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Clinical Trials

Clinical Trials

The Neurology Department is dedicated to advancing research in the prevention, detection and treatment of neurological diseases in areas such as Movement/Parkinson’s, Epilepsy, Multiple Sclerosis, and Neuromuscular disorders. We have numerous clinical trials currently being conducted to help us do just that. If you or anyone you know would be interested in participating in one of these clinical trials, please contact us at or 608-263-5421. To view a full listing of our clinical trials, search


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Prospective Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA)

Researcher:Jennifer Kwon, MD
Sponsor:AveXis, Inc.
Protocol No:AVXS-101-RG-001
IRB No:2018-1013
Objective:The purpose of this registry is to assess the long-term outcomes of participants with SMA in the context of advances in treatment options.
Description:Your child will not be asked to come for any additional clinical visits or have any other laboratory tests done as part of participation in this registry. All data that is collected as part of this registry is taken from the information your child’s study doctor has documented in their medical notes during their normal doctors’ visits, questionnaires for the study that you will be asked to complete during your normal doctors’ visits, as well as tests that were performed during these visits.

SMA treatments will be reviewed to see what effect they have on your child as follows:–To assess the effectiveness of treatments for SMA

–To assess long-term safety
–To assess overall survival of all subjects with SMA
–To assess healthcare utilization
–To assess caregiver burden
–To assess participant functional independence

Information about your child’s SMA treatment will be collected for the registry for a period of up to 15 years.

Key Eligibility:Participants must meet the following criteria;–Subjects with genetic confirmation of SMA
Compensation:You/your child will be compensated $25 as reimbursement for your time completing the questionnaires and travel related to participation in the registry.
Site Contact:Neurology Trials


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New-onset refractory status epilepticus (NORSE)

Researcher: Aaron Struck, MD
Sponsor: Yale University
IRB No: 2018-0175

To learn more about the cause of new-onset refractory status epilepticus (NORSE), to identify why NORSE affects different people differently and to find the best management strategy to treat patients who have the condition.

This is a data/sample repository study. Information from the medical record and samples will be collected during regular hospitalization and at 3 standard care follow up clinic visits within 2 years. Subjects will answer questions about quality of life at these 3 timepoints as well.

Key Eligibility:

–Status Epilepticus refractory to first and second‐line therapy
–No etiology found in the first 24 hours despite extensive work‐up
–Age at least 6 years old

Site Contact:

Neurology Trials

To use [F-18]-FEPPA to evaluate if focal epilepsy patients have increased inflammation ipsilateral to hemisphere of seizure generation relative to age-matched healthy controls and to determine if the time since last seizure correlates with severity of inflammation to test the hypothesis that seizures are temporally related to changes in inflammation.

Researcher: Dr. Aaron F. Struck, MD

IRB No: 2017-0458

Objective: To use [F-18]-FEPPA to evaluate if focal epilepsy patients have increased inflammation ipsilateral to hemisphere of seizure generation relative to age-matched healthy controls and to determine if the time since last seizure correlates with severity of inflammation to test the hypothesis that seizures are temporally related to changes in inflammation.

Description: A total of 40 subjects, 20 patients and 20 healthy controls will be enrolled in this research.

The following data will be obtained from the research participants:

(1) informed consent

(2) PET and MR imaging using [F-18]-FEPPA tracer

(3) blood sample for testing of FEPPA binding

(4) subject-completed questionnaires to provide information regarding frequency and nature of seizures, seizure medications, and cause/onset of seizures, clinical EEG data in the seizure group.

