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Amyotropic Lateral Sclerosis (ALS) clinical trials at UCSD

11 in progress, 9 open to eligible people

Showing trials for
  • A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD

    open to eligible people ages 18 years and up

    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

  • A Study of BIIB067 When 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 BIIB067 when initiated 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 of BIIB067 and to evaluate the effect of BIIB067 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

  • 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

    Part 1: The primary objective is to evaluate the safety and tolerability of BIIB105 in participants with ALS or poly-CAG expansion (polyQ)-ALS. The secondary objectives are to assess the pharmacokinetic (PK) profile in serum and cerebrospinal fluid (CSF), and to evaluate the biomarker effect of BIIB105 in participants with ALS or polyQ-ALS. Part 2: The primary objective is to evaluate the long-term safety and tolerability of BIIB105 in participants with ALS or polyQ-ALS. Parts 1 and 2: The primary objective is to evaluate the long-term safety and tolerability of BIIB105 in participants with ALS or polyQ-ALS. The secondary objectives are to assess the long-term PK profile of BIIB105 in serum and CSF of participants with ALS or polyQ-ALS; to evaluate the long-term biomarker effect of BIIB105 in participants with ALS or polyQ-ALS; to assess the impact of earlier initiation of BIIB105 (i.e., at start of Part 1) compared with delayed initiation of BIIB105 (i.e., at start of Part 2) on biomarkers; to evaluate the long-term effect of BIIB105 on measures of clinical function and to assess the impact of earlier initiation of BIIB105 (i.e., at start of Part 1) compared with delayed initiation of BIIB105 (i.e., at start of Part 2) on measures of clinical function.

    La Jolla, California and other locations

  • 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).

    La Jolla, California and other locations

  • 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.

    La Jolla, California and other locations

  • Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

    open to eligible people ages 18-75

    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:

    La Jolla, California and other locations

  • 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.

    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

  • 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)

    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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