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.

SOD1 Half-Life Measurements in ALS Patients

Understanding the half-life of the SOD1 protein in cerebral spinal fluid of both ALS and control participants will help us in planning clinical trials for ALS patients with SOD1 mutations. This study involves 16 hr IV, as well as blood draws and up to 5 lumbar puncture procedures to obtain CSF.

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Principal Investigators: Tim Miller, MD PhD

Participants: Open to all ALS patients and healthy controls

View Study Flyer (PDF)

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

 


Genomic Translation for ALS Care (GTAC) – Closed for Enrollment

Across the United States and Scotland, this study will enroll 1500 ALS patients. This clinical dataset will be paired with the analysis of blood samples (e.g. DNA) to understand the complex effects of large networks of genes on the risk and clinical course of ALS. This project will link clinical, treatment, and outcome information in order to better understand the underlying development of ALS. The resulting dataset will be made openly and freely available to other researchers. Certified genetic results will be returned to participants.

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Principal Investigators: Tim Miller, MD PhD

Participants: Open to ALS patients

View Study Flier (PDF)

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: Tim Miller, MD PhD

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

View Study Flier (PDF)

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: Tim Miller, MD PhD

Participants: Open to ALS patients and healthy controls

View Study Flyer (PDF)

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: Tim Miller, MD PhD

Participants: Open to ALS patients and healthy spousal/partner controls

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 ALS patients and healthy
controls

View Study Flyer (PDF)

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

 

Therapeutic Trials

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 ALS patients with C9orf72 Genetic Mutation

View Study Flyer (PDF)

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: Tim Miller, MD PhD

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

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 ALS patients with SOD1 Genetic Mutation

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

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


Phase 3 Trial of ODM-109 (Levosimendan)

This Phase III double-blind, placebo-controlled study will test whether it can confirm that levosimendan can improve respiratory function measured by supine SVC in ALS patients. Approximately 450 subjects with ALS will be enrolled and randomized 2:1 into two study arms to receive drug or placebo. Subjects will receive 48 weeks of medication. If using edaravone or riluzole, must be on a stable dose for 4 weeks prior to screening visit.

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Principal Investigators: Tim Miller, MD PhD

Participants: Open to ALS patients within 12-48 months symptom onset

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


Radicava Biomarkers – Pending

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: Tim Miller, MD PhD

Participants: Open to all ALS patients scheduled to receive Radicava

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: Tim Miller, MD PhD

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

View Study Flyer (PDF)

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