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 neurotrials@neurology.wisc.edu or 608-263-5421. To view a full listing of our clinical trials, search clinicaltrials.gov.
CHILD NEUROLOGY
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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 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 |
| Link | clinicaltrials.gov |
Early Biomarkers Study for Prediction of Executive Dysfunction and Cognitive Outcomes Among Healthy Infants and Infants with Prior Neonatal Brain Injuries
| Researcher: | Melisa Carrasco McCaul |
| Sponsor: | Thrasher Early Career Award, Centennial Scholars Program |
| IRB No.: | 2021-1625 |
| Description: | This is a prospective, observational cohort study to examine early biomarkers for executive dysfunction and cognitive disability in infants with and without perinatal brain injury. |
| Objective: | The purpose of the study is to evaluate new methods for assessing risk for executive function and cognitive disability in healthy infants and infants (<24 months of age) with a known history of perinatal brain injury secondary to hypoxic ischemic encephalopathy (HIE), perinatal stroke (PS), neonatal seizures (NS), or premature gestation (PG). |
| Key Eligibility: | Healthy infants and newborns with perinatal brain injury meeting inclusion criteria: those diagnosed during the neonatal period with hypoxic-ischemic encephalopathy, perinatal strokes, neonatal seizures, premature gestational age (born at less than 32 weeks gestational age), having required a cardiac procedure and/or ECMO during the neonatal period. |
| Compensation: | Visit 1 – $100, Visit 2 – $75, Visit 3 – $75. |
| Link: | https://neurology.wisc.edu/research-and-labs/trees-lab/, T.R.E.E.S. Participant Flyer May 2023 Updated |
| Site Contact: | neurotrials@neurology.wisc.edu |
EPILEPSY
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New-onset refractory status epilepticus (NORSE)
Researcher: Aaron Struck, MD
Sponsor: Yale University
IRB No: 2018-0175
Objective:
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.
Description:
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
NEUROMUSCULAR
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A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis (PREVAIL)
| Researcher: | Stephanie Gardon | ||||||
| Sponsor: | Alexion Pharmaceuticals, Inc. | ||||||
| IRB No.: | 2024-0048 | ||||||
| Description: | A study to test the Safety and Efficacy of ALXN1720 in Adults with Generalized Myasthenia Gravis | ||||||
| Objective: | To assess the efficacy of ALXN1720 compared with placebo in the treatment of gMG based on change in MG-ADL total score | ||||||
| Key Eligibility: | 1. Must be ≥ 18 years of age at the time of signing the informed consent 2. Documented diagnosis of gMG ≥ 3 months (90 days) prior to Screening. 3. Positive serological test for AChR autoantibodies at Screening (previous test results, if applicable, must be confirmed by central laboratory during Screening) 4. MGFA Classification II to IV at Screening 5. MG-ADL total score of ≥ 5 at Screening and on Day 1, before randomization 6. Participants receiving treatment with any of the medications listed in Table 6 must have been on a stable dose for the specified periods prior to Screening, with no changes to the regimen expected during Screening or RCT Period. | ||||||
| Compensation: | You may receive compensation for your time when visiting the study site of up to $100 per completed Protocol required on-site visit. | ||||||
| Link: | clinicaltrials.gov | ||||||
| Site Contact: | neurotrials@neurology.wisc.edu | ||||||
PEDIATRICS
MOVEMENT DISORDERS
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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 (topaz.eurekaplatform.org) 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: |
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| 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. |
| Link: | clinicaltrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
Enroll-HD: A Prospective Registry Study in a Global Huntington’s Disease Cohort
| Researcher: | Kathleen Shannon |
| Sponsor: | CHDI Foundation |
| IRB No.: | 2021-0877 |
| Objective: | The primary objective of Enroll-HD is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). Enroll-HD is conceived as a broad-based and long-term project to maximize the efficiencies of non-clinical research and participation in clinical research. With 158 active clinical sites in 19 countries, Enroll-HD is now the largest HD database available and is accessible to any interested researcher – visit www.enroll-hd.org/for-researchers/access-data/ to learn more. |
