Lois Insolia ALS Clinic
Clinical Trials and Studies
Updated March 27, 2023
The Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine is actively involved in multi-center drug trials.
Clinical research in ALS at the Les Turner ALS Center at Northwestern Medicine encompasses a broad range of work, including biomarker studies, open label therapeutic studies, natural history studies and clinical trials.
Choosing to participate in a study is an important personal decision. We encourage you to talk with your doctor and family members or friends about deciding to join a study.
Please talk with your physician about the inclusion/exclusion criteria for participation in any trial. To learn more about clinical trials at Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine, please contact Emma Schmidt, Clinical Research Coordinator, emma.schmidt@northwestern.edu or (312) 503-4362.
To make an appointment at the Lois Insolia ALS Clinic, please call 312 695 7950. Patients are seen by appointment only. The clinical nurse coordinator will make your appointment and discuss the schedule of your visit.
Enrolling ALS Clinical Trials
A Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of SAR443820 in adult participants with amyotrophic lateral sclerosis (ALS), followed by an open-label extension.
Purpose: To learn about the safety and efficacy of SAR443820 in adults with a diagnosis of ALS and to look at the level and action of the study drug in the body and what happens to this level over time. SAR44380 inhibits a receptor in your nervous system called RIPK1. When RIPK1 is activated, it results in inflammation and damage to your cells. Because SAR443820 works by blocking RIPK1, it may help reduce inflammation and damage to cells in your nervous system and interfere with the pathway causing ALS.
About the Phase 2/3 Trial
The HEALEY ALS Platform Trial (first-ever ALS platform trial) designed to decrease the amount of time it will take to find successful therapies, improve active study medication to placebo ratio, and increase access for participants. One of the innovative features of the Platform Trial is that enrollment will continue perpetually as more investigational medications are added over time.
Learn more about the first five companies and their treatments participating in the trial.
HEALEY ALS Platform Trial - Master Protocol
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- HEALEY ALS Platform Trial - Regimen A Zilucoplan (Enrollment Complete)
- HEALEY ALS Platform Trial - Regimen B Verdiperstat (Enrollment Complete)
- HEALEY ALS Platform Trial - Regimen C CNM-Au8 (Enrollment Complete)
- HEALEY ALS Platform Trial - Regimen D Pridopidine (Enrollment Complete)
- HEALEY ALS Platform Trial - Regimen E: Trehalose (Enrolling)
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HEALEY ALS Platform Trial - RegimenF:ABBV-CLS-762 (Not yet recruiting)
Recent News
Please visit HEALEY ALS Platform Trial News for the most up-to-date information regarding the trial.
For additional questions about the Healey ALS Platform Trial, please contact Emma Schmidt, Clinical Research Coordinator, emma.schmidt@northwestern.edu or (312) 503-4362.
About Phase 3 Trial
A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation (ATLAS).
The primary objective of this study is to evaluate the efficacy of BIIB067 when initiated in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability of BIIB067 and to evaluate the effect of BIIB067 on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).
Enrolling Studies: Clinical Research to Understand ALS and PLS
This is a non-interventional (no study drug), natural history study of patient with primary lateral sclerosis (PLS). The purpose of this study is develop a natural history dataset and biorepository of early PLS and well-established PLS cases for future clinical trials. The study will also evaluate differences in disease progression in early PLS and well-established PLS cases.
Patients will be enrolled over 24 months and complete assessments in person and over the phone.
The purpose of this research study is to collect clinical information from people living with ALS and other motor neuron disorders through the natural history of their disease. Data is obtained from the participants during their standard of care visits and by reviewing their medical records.
The objective of compiling clinical, phenotypic data over the course of the participants’ disease history is to identify clinical similarities, improve our understanding of these diseases longitudinally, and identify avenues for novel treatment.
The investigators’ long-term goals are to improve diagnosis and develop effective treatments that arrest or ameliorate symptoms of ALS, and possibly delay or prevent disease onset in individuals at risk for developing familial ALS (FALS).
In order to do this one must understand how disease develops at a molecular level. Identification of genes that increase risk for developing all types of ALS will reveal the pathways of molecular events that are involved in ALS.
For additional questions, please contact Nailah Siddique, RN MSN at nsiddique@northwestern.edu.
The purpose of this registry is to better describe the incidence and prevalence of Amyotrophic Lateral Sclerosis (ALS) in the United States; examine appropriate factors, such as environmental and occupational, that may be associated with the disease; better outline key demographic factors (such as age, race or ethnicity, gender, and family history of individuals who are diagnosed with the disease) associated with the disease; and better examine the connection between ALS and other motor neuron disorders that can be confused with ALS, misdiagnosed as ALS, and in some cases progress to ALS.
Click here to enroll in the National ALS Registry.
For assistance with enrolling, contact Cara F. Gallagher M.A., LCPC, National ALS Registry Associate at 847 745 6053 or cgallagher@lesturnerals.org.
This study is being conducted to help the investigators better understand how the new FDA approved medication Edaravone (also known as Radicava) works in subsets of patients with ALS. The investigators are also trying to understand if there are specific ALS patients, with different presentations of ALS, who might benefit most from this medication. Also, the investigators are following specific biomarkers to determine the optimal treatment duration in patients with different forms of ALS
There is no study medication being offered in this trial. Edaravone is prescribed as part of regular care. In this trial we are collecting blood, urine, and spinal fluid samples in ALS patients who are taking Edaravone and ALS patients who are not taking Edaravone to measure certain markers that could indicate why the drug may be working in a specific type of ALS.
