Clinical Trials

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

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 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 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 purpose of this study is to assess the efficacy of palliative care interventions in improving the quality of life for ALS patients and their caregivers. 

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

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 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 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.

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

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 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 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 assess the functional mobility and self-reported satisfaction with the Xavier electromyography hands-free wheelchair control system in comparison with a standard joystick.

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.

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

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.

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 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 purpose of this study is to test the effects of PHENOGENE-1A, which is the treatment under investigation in this study. This research will investigate if PHENOGENE-1A can help people with ALS by measuring their function using the ALS Functional Rating Scale Revised (ALSFRS-R), measuring lung function using pulmonary function tests (PFTs), such as forced vital capacity (FVC), and measuring neuro-inflammatory biomarkers in the blood.

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 ...

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 evaluate the safety and effectiveness of investigational products for the treatment of ALS.

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 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 ...

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 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.

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.

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.