Inclusion criteria for healthy control: 1) Chronological age between 18 and 65 years 2) Able to safely undergo an MR Scan

Inclusion Criteria for patient:  Patients diagnosed with epilepsy includes: (1) chronologic age between 18 and 65 years, inclusive (2) no gross abnormalities on MRI other than hippocampal sclerosis based on prior clinical imaging (3) no other neurologic disorder, and (4) estimated premorbid IQ (AMNART) ≥ 70. (5) Focal epilepsy

Compensation: Subjects are paid $100

Site Contact: Mariel Kalkach –



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Expanded Access Trial of AMX0035 for Amyotrophic Lateral Sclerosis (ALS)

Researcher:Stephanie Gardon
Sponsor:Amylyx Pharmaceuticals Inc.
Protocol No:A35-006
Objective:The primary objective of this program is to provide access to AMX0035 for the treatment of patients with ALS and to assess safety.
Description:In this expanded access program, approximately 250 participants with ALS will be enrolled from approximately 25 sites in the United States (US). Participants determined eligible at the Screening Visit will be considered enrolled and continue to the Baseline Visit which will include the first dose of AMX0035. Enrolled participants will be resupplied with drug approximately every 12 weeks (±4 weeks) after the Baseline Visit.
Key Eligibility:
  • Male or female, at least 18 years of age (inclusive).
  • Diagnosis of ALS made by a physician experienced with the management of ALS.
  • >36 months from symptom onset defined as first weakness.
  • Capable of providing informed consent.
  • Capable of and willing to follow program procedures.
  • Participants who have established care with a physician experienced in treating patients with ALS involved in the program and will maintain this clinical care throughout the duration of their time in the program.
  • Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the program and 3 months after last dose of AMX0035; women must not be planning to become pregnant for the duration of the program and 3 months after last dose of study drug
  • Men must agree to practice contraception for the duration of the program and for at least 3 months after last dose of program drug; men must not plan to father a child or provide sperm for donation for the duration of the program and 3 months after last dose of study drug.
Compensation:You will not receive any payment for taking part in the Program.
Site Contact:Neurology Trials


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International Pedatric Stroke Study (IPSS)

Researcher:Hrissanthi Ikonomidou
Sponsor:The Hospital for Sick Children ‘Sickkids’
IRB No:2020-1472
Objective:This is the first multi-center, multi-national study of children with stroke. It seeks to build a network of investigators and high-quality disease data to enable population-based studies, natural history studies, and clinical trials to improve the care of children with stroke.
Description:The International Pediatric Stroke Study (IPSS) was established in 2003 as a multi-center, multi-national clinical research registry. Over the years, it has grown to become a highly successful study vehicle for pediatric stroke research across over 100 institutions worldwide. Today, The IPSS continues to serve as the global data and imaging core for multi-disciplinary pediatric experts who perform international collaborative research in an effort to better understand, prevent, and improve outcomes in pediatric stroke. The robust dataset and cohesive network enable high caliber and ground-breaking research in the field.

Participating sites enroll neonates or children who have had an ischemic stroke or are at high risk of having a stroke into the registry. Participants have their medical and research records reviewed for information about their stroke and abstracted into a secure electronic database called REDCap. The IPSS also provides an attractive imaging platform (through the Stroke Imaging Lab for Children, SILC, housed at SickKids) for sites to share clinically acquired brain images that will complement the clinical dataset. The clinical and imaging datasets will improve our understanding of the processes underlying plasticity and recovery in childhood stroke.

All information captured in the study is de-identified.

This study does not require any interventions or additional visits for research. Two outcome measures are typically administered during a follow-up clinic visit: the Paediatric Stroke Outcome Measure (PSOM) and the Recovery and Recurrence Questionnaire (RRQ). The PSOM is used during clinic visits by the study PI or co-investigator; the RRQ is a parental questionnaire that can be completed during clinic visits or by telephone interview for those parents who cannot attend the follow up appointment. Both measures assess neurological function in the following 5 domains: sensory, motor, language, cognition and behavior.

Participating investigators will retain ownership of their own (single-site) data. However, by entering data into the IPSS, co-investigators give implicit permission for the IPSS to analyze the collective dataset. These contributing investigators are recognized through authorship.