| Description: | Enroll-HD is a longitudinal, observational, multinational study that integrates two former Huntington’s disease (HD) registries-REGISTRY in Europe, and COHORT in North America and Australasia-while also expanding to include sites in Latin America. More than 20,000 participants have now enrolled into the study. With annual assessments and no end date, Enroll-HD has built a large and rich database of longitudinal clinical data and biospecimens that form the basis for studies developing tools and biomarkers for progression and prognosis, identifying clinically-relevant phenotypic characteristics, and establishing clearly defined endpoints for interventional studies. Periodic cuts of the database are now available to any interested researcher to use in their research – visit www.enroll-hd.org/for-researchers/access-data/ to learn more. |
| Key Eligibility: | Carriers: This group comprises the primary study population and consists of individuals who carry the HD gene expansion mutation. |
| Controls: This group comprises the comparator study population and consists of individuals who do not carry the HD expansion mutation | |
| These two major categories can be further subdivided into six different subgroups of eligible individuals: | |
| Manifest/Motor-manifest HD: Carriers with clinical features that are regarded in the opinion of the investigator as diagnostic of HD. | |
| Pre-Manifest/-Motor-manifest HD: Carriers without clinical features regarded as diagnostic of HD. | |
| Genotype Unknown: This group includes a first or second degree relative (i.e., related by blood to a carrier) who has not undergone predictive testing for HD and therefore has an undetermined carrier status. | |
| Genotype Negative: This group includes a first or second degree relative (i.e., related by blood to a carrier) who has undergone predictive testing for HD and is known not to carry the HD expansion mutation | |
| Family Control: Family members or individuals not related by blood to carriers (e.g., spouses, partners, caregivers). | |
| Community Controls: Individuals unrelated to HD carriers who did not grow up in a family affected by HD. Data collected from community controls will be used for generation of normative data for sub-studies. | |
| Compensation: | You will not receive payment for participating in Enroll-HD. There is also no cost for the research evaluations performed during this research study. Study participants are eligible for a payment to defer the cost of travel. |
| Link: | ClinicalTrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-148009 in Untreated Parkinson’s Disease
| Researcher: | Kathleen Shannon |
| Sponsor: | Inhibikase Therapeutics, Inc. |
| Protocol No.: | IkT-148009-201 |
| IRB No.: | 2021-0877 |
| Objective: | Tto evaluate the safety, tolerability, and exploratory efficacy of three different doses of IkT-148009 self-administered once daily (QD) with food for 12 weeks in patients with untreated PD. |
| Description: | This is a 12-Week, randomized, double-blind, multi-center, placebo-controlled dose-ranging clinical trial of three IkT 148009 doses in patients with untreated PD designed to assess safety, tolerability, and pharmacokinetics of IkT-148009, an oral, once daily c-Abl tyrosine kinase inhibitor. Secondary and exploratory assessments will evaluate the effect of IkT-148009 on motor and non-motor features of the disease. Participants will undergo screening to evaluate their eligibility to participate in the study to include evaluation of Parkinson’s diagnosis, vital signs, blood chemistry, hematology and urinalysis and complete listing of concomitant medications. Those selected will be enrolled and randomized to one of three active IkT-148009 arms (50/100/200 mg) or a placebo arm (3:1). All clinical staff, study investigators, and participants will be blinded to study assignments throughout the trial. |
| Key Eligibility: | Participants who are diagnosed with PD consistent with UK Brain Bank criteria and MDS Research Criteria; must include bradykinesia with sequence effect and motor asymmetry |
| Receiving no anti-parkinsonian therapy | |
| Modified Hoehn/Yahr Stage < 3.0 | |
| Montreal Cognitive Assessment ≥ 26 | |
| Patient expected to be able to participate in trial without need for additional anti-parkinsonian therapy | |
| Capable of giving signed ICF as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. | |
