Lois Insolia ALS Clinic
Ensayos Clínicos y Estudios
Revisado el 20 de septiembre de 2023
La Lois Insolia Clinic en el Les Turner ALS Center de Northwestern Medicine participa activamente en ensayos farmacológicos multicéntricos.
La investigación clínica en ELA en el Centro de ELA Les Turner de Northwestern Medicine abarca una amplia gama de trabajos, incluidos estudios de biomarcadores, estudios terapéuticos de etiqueta abierta, estudios de historia natural y ensayos clínicos.
Elegir participar en un estudio es una decisión personal importante. Le recomendamos que hable con su médico, así como con sus familiares o amigos, para tomar la decisión de participar en un estudio.
Por favor, consulte con su médico sobre los criterios de inclusión/exclusión para participar en cualquier ensayo. Para obtener más información sobre los ensayos clínicos en la Lois Insolia Clinic en el Les Turner ALS Center de Northwestern Medicine, comuníquese con Emma Schmidt, Coordinadora de Investigación Clínica, emma.schmidt@northwestern.edu o (312) 503-4362.
Para programar una cita en la Lois Insolia Clinic, llame al 312 695 7950. Los pacientes son atendidos solo con cita previa. La coordinadora de enfermería clínica programará su cita y le explicará el horario de su visita.
Inscripción en Ensayos Clínicos de ELA
Acerca del Ensayo de Fases 2/3
El Ensayo Plataforma HEALEY para ELA (primer ensayo plataforma para ELA) está diseñado para reducir el tiempo necesario para encontrar terapias efectivas, mejorar la proporción de medicamentos en estudio activo en relación con el placebo, y aumentar el acceso para los participantes. Una de las características innovadoras del ensayo es que la inscripción continuará de manera perpetua, a medida que se agreguen más medicamentos en investigación con el tiempo.
Conozca más sobre las primeras cinco compañías y sus tratamientos que participan en el ensayo.
Ensayo Plataforma HEALEY para ELA - Protocolo Maestro
- Ensayo Plataforma HEALEY para ELA - Régimen A: Zilucoplan (Inscripción Completa)
- Ensayo Plataforma HEALEY para ELA - Régimen B: Verdiperstat (Inscripción Completa)
- Ensayo Plataforma HEALEY para ELA - Régimen C: CNM-Au8 (Inscripción Completa)
- Ensayo Plataforma HEALEY para ELA - Régimen D: Pridopidina (Inscripción Completa)
- Ensayo Plataforma HEALEY para ELA - Régimen E: Trehalosa (Inscripción Completa)
- Ensayo Plataforma HEALEY para ELA - Régimen F: ABBV-CLS-762 (Inscripción Abierta)
Por favor, visite Noticias del Ensayo Plataforma HEALEY para ELA para obtener la información más actualizada sobre el ensayo.
Para preguntas adicionales sobre el Ensayo Plataforma HEALEY para ELA, comuníquese con Emma Schmidt, Coordinadora de Investigación Clínica, a través del correo electrónico emma.schmidt@northwestern.edu o al (312) 503-4362.
Acerca del Ensayo de Fase 3
Un Estudio de BIIB067 Iniciado en Adultos Clínicamente Presintomáticos con una Mutación Confirmada de Superóxido Dismutasa 1 (ATLAS).
El objetivo principal de este estudio es evaluar la eficacia de BIIB067 cuando se inicia en portadores adultos presintomáticos de una mutación de superóxido dismutasa 1 (SOD1) con niveles elevados de neurofilamento (NF). Los objetivos secundarios del estudio son evaluar la seguridad y tolerabilidad de BIIB067 y evaluar el efecto de BIIB067 en los biomarcadores de farmacodinámica (PD)/respuesta al tratamiento cuando se inicia antes de la aparición de esclerosis lateral amiotrófica (ELA) clínicamente manifiesta en comparación con su inicio en el momento de la manifestación clínica de la ELA.
Estudios en Proceso de Inscripción: Investigación Clínica para Comprender la ELA y la PLS
Expanded Access Program
Este es un estudio no intervencional (sin fármacos en estudio) de historia natural en pacientes con esclerosis lateral primaria (PLS). El propósito de este estudio es desarrollar un conjunto de datos de historia natural y un biobanco de casos de PLS en fases tempranas y bien establecidos para futuros ensayos clínicos. El estudio también evaluará las diferencias en la progresión de la enfermedad en casos de PLS temprana y PLS bien establecida.
Los pacientes se inscribirán durante un período de 24 meses y completarán evaluaciones en persona y por teléfono.
Más Información
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@es.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.
This is a multi-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 300 participants diagnosed with ALS.
Enrollment Completed
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.
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.
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.
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.