Key Eligibility:Arterial Ischemic Stroke (AIS):
– Event diagnosis: Diagnosed after Jan 1, 2003
– Age: Patient between >36 weeks and 18 years of age Notes: preterm included if stroke onset was in childhood
– Clinical Criteria: Focal neurological deficit of acute onset lasting greater than 20 minutes
– Radiological Criteria: CT /MRI showing infarct in location consistent with neurological signs and symptoms Notes: Can include Hypoxic ischemic event with diffuse or bilateral infarction if there is a definite focal, single arterial infarct in a specified vascular territory.Cerebral Sinovenous Thrombosis (CSVT):
– Event diagnosis: Diagnosed after January 1, 2003
– Age: Patient pre-term to 18 years of age Notes: pre-term babies <36 according to IPSS definition, included
– Clinical Criteria: Any transient neurological dysfunction including headache, seizure, decreased level of consciousness, focal neurological signs consistent with CSVT
– Radiological Criteria: Thrombosis of cerebral veins or venous sinus seen on MRI, MR venography or CT venography Notes: CT alone if definite is acceptable for older infants and children, but not for neonates under 1 month of ageCerebral Vascular Conditions (other than CSVT/AIS):
– Presumed Perinatal Ischemic Strokes: Infants and children whose stroke is presumed to have occurred in the prenatal or perinatal period Notes:
– These patients have normal development history up until 4-8 months of age, when they present with early hand preference.
– Arteriopathy without stroke
– Arteritis, vasculitis, vasculopathy, Moya Moya with no clinical events or TIA
– Dissections without stroke (can also include those from traumatic brain injury)
– Watershed infarct with Sickle Cell Disease
Site Contact:Neurology Trials


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PROSEEK: A Phase 2 Study In Early Parkinson's Disease Patients Evaluating The Safety And Efficacy Of Abl Tyrosine Kinase Inhibition Using K0706)

Researcher:Kathleen Shannon
Sponsor:Sun Pharma Advanced Research Company Limited (SPARC)
Protocol No:CLR-18-06
IRB No:2020-0457
Objective:To determine if K0706 reduces the rate of progression of early-stage Parkinson’s disease (PD) versus placebo over 40 weeks, as defined by the sum of the MDS-UPDRS (Movement Disorder Society – Unified Parkinson’s Disease Rating Scale) Parts 2 and 3 scores.
Description:This is a study to evaluate the efficacy, safety and tolerability of two doses of K0706 compared to placebo in subjects with early PD who are not receiving dopaminergic therapy. A subset of eligible subjects may participate in a “Biomarker substudy”.

Subjects will visit the clinic for a screening visit, regular assessments and to receive the study drug for 44 weeks.

Subjects will provide blood to measure standard safety labs and K0706 levels. If subjects consent, additional blood will be collected, processed, and frozen for future analyses of emerging tests related to PD progression and K0706 target engagement (e.g., proteins, genetic markers). Subjects may also be provided with a smartphone with software they will use to record performance of a series of motor tasks at, and / or between visits.

Subjects in the Biomarker substudy will have 3mm skin punch biopsies, DaT SPECT, and CSF collected as exploratory measures of PD progression; CSF will be used to measure K0706 level, and frozen for later analysis of emerging biomarkers (e.g., proteins) associated with PD progression and K0706 target engagement.

Key Eligibility:
  • The subject has given written informed consent and is willing to participate in the study;
  • Subject is able to understand and comply with all study procedures (requires literacy in available language of all patient-reported outcome measures)
  • Males or females aged ≥ 50 years
  • Body mass index (BMI) greater than 18.5 kg/m2 and less than 45 kg/m2;
  • Diagnosed with “Clinically Probable PD” according to the MDS clinical diagnostic criteria, with documented diagnosis of PD per treating physician’s records within three years of the Screening visit. Disease severity according to modified Hoehn & Yahr stage ≤ 2.
  • Projected to not require to start dopaminergic therapy within 9 months from Baseline
  • Female subjects must be not of childbearing potential, e.g., documented evidence that they are surgically sterile (e.g., hysterectomy, partial hysterectomy, bilateral oophorectomy, bilateral tubal ligation), or post-menopausal (at least 12 months since last menses) prior to Screening with serum Follicular Stimulating Hormone (FSH) ≥40 mIU/mL).
  • Male subjects enrolled in the study should not father a child and are advised to prevent passage of semen to their sexual partner during intercourse using an effective method as judged by the Investigator for the duration of the study and for 3 months after the last dose of study drug
  • Willingness to undergo lumbar puncture and skin biopsy for future testing of substances related to PD or K0706 target engagement.
Compensation:You will be paid $47 after each completed in-person study visit.
Site Contact:Neurology Trials

