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 neurotrials@neurology.wisc.edu or 608-263-5421. To view a full listing of our clinical trials, search clinicaltrials.gov.

CHILD NEUROLOGY

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
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

Virtual Abecedarian Approach (A2) For IPNH-NDCP (Infants with Perinatal Neurological Histories – At Risk for Neurodevelopmental Disabilities or Cerebral palsy)

Researcher:	Melisa Carrasco McCaul
Sponsor:	UW Institute for Clinical and Translational Research (ICTR)
IRB No.:	2024-1658
Objective:	To address the feasibility of the Virtual A2 intervention, as well as associated compliance and parent satisfaction.
Description:	This study examines a new special program, the Virtual Abecedarian Approach (Virtual A2), in developing improved cognitive (learning) skills in very young children, ages 6 months to 5 years. The program is meant to help children who may have had health problems that could affect how their brain works or make it harder for them to learn, as can be the case in some children who were born extremely premature or who were diagnosed with hypoxic-ischemic encephalopathy at birth. To achieve this goal, pairs of parent-children will be recruited and randomly assigned to the Virtual A2 intervention vs the control group intervention, by chance.
Key Eligibility:	IPNH-NDCP patients, including:
	Term-born children diagnosed during the neonatal period with hypoxic-ischemic encephalopathy, or Premature children (born at less than 28 weeks gestational age).
	Premature children (born at less than 28 weeks gestational age).
	A parent or legal guardian will also be enrolled as a participant.
Compensation:	Participants will receive compensation annually after completing their Follow-Up Assessment Visits ($100) and children will receive a small toy valued at less than $10.
Link:	ClinicalTrials.gov
Site Contact:	neurotrials@neurology.wisc.edu

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice

Researcher:	Cameron Crockett
Sponsor:	Sarepta Therapeutics, Inc.
Protocol No.:	SRP-9001-401
IRB No.:	2025-0125
Objective:	To describe and compare the change from baseline in time to walk/run 10 meters (10MWR) for delandistrogene moxeparvovec-rokl (delandistrogene moxeparvovec)-treated participants with DMD in routine clinical practice, compared to similar DMD participants receiving care in routine clinical practice.
Description:	The 10-year follow-up period in this observational study will gather data comparing the long-term safety and effectiveness for DMD participants receiving delandistrogene moxeparvovec compared to DMD participants receiving standard of care.
Key Eligibility:	1. Is male at birth 2. Is at least 4 years of age at the time of enrollment. 3. Capable of giving signed informed consent/assent (if applicable), which includes compliance with the requirements and restrictions listed in the consent form and in this protocol and has a parent(s) or legal guardian(s) who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures. 4. Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. 5. A participant recruited to Cohorts 1a or 2: Is ambulant (defined as being able to complete the 10MWR in less than 30 seconds) at screening. A participant recruited to Cohort 1b: Is non-ambulant (defined as being unable to complete the 10MWR in less than 30 seconds). 6. Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.
Compensation:	You will receive $40 per completed ePRO questionnaire, up to $480. Your parent/legal guardian (caregiver, a person who takes care of you) may also receive $40 per completed ePRO questionnaire, up to $480, as appropriate.
Link:	ClinicalTrials.gov
Site Contact:	neurotrials@neurology.wisc.edu

Expanded Access Protocol (EAP) of Apitegromab for Patients With Spinal Muscular Atrophy

Researcher:	Jennifer Kwon, MD
Sponsor:	Scholar Rock, Inc.
Protocol No:	SRK-015-008
IRB No:	2025-0853
Objective:	Describe the safety and tolerability of apitegromab in patients with SMA and evaluate the effectiveness of apitegromab with respect to motor funtion measures.
Description:	The purpose of this EAP is to provide access to apitegromab for eligible patients with SMA receiving the approved SMN therapy nusinersen or risdiplam, or have previously received onasemnogene abeparvovec, or any combination of these SMN therapies and in their Treating Physician’s opinion, have an unmet clinical need, and who cannot enter a suitable clinical study.
Key Eligibility:	Aged ≥2 years of age. Documented diagnosis of 5q SMA. Receiving SMN treatment with nusinersen or risdiplam, or have previously received onasemnogene abeparvovec, or any combination of these SMN therapies. Patients who have previously received onasemnogene abeparvovec must have completed the course of steroids postdose with stable liver function tests and stable medical condition prior to receiving apitegromab. Apitegromab must not be initiated until at least 3 months after onasemnogene abeparvovec infusion. Able to receive intravenous (IV) infusions of apitegromab through a peripheral IV or a long-term IV access device as specified in the Pharmacy Manual. Patients who are expected to have reached reproductive maturity during the EAP must agree to adhere to the specified contraception requirements.
Compensation:	There is no payment for taking part in this program. Apitegromab will be given at no cost.
Link	clinicaltrials.gov
Site Contact:	neurotrials@neurology.wisc.edu

