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Amyotropic Lateral Sclerosis (ALS) clinical trials at UCSD

9 in progress, 5 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).

    La Jolla, California and other locations

  • 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.

    La Jolla, California and other locations

  • 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).

    La Jolla, California and other locations

  • 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.

    San Diego, California and other locations

  • 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.

    San Diego, California and other locations

  • 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).

    La Jolla, California and other locations

  • Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation

    Sorry, in progress, not accepting new patients

    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: up to 3-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

    La Jolla, California and other locations

  • 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)

    San Diego, California and other locations

  • 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.

    San Diego, California and other locations

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

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