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ALS Clinic and Research Center
The experts at the Penn State Health ALS Clinic and Research Center provide a definitive diagnosis and offer comprehensive, multidisciplinary care that provides treatment and support to optimize dignity, independence, and quality of life. Research is an essential part of the clinic. The clinic and research center is funded by the ALS Association Greater Philadelphia Chapter, the ALS Association’s national office, the Harriett H. and Paul G. Campbell Research Fund for ALS, and by private donations from many individuals and groups. Learn more about our comprehensive patient care.
The research studies are focused on three main areas, with opportunities for individuals with ALS and related motor neuron disorders to participate in clinical studies to help advance knowledge and treatment:
- Treatment trials of medications that show promise for slowing or halting the course of ALS disease progression
- The use of technology to assess speech, swallowing, breathing, walking and other essential functions in persons with ALS
- Improving the understanding of ALS and other motor neuron diseases, optimizing care and reducing the burden on persons with ALS and their caregivers
The clinic and research center is recognized by the ALS Association as a Certified Treatment Center of Excellence that evaluates and treats ALS and related conditions. About 100 new patients with ALS are diagnosed at the center every year and more than 230 are followed long-term at any given time.
Treatment trials of medications
Treatment trials of medications that show promise for slowing or halting the course of ALS disease progression
HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a multicenter, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. The trial is designed as a perpetual platform trial. This means that there is a single Master Protocol determining the conduct of the trial.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
A phase 3, multicenter, double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS)
The main purpose of this research study is to evaluate the efficacy and safety of reldesemtiv. By efficacy, we mean the effect of taking reldesemtiv and the participant’s ability to perform certain functional assessments. By safety, we mean the collection of information about whether there are any negative symptoms and/or side effects that may result from taking reldesemtiv. Additionally, this research study will evaluate the pharmacokinetics (PK) of reldesemtiv (how much reldesemtiv gets into the bloodstream and how long it takes the body to get rid of it).
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
HIMALAYA Study – Phase 2 study for SAR443820 in participants with amyotrophic lateral sclerosis (ALS)
This is 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, followed by an open-label extension.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
The use of technology
The use of technology to assess speech, swallowing, breathing, walking and other essential functions in persons with ALS
Assessment of nocturnal hypoventilation in amyotrophic lateral sclerosis
The overall goals of this study are to identify how the measurement of carbon dioxide during sleep can improve recognition of respiratory distress in neuromuscular disease and thus improve timely access to respiratory therapies which prolong survival. Subjects in this study will participate for up to one year, during which their carbon dioxide levels will be measured in the clinic and in their homes during sleep.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Remote pulmonary function testing in amyotrophic lateral sclerosis (ALS)
The specific objective of this proposal is to validate the practice of remote pulmonary function testing (rPFT) through home measurement of breathing function. The central hypothesis is that guided home assessment of breathing function is a valid method for detecting respiratory distress. This study has the potential to transform the current practice of conducting breathing assessments every three months, resulting in timelier detection and management of respiratory distress. The study has two parts. The first part [CLOSED TO ENROLLMENT] is a self-controlled study which will enroll 40 patients from the ALS clinic. On the day of their clinical visit, study participants will perform both a standard PFT with a respiratory therapist and a simulated rPFT. The primary hypothesis is that there is no difference in the results of PFT and rPFT. The second part is a two-arm randomized intervention in 40 patients with ALS. All subjects will perform weekly home respiratory testing for up to a year, and complete questionnaires at regular intervals during this period. Part 2 also incorporates a nurse health coaching component, with subjects randomized to a group that receives monthly nurse coaching, and one that does not.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Digital tools for assessment of motor functions and falls in ALS
This is a 48-week single arm study that incorporates digital tools for assessing motor function in amyotrophic lateral sclerosis (ALS). During the study, neck- and wrist-worn “activity sensors” (PAMSys, BioSensics, Newton, MA) will be worn by subjects while performing tasks of daily living. Subjects will also complete a motor, speech, and handwriting assessment during site visits. They will complete a digital home assessment of speech, handwriting, and pattern tracing tasks throughout the study, and report any falls which occur on the study tablet. We will explore whether sensor-based functional changes are sensitive to self-reported changes over the length of the study.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Spoken communication in people with ALS
This is a zoom-based research study on how people with ALS can improve verbal communication. In this study, you will first be asked to complete a hearing test and a short demographic questionnaire. After that, you will do one of two tasks depending on the condition you are assigned to. In one of the tasks, you would listen to a recorded speech and indicate what the person in the recording said. In the other condition, you would play an interactive game with another participant. In the second condition, you would be interacting via Zoom to complete a task together and your speech will be recorded.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
A multisensory evoked potential brain-computer interface for communication in ALS
