Clinical Trials at Washington University

Click on the tabs below to learn about the Miller Lab’s current clinical studies for people with ALS, PLS, PMA, other neuromuscular diseases, asymptomatic ALS gene carriers, and healthy control subjects.

Answer ALS

This research initiative is the largest of its kind in the history of ALS and aims to establish a comprehensive repository of biofluids and clinical data in order to enable further ALS research.

Participants: Persons with ALS and healthy controls.

Background: The purpose of this research study is to create a large repository of cells called induced pluripotent stem cells (iPSCs), bio-fluid samples (blood and spinal fluid (optional), and cell lines for ALS gene identification. This will be combined carefully with collected measures of the pattern of the symptoms people with ALS have and how these change over time.

What takes place in the study: 5 study visits to include the following: blood draws, muscle strength testing (HHD), questionnaires and breathing test. There is also an optional lumbar puncture.

View study flier

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Cerebrospinal Fluid Biorepository

Cerebrospinal fluid (CSF) is a highly valuble resource for researchers at Washington University and enables a wide variety of research. This study is gathering CSF from people with neuromuscular diseases and from control participants for use in other research studies.

Participants: Healthy volunteers and those diagnosed with a disease of the nerve, muscle or brain. Participants must be 45 to 75 years old, must have a BMI of less than or equal to 35, must not have a bleeding disorder, and must not be taking blood thinners.

Background: Cerebrospinal fluid (CSF) is a clear liquid which bathes the brain and spine. Our researchers gather, bank and study the CSF of individuals with neurodegenerative disorders and healthy volunteers. The CSF provides a valuable comparison which helps us to understand, diagnose and treat neurologic diseases.

What takes place in study: Participation will take approximately 3 hours. There will be about 2 or 3 study visits. There will be a physical exam, spinal tap, and 2 blood draws. Risks will be discussed with volunteers as part of the informed consent process. Participants will receive $200 for completing all parts of the study.

New! Patients with sporadic ALS are needed for this study. View flier here

View study flier for healthy controls

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Cross-Sectional ALS Biofluid Study (CABB)

CABB aims to establish a biofluid repository from people with ALS and control participants to support research and identify biomarkers of ALS and other diseases.

Participants: Persons with ALS, PLS, or PMA diagnosed by a neurologist; asymptomatic ALS gene carriers; and a comparison/control group (do not have ALS, PLS, PMA, or a gene known to cause ALS. These people may have other neurological diseases, such as Kennedy’s Disease or Paralytic Polio.)

Background: The purpose of the study is to extend and enlarge a biofluid repository of blood, plasma, serum, DNA, urine, and cerebrospinal fluid (CSF) collected from people with ALS and control participants to support research and identify biomarkers of ALS and other diseases. The eventual goal is to enroll a total of up to 1,000 study participants.

Recent advances in biomarker discovery in Alzheimer’s disease were based on precisely such a large-scale biofluid repository. Once specific ALS biomarkers have been discovered, these could be used to make earlier diagnoses, monitor disease progression, design new therapies, or test drug efficacy in clinical trials.

What takes place in study: After the initial visit, optional follow-up visits will occur at approximately 6 month intervals for one year. Recording of clinical measures associated with ALS, for example, measures of breathing and blood draws, will occur. All of this can take place during the normal clinic cisit. Though not mandatory, undergoing a lumbar puncture to obtain CSF is encouraged.

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Dominantly Inherited ALS Network (DIALS)

This project aims to establish a supportive platform for people at risk of inheriting ALS and will examine biomarkers and pre-disease progression measures to learn more about families in which ALS is genetically inherited.

Participants: People at risk for inherited forms of ALS and who are not symptomatic.

Background: This project aims to establish a mechanism for genetic testing and research involvement of people at risk for inherited forms of ALS. The study will be conducted over a 2-year period at Massachusetts General Hospital and Washington University, and will involve both single and multi-center hypothesis-driven projects, designed to assess emerging outcome measures and biomarker candidates in the asymptomatic gene carrier population. The overall goal of the DIALS network is to aggressively promote collaboration to define the point of clinical conversion in ALS, to complement the next level of therapeutics targeting early disease.

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Evaluation of Biomarkers in Blood

This biomarker study is collecting biofluids from people with neuromuscular diseases and control participants to probe for ALS-specific biomarkers, which can be used to track disease progression.

Participants: People with neuromuscular diseases OR normal healthy controls.

Background: Understanding the changes in blood that occur during disease helps us better understand the disease and better design new therapies. These changes that occur during disease are often called “biomarkers” in that they mark or identify or are associated with the disease. To understand changes during disease, we also need to understand normal or “controls” without the disease.