| Female participants of childbearing potential and male participants with female partners of childbearing potential must agree to either remain abstinent or use adequate and reliable contraception throughout the study and at least 30 days after the last dose of study drug has been taken | |
| Compensation: | You will receive $100.00 per complete site visit for travel reimbursement. |
| Link: | ClinicalTrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
STROKE
MULTIPLE SCLEROSIS
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Efficacy and Safety of Remibrutinib Compared to Teriflunomide in Participants With Relapsing Multiple Sclerosis
| Researcher: | Natasha Frost |
| Sponsor: | Novartis Pharmaceuticals |
| Protocol No.: | CLOU064C12301 |
| IRB No.: | 2022-1707 |
| Objective: | To compare the efficacy and safety of remibrutinib versus teriflunomide in patients with relapsing multiple sclerosis |
| Description: | The study CLOU064C12301 consists of an initial Core Part (CP) (maximum duration per participant of up to 30 months), followed by an Extension Part (EP, of up to 5 years duration) for eligible participants. The Core Part is a randomized, double-blind, double-dummy, active comparator-controlled, fixed-dose, parallel-group, multi-center study in approximately 800 participants with relapsing multiple sclerosis (RMS). The Extension Part is an open-label, single-arm, fixed-dose design in which eligible participants are treated with remibrutinib for up to 5 years. |
| Key Eligibility: | 18 to 55 years of age |
| Diagnosis of RMS according to the 2017 McDonald diagnostic criteria | |
| At least: 1 documented relapse within the previous year. OR 2 documented relapses within the previous 2 years, OR 1 active Gadolinium (Gd)-enhancing lesion in the 12 months | |
| EDSS score of 0 to 5.5 (inclusive) | |
| Neurologically stable within 1 month | |
| Compensation: | You will be reimbursed according to the following schedule: Up to $52.00 will be reimbursed for each completed visit except for Day 1 and Months 1, 6 and 12. Up to $75.00 will be reimbursed for each of those 4 completed visits. |
| Link: | ClinicalTrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
HEADACHE
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Randomized Study in Children and Adolescents With Migraine: Acute Treatment
| Researcher: | Ali Zandieh |
| Sponsor: | Biohaven Pharmaceutical Holding Company Ltd |
| Protocol No.: | BHV-3000-311 |
| IRB No.: | 2022-1625 |
| Objective: | The purpose of this study is to test the safety and efficacy of BHV-3000 versus placebo in the acute treatment of moderate or severe migraine in children and adolescents. |
| Description: | Biohaven Pharmaceuticals (Biohaven) is studying a drug called rimegepant as a possible treatment for migraine in children and adolescents. This drug has been approved by the United States (US) Food and Drug Administration (FDA) for treatment of migraine in adults but is considered investigational in this study because it has not been approved for use in children and adolescents for migraine. For this study, researchers would like to see whether rimegepant is effective and safe for treating migraine in children and adolescents between the ages of 12 and 17 years. |
| Key Eligibility: | History of migraine (with or without aura) for > 6 months before Screening according to the IHS Classification ICHD-319 specifications for pediatric migraine |
| History of 1 to 8 moderate or severe attacks per month during the 2 months prior to enrollment, with attacks lasting > 3 hours without treatment, and attacks occurring at intervals > 24 hours. | |
| Prophylactic migraine medication are permitted if the dose has been stable for at least 12 weeks prior to the Baseline Visit, and the dose is not expected to change during the course of the study. a. Participants may remain on one (1) medication with possible migraine prophylactic effects, excluding CGRP antagonists [biologic or small molecule], during the treatment phases. b. Concomitant use of a CGRP antagonist, such as erenumab or fremanezumab, is prohibited. c. Previously discontinued prophylactic migraine medication must have done so at least 90 days prior to the Screening Visit. | |
| Verbally distinguish between migraine and other types of headaches. | |
| Participants must have a weight > 40 kg at the Screening Visit. | |
| Adequate venous access for blood sampling. | |
| Male and female participants ≥ 12 to < 18 years of age (participants must not reach their 18th birthday during the study). | |
| Compensation: | Parents/guardians will be paid $50.00 for each completed visit up to $200.00. Your child will also be paid a total of $150.00 for a completed eDiary. |