EPILEPSY

Measuring Brain Complexity to Detect and Predict Recovery of Consciousness in the ICU (COMPASS)

Researcher:	Melanie Boly
Sponsor:	Mass General Brigham
IRB No.:	2024-1466
Objective:	Demonstrate the diagnostic and prognostic utility of TMS-EEG in ICU patients with DoC (n=120)
Description:	Despite growing evidence that TMS-EEG is a reliable tool to detect the brain’s capacity for consciousness, TMS-EEG has not been systematically implemented or tested in the ICU for patients with Disorders of consciousness (DoC). Our long-term goal is to translate TMS-EEG to the ICU to improve the accuracy of diagnosis and prognosis for critically ill patients with DoC.
Key Eligibility:	Age greater than or equal to 18.
	Functionally independent at baseline.
	Acquired brain injury within the last 28 days.
	Disorder of consciousness, as defined by no instance of following commands (i.e., no instance of Glasgow Coma Scale motor score = 6) on two or more consecutive assessments within the past 24 hours.
Compensation:	$50 for completion of the TMS-EEG procedures in the ICU and an additional $50 for completion of study procedures at 6-month follow-up assessment
Link:	ClinicalTrials.gov
Site Contact:	neurotrials@neurology.wisc.edu

New-onset refractory status epilepticus (NORSE)

Researcher:	Megan Berry, 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:	neurotrials@neurology.wisc.edu

NEUROMUSCULAR

RARE DISEASE

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

MOVEMENT DISORDERS

Enroll-HD: A Prospective Registry Study in a Global Huntington’s Disease Cohort

Researcher:	Dr. 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 Study of AAV2-GDNF in Adults with Moderate Parkinson's Disease (REGENERATE-PD)

Researcher:	Dr. Kathleen Shannon
Sponsor:	Asklepios BioPharmaceutical (AskBio)
Protocol No.:	Ask-PD5-CS201
IRB No.:	2024-1320
Objective:	To evaluate the efficacy of AAV2-GDNF to improve or stabilize motor symptoms in subjects with moderate Parkinson’s Disease.
Description:	Testing an experimental treatment called glial cell line-derived neurotrophic factor (GDNF) gene transfer to see if it helps people with Parkinson’s Disease (PD). GDNF gene therapy (AAV2-GDNF) is delivered to a part of the brain involved in PD. This study will evaluate the efficacy and safety of this study treatment and see whether it can improve the course of moderate PD.
Key Eligibility:	Male and female adults 45-75 years of age inclusive, at the time of signing the informed consent.
Key Eligibility:	Diagnosed with Parkinson’s Disease in the past 4 to 10 years (inclusive) as defined by the presence of bradykinesia plus rigidity, rest tremor and/or postural instability.
Link:	ClinicalTrials.gov
Site Contact:	neurotrials@neurology.wisc.edu

STROKE

Please see information from NIH StrokeNet: https://www.neurosurgery.wisc.edu/research/clinical-trials/#nih-strokenet-and-uw-rcc24

MULTIPLE SCLEROSIS

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

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

Effects of CPC on Migraine Headache and Associated Symptoms - Pilot Study

Researcher:	Ali Zandieh
IRB No.:	2025-1278
Objective:	Percentage of participants who self-administer CPC AND complete the data collection forms in REDCap MyCap as per protocol.
Description:	The purpose of this study is to assess the feasibility of a protocol designed to evaluate the effects of CPC on migraine headache and associated symptoms. We hypothesize that sublingual administration of the CPC preparation during a migraine attack relieves headache and decreases the presence of associated symptoms.
Key Eligibility:	Male or female aged 18 to 50 years At least a 1-year history of disabling migraine with or without aura Patients on stable doses of prophylactic migraine medications for at least 3 months prior to study entry may be included. Willingness to provide written informed consent and authorization for the use and release of health and research study information. Ability to read, understand, and complete study-related questionnaires and diary entries in English. Access to a working smart phone or tablet for the MyCap data entry. Women of childbearing potential must agree to use effective contraception or abstinence during the study period and for up to 7 days after the study.
Compensation:	Participants will be compensated up to $200 for the completion of the study.
Site Contact:	neurotrials@neurology.wisc.edu