A brain-computer interface (BCI) is a device that has the potential to restore communication by direct translation of brain signals. The BCI used in this study, the P300 Speller, relies on the generation of a P300 evoked potential when a user is presented a rare and unpredictable target stimulus amidst a larger pool of non-target stimuli. This evoked potential is used to perform selections on the computer. Those with advanced ALS demonstrate decreased capacity for BCI control using the P300 speller. With this study, we aim to use a combination of eye tracking and sensory testing to quantify sensory and cognitive processes necessary for the generation of a P300 response. We will test the performance of a multisensory P300 spelling task, where brain potentials are evoked using a combination of visual, auditory, and tactile stimuli. The goal is to demonstrate the perceptual benefits of multisensory integration and generate evidence for its use in this patient group.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Home telemonitoring of bulbar function by acoustic measurement of swallowing and speech sounds in ALS
Most individuals with ALS experience changes in speech and swallowing over the course of the disease. In some, these are their initial indication of ALS. Identifying these changes, which may be rapid in some individuals, is complicated by the recent acceleration of virtual care delivery. This is a longitudinal home study of ALS patients to assess speech and swallowing function through use of smartphone application. The overall hypothesis is that this monitoring protocol can be used in a way that, 1) is satisfactory to the patient, 2) performs at least as well as standard clinical measures of dysarthria and dysphagia, and 3) resolves the development of emergence of speech and swallowing pathologies in ALS. Patients enrolling in this study will participate for approximately 24 weeks, during which they will have swallowing and speech tests performed, complete surveys, and perform audio recordings of speech on their cellphone. Healthy controls will be enrolled to judge the intelligibility of speech samples provided by patients in the study.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Improving the understanding of ALS
Improving the Understanding of ALS
The Impact of Personalized Prognostic Information for Patients with Amyotrophic Lateral Sclerosis
This study will attempt to determine if patients with ALS wish to receive personalized survival predictions, which patients choose to receive this information, how survival information influences patients’ decisions, as well as if and how predictions benefit patient well-being over time. The overall hypothesis is that patients with ALS who choose to receive personalized predictions would use the information to guide advanced care planning and goals of care discussions, and that with appropriate emotional, mental health, and spiritual support the benefits they receive from the predictions would outweigh the potential harms.
For more information, interested individuals should contact our research team at nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Determining ultrasonographic cross sectional area reference values for vagus and phrenic nerves in patients with motor neuron disease and assess their role as a potential predictor of disease progression
The purpose of this study is to determine ultrasonographic cross sectional area reference values for vagus and phrenic nerves in patients with motor neuron disease and assess their role as a potential predictor of disease progression. Phrenic and vagus nerve will be affected during the course of disease and cause bulbar and respiratory symptoms. We are also recruiting healthy volunteers to participate in this study. They will have 1 ultrasound done so that we can compare our findings to better help us in identifying bulbar and respiratory changes in patients with motor neuron disease.
For more information, interested individuals should contact our research team at nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Treatment trials of medications that show promise for slowing or halting the course of ALS disease progression
HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a multicenter, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. The trial is designed as a perpetual platform trial. This means that there is a single Master Protocol determining the conduct of the trial.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
A phase 3, multicenter, double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS)
The main purpose of this research study is to evaluate the efficacy and safety of reldesemtiv. By efficacy, we mean the effect of taking reldesemtiv and the participant’s ability to perform certain functional assessments. By safety, we mean the collection of information about whether there are any negative symptoms and/or side effects that may result from taking reldesemtiv. Additionally, this research study will evaluate the pharmacokinetics (PK) of reldesemtiv (how much reldesemtiv gets into the bloodstream and how long it takes the body to get rid of it).
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
HIMALAYA Study – Phase 2 study for SAR443820 in participants with amyotrophic lateral sclerosis (ALS)
This is 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, followed by an open-label extension.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
The use of technology to assess speech, swallowing, breathing, walking and other essential functions in persons with ALS
Assessment of nocturnal hypoventilation in amyotrophic lateral sclerosis
The overall goals of this study are to identify how the measurement of carbon dioxide during sleep can improve recognition of respiratory distress in neuromuscular disease and thus improve timely access to respiratory therapies which prolong survival. Subjects in this study will participate for up to one year, during which their carbon dioxide levels will be measured in the clinic and in their homes during sleep.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Remote pulmonary function testing in amyotrophic lateral sclerosis (ALS)
The specific objective of this proposal is to validate the practice of remote pulmonary function testing (rPFT) through home measurement of breathing function. The central hypothesis is that guided home assessment of breathing function is a valid method for detecting respiratory distress. This study has the potential to transform the current practice of conducting breathing assessments every three months, resulting in timelier detection and management of respiratory distress. The study has two parts. The first part [CLOSED TO ENROLLMENT] is a self-controlled study which will enroll 40 patients from the ALS clinic. On the day of their clinical visit, study participants will perform both a standard PFT with a respiratory therapist and a simulated rPFT. The primary hypothesis is that there is no difference in the results of PFT and rPFT. The second part is a two-arm randomized intervention in 40 patients with ALS. All subjects will perform weekly home respiratory testing for up to a year, and complete questionnaires at regular intervals during this period. Part 2 also incorporates a nurse health coaching component, with subjects randomized to a group that receives monthly nurse coaching, and one that does not.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Digital tools for assessment of motor functions and falls in ALS
