Amyotropic Lateral Sclerosis (ALS) clinical trials at University of California Health

27 in progress, 15 open to eligible people

Showing trials for

  • AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

    open to eligible people ages 18 years and up

    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).

    at UCSD

  • BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

    open to eligible people ages 18-80

    The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that will enable people who have lost the ability to speak fluently to communicate via a computer just by trying to speak.

    at UC Davis

  • BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

    ““Turn thoughts into action” for people living with paralysis”

    open to eligible people ages 18-80

    The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with their thoughts.

    at UC Davis

  • Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)

    “Is your speech very hard to understand, or are you unable to talk because of a nerve or muscle problem?”

    open to eligible people ages 22-75

    The Connect-One Study is an early feasibility study to obtain preliminary device safety information for the Connexus Brain-Computer Interface (BCI). The Connexus BCI is intended to be used as: (1) an assistive communication device to decode imagined language correlates and speech for patients with impaired communication as a result of severe loss of voluntary motor control; and (2) to provide control of computer devices for individuals with severe loss of voluntary motor control of the upper extremity.

    at UC Davis

  • ECoG BMI for Motor and Speech Control

    open to eligible people ages 21 years and up

    Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurological disorders.

    at UCSF

  • Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

    open to eligible people ages 18-79

    The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to <80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.

    at UCSD

  • PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS

    open to eligible people ages 18-75

    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.

    at UCSD

  • PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers

    open to eligible people ages 18-80

    This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.

    at UCSF

  • Ranolazine in ALS

    open to eligible people ages 18 years and up

    The purpose of this study is to evaluate safety, effect on cramps, function and quality of life of ranolazine versus placebo for the treatment of ALS.

    at UCSF

  • Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis

    open to eligible people ages 18 years and up

    Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis

    at UC Irvine UCSD

  • VTx-002 in Participants With ALS

    open to eligible people ages 18 years and up

    PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS). Two escalating dose (low dose and high dose) cohorts are planned. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.

    at UCSD

  • ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)

    open to eligible people ages 18 years and up

    ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) represents the formalized integration of ARTFL (U54 NS092089; funded through 2019) and LEFFTDS (U01 AG045390; funded through 2019) as a single North American research consortium to study FTLD for 2019 and beyond.

    at UCLA UCSD UCSF

  • ASSESS ALL ALS Study

    open to eligible people ages 18 years and up

    The ALL ALS Clinical Research Consortium is establishing research to collect a wide range of samples, clinical information and measurements from Amyotrophic Lateral Sclerosis (ALS) symptomatic, ALS gene carriers and control cohorts. This consortium is being funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) and managed by two clinical coordinating centers (CCC) at Barrow Neurological Institute and Massachusetts General Hospital. The clinical sites are distributed across the country, and led by a group of collaborative principal investigators. Once data and samples are collected and harmonized, it will be made available to research community for future research into ALS and related neurological diseases. ASSESS protocol is specific for symptomatic ALS and control participants. This protocol includes both on-site and off-site(remote) participants. The participants will be followed for 24 months (2 years), and will include collection of medical history, clinical outcomes, and blood samples once in 4 months. Additionally, the participants will complete patient reported outcomes and speech recordings once a month. Participants who are coming into clinic may also provide optional Cerebrospinal Fluid (CSF) samples.

    at UC Irvine UCLA UCSD UCSF

  • Clinical Procedures to Support Research in ALS

    open to eligible people ages 18 years and up

    The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders.

    at UC Irvine

  • Target ALS Biomarker Study; Longitudinal Biofluids, Clinical Measures, and At Home Measures

    open to eligible people ages 18 years and up

    The goal of the study is to generate a biorepository of longitudinal biofluids-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. To accomplish these goals, we will enroll 800 Amyotrophic Lateral Sclerosis (ALS) patients and 200 healthy controls from sites globally, over a 5 year time frame. Additionally, speech and motor function and spirometry measures will be collected bi-weekly in a subset of participants. ALS participants will be asked to come to the clinic for 5 study visits approximately every 4 months. Healthy participants will be coming for 2 study visits with a 12-month interval between visits. These samples and clinical information will be stored in a de-identified manner and made available for investigators to use in future research studies.

    at UCSD

  • RAPA-501 Therapy of ALS Expanded Access Protocol

    Sorry, currently not accepting new patients, but might later

    RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).

    at UC Irvine UCSF

  • Three Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01, CNMAu8.EAP02, and CNMAu8.EAP06 for ALS

    Sorry, not accepting new patients

    The primary objective of the intermediate expanded access protocol is to provide access to the investigational product, CNM-Au8, to up to 300 people living with ALS (pALS). No formal clinical hypotheses are being evaluated with concurrent controls. Secondary objectives include assessment of the safety of CNM-Au8 treatment in pALS. Safety will be assessed through the frequency of serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) assessed as 'severe', discontinuations due to TEAEs, and laboratory abnormalities assessed as clinically significant during routine clinical monitoring (as applicable).

    at UC Irvine

  • FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

    Sorry, in progress, not accepting new patients

    The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).

    at UCSD

  • HEALEY ALS Platform Trial - Master Protocol

    Sorry, in progress, not accepting new patients

    The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

    at UC Irvine UCSD UCSF

  • Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

    Sorry, in progress, not accepting new patients

    This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.

    at UC Irvine

  • Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)

    Sorry, in progress, not accepting new patients

    This is a multicenter, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early-stage ALS (within 2 years of ALS symptoms onset). The study comprises a core double-blind (DB) 40-week treatment period followed by an open label extension (OLE).

