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

22 in progress, 10 open to eligible people

Showing trials for
  • BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

    open to eligible people ages 18 years and up

    The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).

    at UCSD

  • 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

  • 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

  • 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

  • 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)

    open to eligible people ages 10 years and up

    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

  • 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

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

    open to eligible people ages 18 years and up

    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

  • 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

  • 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

    Brief Summary: The goal of the 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. To accomplish these goals, we will enroll 200 Amyotrophic Lateral Sclerosis (ALS) patients and 80 healthy controls from multiple sites, over a 5 year time frame. Additionally, speech measures will be collected on weekly basis at home for all participants. The measurements are performed using a speech recording application installed on their personal device. For a subset of both ALS and healthy participants, we will also collect at-home vital capacity on a weekly basis. It is expected that increased frequency data sampling of these outcome measures will help in better tracking of disease progression. Biofluids and clinical information are collected over a 20-month time frame for each individual enrolled in the research study. ALS participants will be coming to clinic for 5 study visits with a 4-month interval between visits. 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

  • Fosigotifator in Patients With Amyotrophic Lateral Sclerosis

    Sorry, in progress, not accepting new patients

    Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects will receive Fosigotifator.

    at UC Irvine

  • Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)

    Sorry, in progress, not accepting new patients

    A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.

    at UCSF

  • TPN-101 in Patients With C9ORF72 ALS/FTD

    Sorry, in progress, not accepting new patients

    This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

    at UC Irvine UCSD UCSF

  • Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

    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 UCSF

  • AMX0035 for Amyotrophic Lateral Sclerosis Treatment

    Sorry, in progress, not accepting new patients

    The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS

    at UC Irvine

  • PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

    Sorry, in progress, not accepting new patients

    This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.

    at UC Irvine

  • 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, accepting new patients by invitation only

    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

  • Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)

    Sorry, in progress, not accepting new patients

    REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and protein biomarkers will also be investigated.

    at UC Davis UCLA UCSF

  • RAPA-501 Therapy for ALS Expanded Access Protocol

    Sorry, not accepting new patients

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

    at UC Irvine UCSF

  • Two Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01 and CNMAu8.EAP02 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

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

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