Amyotropic Lateral Sclerosis (ALS) clinical trials at UCSD

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).

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

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)

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.