| Link: | ClinicalTrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
Long-term Safety Study of Rimegepant in Pediatric Subjects for the Acute Treatment of Migraine
| Researcher: | Ali Zandieh |
| Sponsor: | Biohaven Pharmaceuticals, Inc. |
| Protocol No.: | BHV-3000-312 |
| IRB No.: | 2022-1624 |
| Objective: | The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of moderate or severe migraine in children and adolescents. |
| Description: | Biohaven Pharmaceuticals (Biohaven) is studying a drug called rimegepant as a possible treatment for migraine in children and adolescents. This drug has been approved by the United States (US) Food and Drug Administration (FDA) for treatment of migraine in adults but is considered investigational in this study because it has not been approved for use in children and adolescents for migraine. For this study, researchers would like to see whether rimegepant is effective and safe for treating migraine in children and adolescents between the ages of 12 and 17 years. |
| Key Eligibility: | History of migraine (with or without aura) for ≥ 6 months before Screening |
| History of 1 to 8 moderate or severe attacks per month during the 2 months prior to enrollment. | |
| 1 or more migraine days requiring treatment during the Observation Phase. | |
| Prophylactic migraine medication is permitted if the dose has been stable for at least 12 weeks prior to the Baseline Visit | |
| Ability to distinguish between migraine and other types of headaches. | |
| Weight ≥ 40 kg at the Screening Visit. | |
| Adequate venous access for blood sampling. | |
| Male and female participants ≥ 12 to < 18 years of age (participants must not reach their 18th birthday before enrollment into the study) | |
| Compensation: | Parents/guardians will be paid $50.00 for each completed visit. Children will receive $250.00 for successful eDiary compliance. |
| Link: | ClinicalTrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
GENERAL NEUROLOGY
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CADASIL Consortium
| Researcher: | Dr. Jane Paulsen |
| Sponsor: | NIH |
| IRB No.: | 2021-1033 |
| Objective: | The objective of the proposed research is to exploit an autosomal dominant vascular dementia as a model to investigate specific features of VCID and to examine interactions with risk factors impacting the aging life course. |
| Description: | The study will enroll a total of 500 participants with a CADASIL family history who have had a genetic test for a NOTCH3 variant. Participants will complete: a clinical interview, a neurological exam, neurocognitive and behavior assessments, MRI, and a blood draw at each study visit. Participants will complete 3 in-person visits in total as part of this study: baseline, visit 2 (18 months after baseline), visit 3 (36 months after baseline). Additional contact will occur by phone, mail, email or the internet and will be referred to as “remote visits”. |
| Key Eligibility: | Inclusion Criteria for CADASIL Participants: a) Age at least 18 years old b) positive NOTCH3* genetic testing; or a positive skin biopsy; or willingness to have a NOTCH3 genetic test prior to enrolling and are at-risk for, or diagnosed clinically, with CADASIL c) willing to commit and complete three in-person visits (baseline and 18-month follow-up and 36-month follow-up) as well as remote visits as needed by phone, email, mail or internet. d) All medications will be allowed although the protocol will mandate documentation of medications and our analyses will particularly assess potential impact of medications on outcomes . e) Able to undergo an MRI scan and blood draw at each visit f) A study companion who knows the participant well (>= 3 hours/month of contact) and can provide additional information (either remotely or in-person) g) A functional capacity equivalent to less than 4 on the Modified Rankin Scale.Inclusion Criteria for Healthy Controls (HC) a) Will meet same criteria as CADASIL participants except are or were at risk for CADASIL but have Negative NOTCH3* genetic testing |
| Compensation: | Participants will be reimbursed for each part of the study they complete. • Clinical assessment and completion of other study forms (remote and/or in-person): $25 per visit • MRI scan: $50 per visit • Blood draws: $25 per visit Travel, lodging, and meals will either be provided, or participants may receive partial or complete reimbursement. In addition, airport shuttle, parking, mileage, and tolls may be provided or reimbursed to the participant when applicable. |
| Link: | clinicaltrials.gov |
| Site Contact: | neurotrials@neurology.wisc.edu |