Trial of Parkinson’s And Zoledronic Acid (TOPAZ)

Researcher:Dr. Kathleen Shannon
Sponsor:National Institute of Aging
IRB No.:1928009
Objective:The primary aim of the study is to evaluate the efficacy of a single infusion of zoledronic acid (ZA, 5mg) to reduce the risk of clinical fractures in Parkinson’s Disease or neurodegenerative parkinsonism.
Description:This home-based randomized clinical trial is designed to test the efficacy of ZA-5 mg in Parkinson’s disease (PD) and parkinsonism patients. This trial will also address barriers to treatment of patients with PD and parkinsonism by providing rigorous evidence about whether ZA reduces fracture risk in patients with PD and parkinsonism, simplifying treatment by giving ZA at home without extra medical visits and BMD testing, and overcoming poor persistence with oral therapies because one infusion may prevent bone loss for at least 2 years. The outcome of this trial will demonstrate how a home-based fracture prevention can reach older PD patients who would not otherwise receive treatment to reduce their high risk of fractures.

Patients with PD will be recruited throughout the US by participating neurologists and health networks as well as the Parkinson’s Foundation. Patients may also self-refer to the study. Interested patients can access study information on a study website ( as well as through the Parkinson’s Foundation Helpline. Patients who wish to enroll will be directed to an interactive electronic consent (eConsent). Following eConsent, participants complete a screening questionnaire (to confirm eligibility), followed by a baseline questionnaire. If a participant is determined to be eligible following these steps, they may be scheduled for a Telemedicine assessment to further confirm the PD or parkinsonism diagnosis, unless they have been referred directly from a participating neurologist. If confirmed, the participant will be mailed a supply of vitamin D3 800-1000 IU and instructed to take one vitamin D tablet every day for 2 months. Lastly, a Nurse Home Visit will be scheduled and conducted to confirm final eligibility, randomization, and administration of the study drug, if appropriate. Participants who are randomized will be contacted every 4 months for at least 2 years to determine if they have had any fractures.

Key Eligibility:
  • Men and women age 60 or older
  • Current Parkinson’s Disease or neurodegenerative parkinsonism diagnosis (including progressive supranuclear palsy, multiple system atrophy, cortical basal degeneration, vascular parkinsonism, dementia with Lewy bodies or another form of neurodegenerative parkinsonism)
  • Willing and able to continue in follow-up for at least 2 years
  • Willing and able to provide informed consent
Compensation:Participants who complete a Nurse Home Visit and are randomized will be offered an honorarium (for time and effort) of a $100 gift card, a $50 gift card upon completion of the measures in the 1st year of follow-up (through Month 12), another $50 gift card upon completion of the measures in the 2nd year of follow-up (through Month 24), and a $50 gift card for each completed year of follow-up thereafter, as appropriate.


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C3FIT (Coordinated, Collaborative, Comprehensive, Family-based, Integrated and Technology-enabled care): A Comparative Effectiveness Randomized Trial to Improve Stroke Care Delivery

Researcher:Eric Adelman
Sponsor:The Patient Centered Outcomes Research Institute (PCORI)
Protocol No:PCS-2017C3-9081
IRB No:190704
Objective:The current standard of stroke care is the JC-certified CSC/PSC care system, a collection of proven individual processes of care, but a system that lacks coordination for the post-stroke care of patients. The C3FIT trial serves as the opportunity to assess potential impact on patient quality of life and functional outcomes resulting from the improved integration of care provided by the ISPU approach.

C3FIT’s overall goal is to assess if patient outcomes are improved when the CSC/PSC system is supplemented with an Integrated Stroke Practice Unit (ISPU) system of care, a patient-centric model of care involving the patient and caregiver/family that coordinates care from the acute management through the rehabilitation and recovery of the patient.

