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.
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
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.
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
Neuromuscular Biomarker 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.
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.
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.
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
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 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.
Now enrolling:
Regimen E: Trial of Trehalose (SLS-005)
Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood-brain barrier, stabilizes proteins and, importantly, activates autophagy, which is the process that clears pathologic material from cells. In animal models of ALS, treatment with trehalose delayed onset of disease, prolonged survival and preserved motor function and motor neurons in the spinal cord. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a myriad of diseases with pathologic accumulation of storage material. The drug is manufactured by Seelos Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases.
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.
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.
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/
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.
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
Center for Outpatient Health
Clinical Translational Research Unit (CTRU)
- Physical Address: 4901 Forest Park Avenue, St. Louis, MO, USA
Located on the 5th floor of the Center for Outpatient Health building.