the program.

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 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 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 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 ...

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 purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study.

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 ...

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 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 primary objective of this study is to assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD).

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 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 ...

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 ...

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. ...

This study is being done to collect skin samples from people with and without neurodegenerative and vascular disorders including Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), stroke and many others. We will use these skin samples to make and bank (store) a group of cells (cell line) called inducible pluripotent stem (iPS) cells.

The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)] experiencing muscle cramps. Participants will be asked to take two study products during the course of the study. One of these study products will be a placebo. Approximately 120 participants in approximately 30 study centers across the United States are expected to take part. Participants will be in the study for approximately 3 months and visit the study clinic 3 times.

The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)] experiencing muscle cramps. Participants will be asked to take two study products during the course of the study. One of these study products will be a placebo. Approximately 120 participants in approximately 30 study centers across the United States are expected to take part. Participants will be in the study for approximately 3 months and visit the study clinic 3 times.

This study proposes to identify the molecular defect in families with a clinical phenotype of “probable Inclusion Body Myositis” (“probable IBM”) but affecting  more than one family members, with or without family history of ALS.