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.

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


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


IMC – Intermuscular Coherence: A Biomarker for Early Diagnosis and Follow Up of ALS

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 30 minutes to complete.

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Principal Investigators: Robert Bucelli, MD, PhD

Participants: Open to patients who are undergoing a clinical EMG

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


Target ALS Longitudinal Biofluid Study

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 16 months from both ALS participants and healthy volunteers. 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.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with ALS, pre-symptomatic ALS gene carriers, and healthy volunteers

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


Radicava Biomarkers (REFINE)

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

 Regimen D: Trial of Pridopidine
This regimen is being conducted to see if Pridopidine is safe and effective in people diagnosed with ALS. This medication is designed to exert neuroprotective effects and increase motor neuron survival.
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


COURAGE-ALS (CY 5031) – Phase 3 Trial of Reldesemtiv

COURAGE-ALS is a Phase 3 trial of reldesemtiv in patients with ALS. Reldesemtiv is a fast skeletal muscle troponin activator (FSTA) designed to increase skeletal muscle contractility. Participants will be randomized 2:1 to receive 300 mg of reldesemtiv or matching placebo dosed orally twice daily for 24 weeks, followed by a 24-week open-label period in which all participants will receive 300 mg of reldesemtiv twice daily. The trial will enroll approximately 555 people worldwide.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to 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 BIIB105 for sALS with or without Ataxin-2 expansion

This is a Phase 1, randomized, double-blind, placebo-controlled evaluation of the safety and tolerability of BIIB105 in approximately 70 participants with sporadic ALS with or without Ataxin-2 expansion over 13 sites.  BIIB105 is an antisense oligonucleotide (ASO) drug developed for the treatment of sporadic ALS.  It is administered into the spinal fluid via lumbar puncture.  The ASO targets Ataxin-2 and potentially reduces levels of Ataxin-2 protein.  This study will last approximately 29 weeks with a 4-week screening period, 13-week treatment period with BIIB105 and a 12-week follow-up period.

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Principal Investigators: Robert Bucelli, MD, PhD

Participants: Open to patients with sporadic ALS with or without Ataxin-2 mutation

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


ATLAS – Phase 3 Trial of Tofersen

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.

For a more details, please visit:
https://www.alsatlasstudy.com/

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Principal Investigators: Robert Bucelli, MD, PhD

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

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


FUSION (ION363CS1) – Phase 1-3 Trial of ION363

FUSION is a Phase 1-3 trial of ION363 (also known as “jacifusen”). 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 29 weeks, followed by part two, which will be an open-label period in which all patients in the trial will receive ION363 for 73 weeks.

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Principal Investigators: Robert Bucelli, MD, PhD

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

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


Alector AL001-ALS-201 – Phase 2 Trial of AL001

This is a Phase 2 trial evaluating AL001 in patients with C9orf72-associated ALS. AL001 is an antibody designed to increase levels of progranulin, a protein in the body that may protect nerve cells against damage. Participants will be randomized 2:1 to either AL001 or placebo administered via IV infusion every 4 weeks for 24 weeks with an 8-week follow-up period. About 45 participants will take part in this study across 12 sites in the United States.

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Principal Investigators: Timothy Miller, MD, PhD

Participants: Open to patients with ALS who carry a C9orf72 genetic mutation

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