What study is right for you?
Contact us at (1-844) ALS-Care or ALS@wustl.edu.

Observational Studies

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

Cerebrospinal Fluid (CSF) Repository

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.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with ALS and healthy controls

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


The DIALS (Dominant Inherited ALS) Network

This 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. Study visits are every 6 months up to 5 years.

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Principal Investigators: Timothy Miller, MD, PhD

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

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


Symptom Monitoring Application in Real Time (SMART) ALS

A pilot study focused on the feasibility and sensitivity of a smartphone application to monitor and detect ALS progression over time. Participants will perform a series of neurocognitive tasks at two in-person research visits during a 12-month study period. They will also be enrolled in a smartphone application (available for iOS and Android) that can track activity levels over twelve months. The goal of this study is to develop new diagnostic tools that could detect early signs and symptoms of ALS.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with ALS and healthy controls

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


MRI-ALS Study

This study is looking at a new Magnetic Resonance Imaging (MRI) technique to determine whether it may be possible to measure upper motor neuron loss in the area of the brain preferentially affected by ALS. Standard MRI techniques are unable to detect changes in this region and this study aims to determine whether this test could be used for diagnostic purposes. Total visit time is approximately 2 hours, which will include a 1-hour MRI scan, a brief exam and study questionnaires. Participants will need to meet certain eligibility requirements in order to participate. Please contact our coordinators if you are interested in participating and we can help determine your eligibility. 

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Principal Investigators: Robert Bucelli, MD, PhD

Participants: Open to patients with all forms of ALS

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


DNA Repository

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.

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Principal Investigators: Chris Weihl, MD, PhD

Participants: Open to patients with ALS and healthy controls

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


Radicava Biomarkers (REFINE) – Open for Enrollment

This is a prospective, observational, longitudinal, multicenter study. Participants will be followed for up to 24 weeks (through 6 cycles) for blood and urine samples. The study will investigate a selected panel of biomarkers in patients with ALS and treated with Radicava.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with ALS scheduled to receive Radicava

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu

Therapeutic Trials

HEALEY ALS Platform Trial

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 three regimens (or research drugs). The three regimens currently being offered at Washington University are Zilucoplan, Verdiperstat, and CNM-Au8. 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.

The three regimens being offered at Washington University in St. Louis are:

Regimen A: Trial of Zilucoplan
This regimen is being conducted to see if zilucoplan is safe and effective in people diagnosed with ALS. This medication works by inhibiting tissue damage caused by the immune system.
Developed By: Ra Pharmaceuticals

Regimen B: Trial of Verdiperstat
This regimen is being conducted to see if verdiperstat is safe and effective in people diagnosed with ALS. This medication works by reducing neural inflammation.
Developed By: Biohaven Pharmaceuticals

 Regimen C: Trial of CNM-Au8
This regimen is being conducted to see if CNM-Au8 is safe and effective in people diagnosed with ALS. This medication provides an energetic assist to impaired motor neurons and helps improve their ability to function normally.
Developed By: Clene Nanomedicine

For a more details, please visit:
https://www.massgeneral.org/neurology/als/research/first-platform-trial-treatments

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Principal Investigators: Timothy Miller, MD, PhD

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

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


Phase 1 Trial of BIIB100 (Oral Small molecule inhibitor of XPO1) – Open for Enrollment

This is a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of a single dose of BIIB100 in adults with ALS. Approximately 40 participants will be enrolled over 10 US sites. The study will last up to 6 weeks and involves a screening visit, a two-night stay in the clinical research unit, and 2 follow-up visits.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with ALS without an implanted port/catheter

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


Phase 1 Trial of BIIB078 (C9orf72 ASO)

This is a Phase 1, randomized, double-blind, placebo-controlled evaluation of the safety and tolerability of BIIB078 in 60 patients with C9orf72-ALS over 20 sites. BIIB078 is an antisense oligonucleotide drug designed to reduce levels of C9orf72 in ALS patients with mutations in the C9orf72. The study will last approximately 40 weeks, with a 6-week screening period, a 12-week treatment period and 22-week follow-up.

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Principal Investigators: Robert Bucelli, MD, PhD

Participants: Open to patients with ALS that carry C9orf72 Genetic Mutation 

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


Phase 3 Trial of BIIB067 (VALOR)

Biogen Idec and Ionis Pharmaceuticals are sponsoring this Phase 3 randomized, double-blind clinical trial of BIIB067. BIIB067 is an antisense oligonucleotide drug designed to reduce levels of SOD1 protein in ALS patients with mutations in SOD1. This study builds on the Phase I/II trial led by Dr. Robert Bucelli. 60 patients with ALS will be enrolled in the trial throughout the United States, Canada, and Europe. There is also an open label extension study for patients who have completed the trial. The study will last approximately 36 weeks, consisting of a 4-week screening period, a 24-week treatment period (8 doses of BIIB067) and at least 1 follow-up visit.

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Principal Investigators: Robert Bucelli, MD, PhD

Participants: Open to patients with ALS that carry SOD1 Genetic Mutation

VALOR Study Website:
https://www.alsvalorstudy.com/

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu


Sinemet (Carbidopa/levodopa) in ALS and PLS

This is a placebo-controlled, double-blind, randomized crossover study. It will evaluate the effect of carbidopa/levodopa on spasticity in ALS and PLS patients using questionnaires about symptom severity as well as tests for upper and lower body function. This pilot study at Wash U will last a total of 6 weeks, where participants will be treated for 3 weeks with carbidopa/levodopa and 3 weeks with placebo. There will be a total of 3 study visits.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with  ALS or PLS who have clinically significant spasticity

Contact Information:
Phone: (1-844) ALS-Care
ALS@wustl.edu