This is a 48-week single arm study that incorporates digital tools for assessing motor function in amyotrophic lateral sclerosis (ALS). During the study, neck- and wrist-worn “activity sensors” (PAMSys, BioSensics, Newton, MA) will be worn by subjects while performing tasks of daily living. Subjects will also complete a motor, speech, and handwriting assessment during site visits. They will complete a digital home assessment of speech, handwriting, and pattern tracing tasks throughout the study, and report any falls which occur on the study tablet. We will explore whether sensor-based functional changes are sensitive to self-reported changes over the length of the study.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Spoken communication in people with ALS
This is a zoom-based research study on how people with ALS can improve verbal communication. In this study, you will first be asked to complete a hearing test and a short demographic questionnaire. After that, you will do one of two tasks depending on the condition you are assigned to. In one of the tasks, you would listen to a recorded speech and indicate what the person in the recording said. In the other condition, you would play an interactive game with another participant. In the second condition, you would be interacting via Zoom to complete a task together and your speech will be recorded.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
A multisensory evoked potential brain-computer interface for communication in ALS
A brain-computer interface (BCI) is a device that has the potential to restore communication by direct translation of brain signals. The BCI used in this study, the P300 Speller, relies on the generation of a P300 evoked potential when a user is presented a rare and unpredictable target stimulus amidst a larger pool of non-target stimuli. This evoked potential is used to perform selections on the computer. Those with advanced ALS demonstrate decreased capacity for BCI control using the P300 speller. With this study, we aim to use a combination of eye tracking and sensory testing to quantify sensory and cognitive processes necessary for the generation of a P300 response. We will test the performance of a multisensory P300 spelling task, where brain potentials are evoked using a combination of visual, auditory, and tactile stimuli. The goal is to demonstrate the perceptual benefits of multisensory integration and generate evidence for its use in this patient group.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Home telemonitoring of bulbar function by acoustic measurement of swallowing and speech sounds in ALS
Most individuals with ALS experience changes in speech and swallowing over the course of the disease. In some, these are their initial indication of ALS. Identifying these changes, which may be rapid in some individuals, is complicated by the recent acceleration of virtual care delivery. This is a longitudinal home study of ALS patients to assess speech and swallowing function through use of smartphone application. The overall hypothesis is that this monitoring protocol can be used in a way that, 1) is satisfactory to the patient, 2) performs at least as well as standard clinical measures of dysarthria and dysphagia, and 3) resolves the development of emergence of speech and swallowing pathologies in ALS. Patients enrolling in this study will participate for approximately 24 weeks, during which they will have swallowing and speech tests performed, complete surveys, and perform audio recordings of speech on their cellphone. Healthy controls will be enrolled to judge the intelligibility of speech samples provided by patients in the study.
Contact: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Improving the Understanding of ALS
The Impact of Personalized Prognostic Information for Patients with Amyotrophic Lateral Sclerosis
This study will attempt to determine if patients with ALS wish to receive personalized survival predictions, which patients choose to receive this information, how survival information influences patients’ decisions, as well as if and how predictions benefit patient well-being over time. The overall hypothesis is that patients with ALS who choose to receive personalized predictions would use the information to guide advanced care planning and goals of care discussions, and that with appropriate emotional, mental health, and spiritual support the benefits they receive from the predictions would outweigh the potential harms.
For more information, interested individuals should contact our research team at nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Determining ultrasonographic cross sectional area reference values for vagus and phrenic nerves in patients with motor neuron disease and assess their role as a potential predictor of disease progression
The purpose of this study is to determine ultrasonographic cross sectional area reference values for vagus and phrenic nerves in patients with motor neuron disease and assess their role as a potential predictor of disease progression. Phrenic and vagus nerve will be affected during the course of disease and cause bulbar and respiratory symptoms. We are also recruiting healthy volunteers to participate in this study. They will have 1 ultrasound done so that we can compare our findings to better help us in identifying bulbar and respiratory changes in patients with motor neuron disease.
For more information, interested individuals should contact our research team at nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Faculty
Staff
Contact us
To contact any of our staff members: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
Contact us
To contact any of our staff members: nervemuscle@pennstatehealth.psu.edu or 717-531-8ALS (8257)
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We have developed a number of resources that can be used by ALS health care professionals to assess patients and their caregivers. For questions or if you need additional information on these instruments, please contact nervemuscle@pennstatehealth.psu.edu.
Support for Caregivers
Download our ALS Caregiver Assessment
ALS and frontotemporal dementia (FTD)
Download our ALS/FTD Caregiver Assessment
Download the Cognitive Intervention Program Presentation
Improving end-of-life care and decision making
Download the Communication and Treatment Preference Assessment
Philanthropic support is important to the ALS Clinic and Research Center. Tax-deductible gifts to help advance ALS research and care may be made via the Penn State College of Medicine Office of Development and Alumni Relations.
Did you know you can make a gift through your will to support ALS Research?
To learn more, please contact Owen Thomas, our ALS Gift Officer, at 717-531-8497 or oit1@psu.edu.
Contact Us
For more information about ALS research, or to speak with a research coordinator, please call 717-531-8ALS (8257) or e-mail nervemuscle@pennstatehealth.psu.edu.