Description:C3FIT is a pragmatic randomized trial of 18 Joint Commission-certified Comprehensive or Primary Stroke Centers (CSC/PSC) throughout the United States. C3FIT’s specific aim is to assess the superiority of patient-centered outcomes between CSC/PSC and Integrated Stroke Practice Unit (ISPU) models to determine if integrated care models are more effective in nodes of care across the patient recovery continuum. Vanderbilt University Medical Center (VUMC) will guide C3FIT as the Clinical Coordinating Center.
  • ​Age 18+.
  • Clinical diagnosis of acute stroke with brain imaging compatible with intracerebral hemorrhage or ischemic stroke (including normal brain scan)
  • English or Spanish speaking subjects.
  • Patient admitted within 7 days of their index stroke event.
  • Patient is discharged alive and not to hospice care.
  • Patient living at discharge within the geography of recruitment for that C3FIT site.
  • Pre-morbid mRS Rankin score of 0-1.
  • Patient and/or surrogate give consent to participate after an informed consent process.
  • Patients who go to rehabilitation inpatient therapy or other care facilities are eligible, as long as they reside in the geographic are of recruitment and do not go to hospice care.
Contact:Neuro Trials

Study to Evaluate the Efficacy and Safety of Intravenous BIIB093 (Glibenclamide) for Severe Cerebral Edema following Large Hemispheric Infarction (CHARM)

Researcher:Jamie Elliot
Protocol No.:252LH301
IRB No.:20181333
Objective:The primary objective is to determine if BIIB093 improves functional outcome at Day 90 as measured by the modified Rankin Scale (mRS) when compared with placebo in subjects with LHI (large hemispheric infarction).
DescriptionThis is a Phase 3 study of subjects aged 18 to 85. Following a 3-stage 72-hour continuous infusion of BIIB093 or matching placebo, subjects will receive efficacy and safety evaluations for 90 days. The primary efficacy assessment will be the mRS at Day 90. The study will be conducted as a single study in two parts. Part 1 of the study is comprised of the baseline visit, Study Drug infusion and efficacy and safety period through Study Day 90 (primary endpoint). Part 2 will be a LHI follow-up period from Day 91 to Month 12.
Key Eligibility:
  • A clinical diagnosis of acute ischemic stroke in the middle cerebral artery (MCA) territory
  • Aged 18 to 85 years old, inclusive, at the time of informed consent.
  • Screening NIHSS (National Institutes of Health Stroke Scale) ≥10.
  • Prior to the current stroke, no significant disability in the opinion of the Investigator (able to independently perform all duties and activities of daily living without assistance from a caregiver, spouse, or another person).
  • A large hemispheric infarction defined, in order of preference, as either:
    a) a magnetic resonance imaging (MRI) diffusion-weighted imaging (DWI) lesion volume of 80 to 300 cm3, or
    b) a computed tomography perfusion (CTP) core lesion volume of 80 to 300 cm3, or
    c) an Alberta Stroke Program Early computed tomography (CT) Score (ASPECTS) on non-contrast computed tomography (NCCT) of 1 to 5 with involvement of at least 2 defined cortical regions, if lesion volume from MRI DWI or CTP is not available.
  • For subjects who receive thrombectomy prior to randomization, inclusion into the study must be based on post-thrombectomy MRI-DWI.
  • Study drug treatment infusion should be initiated as soon as possible but no later than 10 hours after time of symptom
Compensation:You will receive a stipend of $55.44 per visit for specific visits and may also be reimbursed for travel costs.


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Determining the Effectiveness of early Intensive versus escalation approaches for the treatment of relapsing-remitting Multiple Sclerosis (DELIVER-MS).

Researcher:Natasha Frost
Sponsor:PCORI (Patient-Centered Outcomes Research Institute)
Protocol No:DELIVER-MS-01
IRB No:2019-0405
Objective:To determine whether early treatment with a highly effective disease modifying therapy (DMT), such as monoclonal antibodies, improve prognisis for people with MS.
Description:The DELIVER-MS study seeks to answer the question: Does early treatment with highly effective DMT improve the prognosis for people with MS? This is an area of significant controversy and no data currently exist to guide treatment choices for patients and clinicians. The study results will help guide overall treatment philosophy and will be applicable not only to a wide range of existing therapies but also to new therapies, meeting a significant unmet need in patient decision making and aiding the decision for medication approval by third parties.