The purpose of this study is to develop a practice for measuring the efficacy of ultrasound-guided shoulder joint injections in patients with ALS who have exhibited shoulder pain and/or adhesive capsulitis. Patients will receive corticosteroid injections as part of their normal or standard care for shoulder pain in ALS (not provided by the study). They will then be asked to complete questionnaires at Baseline and over the phone over approximately 3 months.
For more information contact Emma Schmidt, Clinical Research Coordinator, emma.schmidt@northwestern.edu or (312) 503-4362.
Expanded Access Program
The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.
Enrollment Completed
This is a non-interventional study (no study drug). We are measuring home-based digital assessments.
The purpose of this study is to evaluate the feasibility, patient-acceptability, adherence and validity of home-based collection of digital measures that are relevant to the assessment of disease in ALS patients. The goal of this study is to determine which home-based digital measurements are reliable predictors of change in ALS status, when compared to the conventional scale, the ALSFRSR.
For more information contact Emma Schmidt, Clinical Research Coordinator, emma.schmidt@northwestern.edu or (312) 503-4362.
About the Phase 3 Trial
Researchers are studying the investigational therapy because they believe it may reduce the amount of harmful or toxic SOD1 proteins and slow the progression of the disease.
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), and efficacy of BIIB067 administered to participants with ALS and confirmed SOD1 mutation.
Learn more:
A Phase 2a Open-Label Preliminary Safety, Efficacy, and Biomarker Study of WP-0512 in Patients with Amyotrophic Lateral Sclerosis (ALS).
Creation of a large repository of induced pluripotent stem cells (iPSC), 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. People with other motor neuron diseases and healthy controls will be included as comparisons.
About the Phase 3 Trial
Researchers are studying the investigational therapy because they believe it may reduce the amount of harmful or toxic SOD1 proteins and slow the progression of the disease.
The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Tofersen) in adult with ALS. The primary objective of Part C of this study is to evaluate the clinical efficacy of BIIB067 administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation.
The secondary objective of Parts A and B of this study is to evaluate the effects of BIIB067 on levels of SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, and pharmacodynamic (PD) effects of BIIB067 administered to adult participants with ALS and confirmed SOD1 mutation.
Northwestern is only participating in Part C of the Valor study, which recruits "Fast Progressor" forms of the SOD-1 gene.
Learn more:
The purpose of this study is to evaluate the safety of intramuscular (IM) administration of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS) as compared to Placebo. Safety will be assessed by incidences of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), injection site reactions (ISRs) and other adverse events of special interest (AESIs), and the clinically significant laboratory values after injections of Engensis compared to Placebo. Exploratory endpoints include assessment of muscle function using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) and ALSFRS-R subscores for Fine and Gross Motor Function; muscle strength by quantitative testing using handheld dynamometry (HHD) and the Accurate Test of Limb Isometric Strength (ATLIS) where available; quality of life using the ALS Assessment Questionnaire (ALSAQ-40); patient global impression of change (PGIC), clinical global impression of change (CGIC), and clinical global impression of severity (CGIS); and evaluation of lung function using Slow Vital Capacity (SVC). Muscle biopsies will be performed during the study for future biomarker analyses.
About Phase 3 Trial
The Phoenix Trial is a randomized, double blind, placebo-controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS. AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R and survival over 48 weeks. The trial will also assess the effects of AMX0035 on slowing vital capacity, quality of life, and plasma biomarkers of ALS.
For more information, visit: https://clinicaltrials.gov/ct2/show/study/NCT05021536 and https://www.amylyxalstrial.com/
About Phase 3b Trial
To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48.
About the Phase 3 Trial
Masitinib is a selective, oral tyrosine kinase inhibitor with neuroprotective capability demonstrated via numerous preclinical studies. Two of masitinib's main cellular targets are the mast cell and microglia cell. It is well-established that mast cells play a prominent role in neuroinflammatory processes. Microglia, resident immune cells of the central nervous system (CNS), also constitute an important source of neuroinflammatory mediators and may have fundamental roles in numerous neurodegenerative disorders. The development of masitinib in ALS is therefore based on the pharmacological action of masitinib in microglia cells and mast cells, thereby slowing microglial-related disease progression, reducing neuro-inflammation, and modulating the neuronal microenvironment in both central and peripheral nervous systems. This is a multicenter, double-blind, randomized, placebo-controlled, parallel-group (two ascending dose titrations of masitinib and matching placebo), comparative study of oral masitinib in the treatment of patients with amyotrophic lateral sclerosis (ALS).
More Information
Recent News
AB Science announces a voluntary hold in the clinical studies of masitinib worldwide (June 1, 2021)
REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and protein biomarkers will also be investigated.
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the endoplasmic reticulum (ER) and mitochondria. This Expanded Access Program is designed to provide expanded access to AMX0035 for the treatment of people living with ALS and assess safety in diverse populations/stages of ALS over 48 weeks.
Dr. Senda Ajroud-Driss, Director of our Lois Insolia ALS Clinic at the Les Turner ALS Center discusses the breakthrough Healey ALS Platform Trial and why it’s a game-changer for ALS research.
Additional Resources
Learn more about ALS Clinical Research, view educational webinars and how to participate in the Clinical Research Learning Institute (CRLI).
We are a partner in Answer ALS, a large research study building the most comprehensive clinical, genetic, molecular and biochemical assessment of ALS, and openly sharing the results with the global research community
ALS Signal: Clinical Research Dashboard designed for and by patients and caregivers. This tool will provide an overview of treatments and supplements that are being tested in the USA and across the world.
