WashU Clinical Trials

Observational studies and therapeutic trials are vital to ALS research. By learning more about the disease and our patients, we can advance the investigation of future treatments.

ASSESS ALL ALS is part of the newly established ALL ALS Research Consortium, an NIH-funded initiative to advance ALS research. This study collects clinical data and blood samples from people with ALS which will be made broadly available to the research community. In-person and remote participation options are available.

Principal Investigator: Timothy Miller, MD, PhD

Participants: Open to patients with ALS and healthy controls

Also a part of the ALL ALS Research Consortium, the PREVENT study will provide genetic testing of known ALS causative genes and perform longitudinal evaluations for people with genetic ALS in their family. This study will identify biomarkers and early disease measures of disease onset. Genetic testing can be done without revealing results. The study involves annual in-person visits plus remote visits every 4 months.

Principal Investigator: Timothy Miller, MD, PhD

Participants: Open to first-degree relatives of patients with dominantly-inherited ALS or FTD

This study will collect a wide range of samples, clinical information, and at-home measurements for future ALS and neurologic disease research. Samples such as blood, cerebral spinal fluid (CSF), and urine will be collected over a period of 12 to 16 months from both healthy volunteers and ALS participants, respectively. All samples collected will be stored in a central repository and made available to the wider research community in an effort to identify biomarkers and treatment targets for ALS. Participants will also undergo speech and breathing measurements at home via an app on a smartphone or tablet.

Principal Investigator: Timothy Miller, MD, PhD

Participants: Open to patients with ALS and healthy volunteers

This project will collect one blood sample from which your DNA is extracted and banked in our DNA database. The database enables researchers to perform research into the genetic causes of ALS.

Principal Investigator: Timothy Miller, MD, PhD

Participants: Open to patients with Neuromuscular Disorders, ALS and healthy controls

This project will collect blood samples over time to help identify changes in biomarkers which may be therapeutic targets. Blood collections can be done at regular clinic visits.

Principal Investigator: Timothy Miller, MD, PhD

Participants: Open to patients with Neuromuscular Disorders, ALS and healthy controls

CSF is fluid that bathes the brain and spinal cord and protects them from damage. Since CSF is so closely involved in the nervous system, this fluid provides one of the most useful pictures of changes that occur during neurodegeneration in ALS. This project involves collection of CSF via a lumbar puncture. This procedure takes approximately two hours and can be scheduled at your convenience.

Principal Investigator: Timothy Miller, MD, PhD

Participants: Open to patients with Neuromuscular Disorders, ALS and healthy controls

This study is looking at clinical factors that lead to the best quality of life outcomes post feeding tube placement. It is open to persons with ALS who are planning to get a feeding tube within the next few months for any reason. It consists of 3 sets of questionnaires to be administered pre- and post-feeding tube placement. This study can be conducted inperson or remotely via email or phone/zoom calls. Patients do not need to be having the feeding tube placed at BJH in order to participate.

Principal Investigator: Sean Smith, MD

Participants: Open to ALS patients who plan to get a feeding tube

This is an observational study done in conjunction with a clinical EMG to determine whether surface electromyography (sEMG), a method of measuring muscle activity through the skin, is a valid tool to diagnose ALS at an early stage as well as assess the disease progression in future clinical trials. The study, which involves placing electrodes on participants’ arms and legs and then contracting certain muscles, takes approximately one hour to complete.

Principal Investigator: Sean Smith, MD

Participants: Open to patients who are undergoing a clinical EMG

This is a Phase 2/3 multi-center, multi-drug clinical trial to evaluate the safety and efficacy of investigational products for the treatments of ALS. Eligible participants will be randomized to one of multiple regimens (or research drugs). Within each arm, each participant will be randomized to either drug or placebo. The treatment will last a maximum of 24-weeks with an optional open label extension. For each drug, study visits which will occur every 4 weeks either in clinic (seven clinic visits) or phone-based. Once a participant has completed a drug, they can be re-randomized back into the study for a different drug.

Principal Investigator: Timothy Miller, MD, PhD

Participants: For patients with all forms of ALS, please contact us to see if you meet criteria

ATLAS is a Phase 3 trial of tofersen in pre-symptomatic SOD1 gene carriers. Tofersen is an antisense oligonucleotide (ASO) drug designed to reduce levels of SOD1 protein. Participants initially take part in a natural history run-in period during which a biomarker called neurofilament light chain (NfL) is measured monthly via blood draws either at home or in clinic. If the NfL level reaches a certain threshold, treatment with tofersen/placebo is initiated. Participants who develop ALS during the trial may be eligible to enroll in an open label extension. The trial will enroll approximately 150 participants worldwide.

Principal Investigator: Robert Bucelli, MD, PhD

Participants: Open to adults with a confirmed SOD1 mutation who are pre-symptomatic for ALS

FUSION is a Phase 1-3 trial of ION363. ION363 is an antisense oligonucleotide (ASO) drug in development for patients with a rare genetic form of ALS caused by mutations in the FUS gene. The trial is a global, multi-center study in up to 64 patients. Part one of the trial will consist of patients randomized to receive a multi-dose regimen of ION363 or placebo for 61 weeks, followed by part two, which will be an open-label period in which all patients in the trial will receive ION363 for 85 weeks.

Principal Investigator: Sean Smith, MD

Participants: Open to patients with ALS who carry a FUS genetic mutation