Visits will include a Screening Visit, Randomization and Baseline Visit, in-person visits at 6, 12, 24 and 36 months and telephone visits at 3, 9, 15, 18, 21, 27, 30, 33 months.

  • ​Men and women aged 18 to 60 years.
  • Established diagnosis of MS, as defined by the 2017 revision of McDonald Diagnostic Criteria (99).
  • Relapsing-remitting multiple sclerosis disease course as defined by the 2013 revisions of the MS clinical course definition (4).
  • Participants must have evidence of active disease based on:
    – one or more MS relapses within the last 18 months prior to screening visit or
    – radiological evidence of MS activity (≥2 new T2 lesions within the last 12 months from screening or ≥1 GdE demonstrated on brain or spinal cord MRI performed within the last 12 months of screening).
  • Participants must be ambulatory with disease onset ≤ 5 years and treatment-naïve (i.e., no MS disease modifying therapy at any time in the past).
  • Participants must be eligible to receive at least one form of disease modifying therapy within each treatment arm
  • Kurtzke Expanded Disability Status Scale at Baseline visit ≤ 6.5
Contact:Neuro Trials

A Multicenter, Longitudinal, Open-Label, Single-Arm Study Describing Cognitive Processing Speed Changes in Relapsing Multiple Sclerosis Subjects Treated with Ozanimod (RPC-1063)

Researcher:Natasha Frost
Sponsor:Celgene International
Protocol No.:RPC-1063-MS-001
IRB No.:20192310
Objective:The purpose of this study is to describe clinically meaningful changes in performance on the SDMT (Symbol Digit Modalities Test) over a 3-year period in subjects with early MS receiving ozanimod HCl 1 mg.
Description:This is a multicenter, longitudinal, single-arm, open-label study to describe the change from baseline in cognitive processing speed, measured by the SDMT, in subjects with relapsing multiple sclerosis (RMS) treated with ozanimod HCl 1 mg at 3 years.

All subjects will receive orally administered ozanimod HCl 1 mg. The primary efficacy endpoint is the proportion of subjects with a clinically meaningful increase in raw score of ≥ 4 points or 10% from baseline (improved). The treatment period is 36 months. For all subjects who finish the subject and for those who discontinue, there will be a 30-day (± 15 days) and a 90-day (± 10 days) Safety Follow-up Visit. There is no planned protocol extension following the end of the study. Approximately 250 subjects with RMS will be recruited for this study.

Subjects with RMS will be enrolled in this study if they have received ≤ 1 DMT, have an EDSS ≤ 3.5, and have been diagnosed with RMS within 5 years of study entry.

Key Eligibility:
  • Subject must understand and voluntarily sign an informed consent form (ICF) prior to any study-related assessments/procedures being conducted.
  • Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
  • Subject is male or female 18 to 65 years of age (inclusive) at the time of signing of the ICF.
  • Subject has a diagnosis of MS according to the 2010 or 2017 Revised McDonald criteria.
  • Subject has ≤ 5 years since time of relapsing multiple sclerosis diagnosis.
  • Subject has ≤ 1 approved relapsing multiple sclerosis disease modifying therapy at time of study entry.
  • Subjects has a disability status at screening with an EDSS (Expanded Disability Status Scale) score  ≤3.5 (inclusive).
  • Subject has no history of relapse with onset from 30 days prior to Screening. During this period, subjects must have been clinically stable, without systemic corticosteroid treatment or adrenocorticotrophic hormone (ACTH).
  • Subjects must have documentation of positive varicella zoster virus (VZV) immunoglobulin G (IgG) antibody status or complete VZV vaccination at least 30 days prior to enrollment.
  • Females of childbearing potential (FCBP) must agree to practice a highly effective method of contraception throughout the study and until completion of the 90-day Safety Follow-up Visit. Highly effective methods of contraception are those that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly.
Compensation:You will receive up to a maximum of $40 per completed study visit for your travel time. If you do not complete the entire study, you will be paid for each study visit you do complete.