What takes place in study: Serial blood draws at each clinic visit (or scheduled through an outside clinic or at-home visit)

View study flier

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Genomic Translation of ALS Clinical Care (GTAC)

GTAC will analyze biofluids and clinical data from ALS patients with the goal of understanding the genes underlying ALS development and progression.

Participants: Persons with ALS (defined by EEC), primary lateral sclerosis (PLS), and progressive muscular atrophy.

Background: Across the United States and Scotland, this study will enroll and actively follow 1500 ALS patients with current clinic contact. This rich, clinical dataset will be paired with the analysis of collected blood samples (e.g. DNA) to clarify the complex effects large networks of genes have on the risk and clinical course of ALS. This project will link clinical, treatment, and outcome information to genetics in order to better understand the underlying manner of ALS development. The resulting dataset will be made openly and freely available to other researchers.

View study flier

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Phase II Study of AMX0035 (Amylyx)

Amylyx is a Phase II study that will test the effectiveness of two experimental drugs that have been tested separately before. Amylyx asserts that the combination of these compounds will have a better effect on ALS disease progression.

NOT YET RECRUITING

Participants: Persons with sporadic or familial “definite” ALS (as defined by El Escorial criteria).

Background: This is a multicenter, randomized, double-blind, parallel-group, placebo-controlled study of AMX0035 in 132 subjects with ALS. There will be two study arms. 132 patients will be consented and will be randomized 2:1 to receive study drug or placebo.

What takes place in study: For the first three weeks patients, participants in the active arm will receive 3g PB twice a day and 1g TUDCA once a day orally and then increase to twice a day orally for the remainder of the 24 week study period. Participants in the placebo arm will complete the same regimen with a taste-matched placebo. AMX0035 will be presented as a 4 gram sachet to be suspended in water and taken with a glass of water twice a day before a meal. Subjects will receive 24 weeks of medication.

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Phase III Study of AB1010 (Masitinib)

Masitinib is an experimental drug that is expected to have a positive effect on symptoms associated ALS and the degenerative process. This is a Phase III randomized, placebo-controlled trial.

NOT YET RECRUITING

Participants: Persons with sporadic or familial ALS, defined by “clinically probable” or “definite” ALS with El Escorial criteria.

Background: This study is a prospective, multicenter, randomized, double-blind, placebo-controlled, parallel group, phase 3 study to compare the efficacy and safety of AB1010 in combination with riluzole versus placebo in combination with riluzole for the treatment of patients suffering from ALS.

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

SOD1 Half-life Study in SOD1 ALS Patients

The goal of this study is to measure the half-life of SOD1 protein in ALS patients with SOD1 mutations. This information will aid in planning clinical trials in this population, and will increase the likelihood of success of SOD1-targeting therapies.

Participants: Persons with ALS and healthy controls.

Background: SOD1 is a protein that is implicated in certain types of ALS and may also serve as an important pharmacodynamics marker, which is a tool investigators can use to assess whether a new therapy is working in ALS patients. Understanding the half-life of the SOD1 protein in cerebral spinal fluid in both ALS and controls will help us in planning clinical trials for ALS patients with SOD1 mutations, and will increase the likelihood of success of therapies designed to lower or change levels of SOD1.

What takes place in study: 10-day controlled diet, blood draws and 4 lumbar puncture procedures to obtain CSF.

New! Patients with sporadic ALS are needed for this study. View flier here

View study flier

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

Understanding C9ORF72 Related ALS

This is a natural history study of ALS patients with C9ORF72 mutations. Natural history studies allow clinicians to better plan clinical trials and also provides researchers with a valuable resource of biofluids for use in future research.

New! View the Northeast Amyotrophic Lateral Sclerosis (NEALS) Consortium Educational Webinar featuring this study.

Participants: People with a genetic form of ALS caused by a mutation in C9ORF72

Background: For some people, ALS is caused by a mutation in the C9ORF72 gene. We are interested in better understanding ALS in this subset of patients and how C9ORF72-related ALS differs from other forms of ALS, such as SOD1-related ALS. This information is helpful when planning clinical trials in the C9ORF72 population.

What takes place in study: Recording of clinical measures associated with ALS, for example measures of breathing and blood draws. All of this can take place during the normal clinic visit. Though not mandatory, undergoing a lumbar puncture to obtain cerebral spinal fluid (CSF) is encouraged.

View study flier

Contact Information:
neuroclinicalstudies@neuro.wustl.edu
314-362-6159

 

Make A Difference