Amyotropic Lateral Sclerosis (ALS) clinical trials at University of California Health
24 in progress, 14 open to eligible people
A Study of 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
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
open to eligible people ages 18 years and up
The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS). The ALSpire Study consists of two parts: - Part 1: 6-month placebo-controlled study. During Part 1, participants are randomly assigned to receive either BIIB105 or placebo in a 3:1 or 2:1 ratio (depending on the participant's assigned Cohort). - Part 2: 2-year long-term open-label extension. During Part 2, all participants receive BIIB105. The objectives of the study are to evaluate: - The safety and tolerability of BIIB105 in people with ALS - What the body does to BIIB105 (also called "pharmacokinetics") - What BIIB105 does to the body (also called "pharmacodynamics") - Whether BIIB105 can slow the worsening of clinical function
at UCSD
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 12 years and up
The primary purpose of this study is to evaluate the clinical efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
at UCSD
A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)
open to eligible people ages 18 years and up
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
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
Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS
open to eligible people ages 18-80
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
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)
open to eligible people ages 18-80
This is a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, 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 open label, longterm extension period. Study ACT16970 consists of 2 parts (A and B) as follows: Part A is a 24week, 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), use of edaravone (yes vs no) and use of the combination of sodium phenylbutyrate and taurursodiol (named Relyvrio in the United States of America [USA] and Albrioza in Canada) (yes vs no). Participants will attend in clinic 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. All ongoing participants in Part A 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, except those who discontinued Investigational Medicinal Product (IMP) treatment permanently in Part A , will receive BID oral tablets of SAR443820 in Part B.
at UC Irvine UCSD
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
open to eligible people ages 18-80
This study will assess the effects and safety of PTC857 treatment in participants diagnosed with ALS.
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
Intermuscular Coherence as a Biomarker for ALS
open to eligible people ages 20-80
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
Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)
open to eligible people ages 18 years and up
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
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
A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis
Sorry, in progress, not accepting new patients
ABBV-CLS-7262 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 ABBV-CLS-7262.
at UC Irvine
A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
Sorry, in progress, not accepting new patients
A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.
at UCSF
A Phase 2 Study to Evaluate Safety of 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
A Phase 2a Study of 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
A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis
Sorry, in progress, not accepting new patients
This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL343 in participants with amyotrophic lateral sclerosis (ALS)
at UCSD
Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS
Sorry, in progress, not accepting new patients
To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48:
at UC Irvine UCLA UCSD
HEALEY ALS Platform Trial - Master Protocol
Sorry, not currently recruiting here
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
Phase III Trial of 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
Intermediate Expanded Access Protocol (EAP) CNMAu8.EAP02
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
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
Our lead scientists for Amyotropic Lateral Sclerosis (ALS) research studies include Peter Ljubenkov, MD Howie Rosen, MD Karunesh Ganguly, MD, PhD Ali A Habib, MD Namita Goyal, MD John Ravits Adam Boxer, MD, PhD.
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