Neuromuscular Disease Trials

We are only recruiting patients who have not started their edaravone treatment.

Location of study: Les Turner ALS Center at Northwestern Medicine, 259 E. Erie St., Lavin 19, Chicago, IL 60611.

In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.

The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.

Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.

The following regimens are active in the trial:

Regimen A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8 Regimen D - Pridopidine Regimen E - Trehalose

New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.

This is a non-interventional (no study drug), natural history study of patient with primary lateral sclerosis (PLS).

The purpose of this study is to to develop a natural history dataset and biorepository of early PLS and well-established PLS cases for future clinical trials. The study will also evaluate differences in disease progression in early PLS and well-established PLS cases.

Patients will be enrolled over 24 months and complete assessments in person and over the phone.

Tofersen (also called BIIB067) is currently being evaluated for the treatment of adults with familial ALS associated with a mutation in the SOD1 gene. The optimal timing for initiation (i.e., prior to or after the emergence of clinically manifested disease) of tofersen is unknown. This study will evaluate the impact of initiating tofersen based on biomarker evidence of disease activity, prior to the emergence of clinical symptoms or signs that definitively indicate ALS.

This study will look at the long-term safety and effect (how well it works) of efgartigimod in people with active idiopathic inflammatory myopathy, also known as myositis. The study will include adult participants with different forms of myositis: dermatomyositis, immune-mediated necrotizing myopathy, and polymyositis (including antisynthetase syndrome). Efgartigimod is being tested in this study, it is the same study drug tested in ARGX-113-2007. It is the same study drug tested in the ARGX-113-2007 study. It is a piece of a human antibody modified to bind better to a specific protein called FcRn. Antibodies are proteins that fight and prevent infections. FcRn helps maintain antibody levels. Antibody levels are reduced after efgartigimod binds to FcRn. This means that the levels of antibodies attacking your own body are also reduced. IRB #: STU00220056-MOD0004 Approved by NU IRB for use on or after 3/31/2025 through 2/16/2026. Permission to Take Part in a Human Research Study ARGX-113-2011 Participant Informed Consent Document US version 4.0 12Feb2025 NU site version date: 26Feb2025 Page 2 of 13 HRP-592 / v.11-10-2023 Efgartigimod is similar to antibodies naturally present in the human body. It is called a “biological” drug because it is a piece of protein produced in cells of mammals. Efgartigimod may help improve myositis. Efgartigimod has been approved by the Food and Drug Administration (FDA) as a treatment for adult patients with myasthenia gravis, a rare disease causing muscle weakness. It is considered experimental because health authorities have not approved it for myositis. Everyone will get efgartigimod during the treatment period

The aim of this study is to measure the efficacy (how well it works) and safety of

efgartigimod compared with placebo (inactive substance) in participants with ocular

myasthenia gravis (oMG). The study will also measure the long-term efficacy, safety, and

tolerability of efgartigimod.

The primary purpose of this study (also referred to as “PREVENT”) is to collect a wide range of biofluid samples (cerebral spinal fluid and blood), clinical information, and other health and well-being information from individuals who are genetically at risk for ALS or a related disorder, Frontotemporal Dementia (FTD). This will help us better understand ALS and how the disease progresses by looking for measurable differences that will help us identify how and when the body changes in response to ALS causative gene variants.

Samples of blood and fluid surrounding the spinal cord and brain, known as cerebral spinal fluid (CSF), collected during this study will be stored in a secure, central location, called a biorepository. Other data that are collected will be stored in a secure central data repository. The data and samples will be maintained for many years and will be made available to researchers for use in future studies. We hope the information we collect will guide the direction of future research and lead to more informative, targeted drug development for halting, repairing, and/or preventing ALS.

We are doing the research to learn about the safety and tolerability of digoxin in individuals with ALS and also to better understand if digoxin has an effect on slowing neurodegeneration in ALS. Digoxin is approved by the U.S. Food and Drug Administration (FDA) to treat heart failure and atrial fibrillation in adults. Digoxin is not approved by the FDA to treat ALS.