    at UCSD UCSF

  • Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms

    Sorry, in progress, not accepting new patients

    In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation. One of the causes of ALS is a change (or mutation) in the SOD1 gene, which causes abnormal SOD1 protein to be made. This abnormal protein can cause the motor neurons in people with ALS to break down and die. When neurons are damaged, they release a protein called neurofilament light chain, also known as NfL. Previous studies have shown that NfL levels are higher in people with SOD1-ALS, even before any ALS symptoms appear. In this study, researchers will look at the effect of tofersen treatment in the earlier stages of the disease, even before ALS symptoms appear. Researchers believe that giving tofersen to patients as early as possible may slow the onset of ALS symptoms and the progression of the disease. This study will have 4 parts. In Part A, participants will have their health monitored and NfL levels tested on a monthly basis, but they will not receive any study drug. Tofersen or placebo will be given to participants in Parts B, while all participants will receive tofersen in Parts C and D. The main question that researchers want to answer is: • How long does it take for ALS symptoms to appear? Researchers will also learn more about: - How many participants develop ALS symptoms within 24 months of the start of Part B - Changes in participants' ability to perform their daily activities - Changes in participants' ability to breathe - How many participants die or need long-term breathing support - Changes in NfL levels in the blood - Changes in SOD1 protein levels in the fluid around the brain and spinal cord - How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug. The study will be done as follows: - Participants will first be screened to check if they can join the study. The screening period will be up to 6 weeks. Participants will join the study only after reading and signing an informed consent form, also known as an ICF. - In Part A, participants with presymptomatic ALS will not get any study drugs. Researchers will check the levels of NfL in their blood. Participants will be closely checked for any signs or symptoms of ALS. - Once a participant's NfL levels rise to a certain amount, they will move onto Part B. - In Part B, participants will be randomly assigned to get either tofersen or a placebo. Neither participants nor researchers will know who gets tofersen or the placebo during Part B. A placebo looks like the study drug but contains no medicine. - Participants will get the first 3 doses of tofersen once every 14 days. Afterwards, they will continue getting maintenance doses every 28 days. - All doses will be given through an intrathecal (IT) injection done through a lumbar puncture procedure. This involves injecting the drug into the fluid surrounding the spinal cord in the lower back. - Participants who develop ALS symptoms during Part B will move to Part C to receive tofersen. - Participants who develop ALS symptoms during Part A will move to Part D to receive tofersen. - In both Parts C and D, participants will know they are receiving tofersen. - Throughout the study, doctors will measure any changes to the participants' function and quality of life using different questionnaires. Doctors will also take blood and urine samples. They will also check the participants' overall health and ask how they are feeling. - Participants will have a follow-up visit 28 days after their last dose of study drug. - Participants will be in this study for up to about 6.5 years.

    at UCSD

  • MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS

    Sorry, in progress, not accepting new patients

    A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.

    at UC Irvine

  • MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)

    Sorry, not yet accepting patients

    The goal of this two-part clinical trial is: 1) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period. Researchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS. Participants will: Receive NurOwn or a placebo every 8 weeks for 24 weeks. After that, every participant will receive NurOwn every 8 weeks for an additional 24 weeks. They will visit the clinic approximately every 8 weeks for checkups and tests.

    at UCSD UCSF

  • Intermuscular Coherence as a Biomarker for ALS

    Sorry, not currently recruiting here

    The specific aims of this study are to: 1. Determine if a painless and quick measurement of muscle activity using surface electrodes can help with the diagnosis of ALS. Specifically, we ask if a measure of intermuscular coherence (IMC-βγ), when added to current diagnostic criteria (Awaji criteria), can differentiate ALS from mimic diseases more accurately and earlier than currently possible. 2. Characterize IMC-βγ in neurotypical subjects by age, sex, race, and ethnicity. 3. Follow a cohort of ALS patients longitudinally to determine if IMC-βγ changes with ALS disease progression and whether such changes correlate with functional and clinical scores, or survival.

    at UC Irvine

  • Phenotype, Genotype & Biomarkers in ALS and Related Disorders

    Sorry, in progress, not accepting new patients

    The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.

    at UCSD

  • PREVENT ALL ALS Study

    Sorry, in progress, not accepting new patients

    The ALL ALS Clinical Research Consortium is establishing research to collect a wide range of samples, clinical information and measurements from Amyotrophic Lateral Sclerosis (ALS) symptomatic, ALS gene carriers and control cohorts. This consortium is begin funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) and managed by two clinical coordinating centers (CCC) at Barrow Neurological Institute and Massachusetts General Hospital. The clinical sites are distributed across the country, and led by a group of collaborative principal investigators. Once data and samples are collected and harmonized, it will be made available to research community for future research into ALS and related neurological diseases. PREVENT protocol is specific for asymptomatic participants who are genetically at risk for ALS. The participants will be followed for up to 36 months (3 years), and will include 4 in-person on-site visits once a year and 6 off-site(remote) visits once in 4 months. The study includes collection of medical history, clinical outcomes, and blood samples once in 4 months. Additionally, the participants will complete patient reported outcomes and speech recordings once in 4 months. Participants may also provide optional Cerebrospinal Fluid (CSF) samples.The participants may also opt into a sub-study if they are interested in genetic testing for ALS causative genes. The sub-study will involve a minimum of 3 visits over a course of 2-3 months. This will include a screening/pre-test genetic counseling visit, a return of genetic results and a post-test counseling visit.

    at UC Irvine UCSD UCSF

Our lead scientists for Amyotropic Lateral Sclerosis (ALS) research studies include .

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