Research Header

Clinical Trials

Clinical Trials

To view a full listing of our clinical trials, search clinicaltrials.gov.

CHILD NEUROLOGY

This is an accordion element with a series of buttons that open and close related content panels.

A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2

Researcher:Jennifer Kwon, MD
Sponser:AveXis, Inc.
Protocol No:AVXS-101-CL-304
IRB No:2018-0222
Objective:The purpose of this research study is to determine the safety and effectiveness of the study treatment, AVXS-101, in children 6 weeks of age or younger with SMA.

 

Description:The name of the study treatment used in this study is AVXS-101. AVXS-101 is gene replacement therapy. Administered by intravenous infusion (a needle inserted into a vein), AVXS-101 is designed to deliver a new gene that can produce the Survival Motor Neuron (SMN) protein. SMN is critical to the function of the nerves that control muscles and is missing in children with SMA. Increased production of the protein may lead to improvements in muscle strength and function.

The study treatment will be given in an open label fashion, which means that the subject and parent(s)/legal guardian(s) and the study doctor and staff all will be made aware that the child is taking active study treatment.

Following the study drug administration, the child will be observed in the hospital room for 24 hours.

This study includes a screening period, a gene replacement therapy period, and a follow-up period.

 

Key Eligibility:Patients must meet the following criteria;–Genetic diagnosis of SMA based on gene mutation analysis

–Patients must be < 6 weeks (</= 42 days) of age at the time of AVXS-101 infusion.

–Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy.

–Up-to-date on childhood vaccinations. Seasonal vaccinations that include palivizumab prophylaxis (also known as Synagis) to prevent respiratory syncytial virus (RSV) infections are also recommended in accordance with American Academy of Pediatrics (27).

–Compound muscle action potential (CMAP) ≥2mV at Baseline; centralized review of CMAP data will be conducted.

–Able and willing to follow the Consensus Statement for Standard of Care in Spinal Muscular Atrophy (J Child Neurol.2007;22[29]:1027-1049).

—Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule.

 

Compensation:All participants will receive treatment with the experimental
therapy. Study-related visits, tests, and treatments will be provided to participants at no cost.Travel assistance may be available.
Link:clinicaltrials.gov
Site Contact:Kim Janko

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:Kim Janko

Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion (STR1VE)

Researcher:Jennifer Kwon, MD
Sponsor:AveXis, Inc.
Protocol No:AVXS-101-CL-303
IRB No:2017-1029
Objective:The purpose of this research study is to determine the safety and efficacy of the study treatment, AVXS-101, in children 6 months of age or younger with SMA Type 1. This study will try to find out if it is safe to deliver the SMN gene into the blood stream via intravenous (IV) infusion by a method called “gene transfer”.
Description:The name of the study treatment used in this study is AVXS-101. AVXS-101 is gene replacement therapy. Administered by intravenous infusion (a needle inserted into a vein), AVXS-101 is designed to deliver a new gene that can produce the Survival Motor Neuron (SMN) protein. SMN is critical to the function of the nerves that control muscles and is missing in children with SMA. Increased production of the protein may lead to improvements in muscle strength and function.

The study treatment will be given in an open label fashion, which means that the subject and parent(s)/legal guardian(s) and the study doctor and staff all will be made aware that the child is taking active study treatment.

Following the study drug administration, the child will be observed in the hospital room for 48 hours.

This study includes a screening period, a gene replacement therapy period, and a follow-up period.

Key Eligibility:–Patients with SMA Type 1 as determined by the following features;

–Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 1 or 2 copies of SMN2 (inclusive of the known SMN2 gene modifier mutation (c.859G>C))2.

–The first three patients enrolled must meet the criteria for the Intent-To-Treat Population.

–Patients must be < 6 months (< 180 days) of age at the time of AVXS-101 infusion.

–Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy.

–Up-to-date on childhood vaccinations. Seasonal vaccinations that include palivizumab prophylaxis (also known as Synagis) to prevent respiratory syncytial virus (RSV) infections are also recommended in accordance with American Academy of Pediatrics (27).

–Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule.

Compensation:All participants will receive treatment with the experimental
therapy. Study-related visits, tests, and treatments will be provided to participants at no cost.Travel assistance may be available.
Link:clinicaltrials.gov
Site Contact:Kim Janko

EPILEPSY

This is an accordion element with a series of buttons that open and close related content panels.

A Multicenter, Double-Blind, Randomized, Placebo-Controlled Trial With an Open-Label Extension Phase of Perampanel as Adjunctive Treatment in Subjects at Least 2 years of Age With Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome

Researcher:Anne Josiah, MD
Sponsor:Eisai Inc.
Protocol No.:E-2007-G000-338
IRB No.:2016-5089
Objective:To demonstrate that perampanel given as adjunctive anti-epileptic treatment is superior to placebo in reducing the incidence of drop seizures during 18 weeks of treatment in subjects with inadequately controlled seizures associated with Lennox-Gastaut Syndrome
Description:The purpose of the research is to study whether the investigational drug perampanel, when used with other treatments, is more effective at treating patients with Lennox-Gastaut Syndrome compared to using only standard treatments. Participants will have a 4-8 week Screening/Baseline period to assess eligibility, including seizure activity assessed with diary data. Participants who pass screening will be randomly assigned to receive Perampanel or placebo in pill form to take nightly by mouth for 18 weeks (~4-5 months). During the first 6 weeks (Titration period), the dose will be gradually increased from 2 mg up to a maximum of 8 mg and maintained for 2 weeks. During the next 12 weeks (Maintenance period), participants remain on the dose determined during Titration. During Titration and Maintenance, participants will have 6 scheduled study visits, each lasting about 1 hour. Participants will have a follow-up visit 1 week and again 4 weeks after they stop taking study drug. Participants will be in the study for up to 30 weeks. Participants who complete the Core Study and are eligible to participate in the Extension Study will receive perampanel for up to 52 weeks. The Extension Study involves up to 8 scheduled study visits.
Key Eligibility:Subjects must:Have diagnosis of LGS as evidenced by: More than one type of generalized seizures, including drop seizures (atonic, tonic, or myoclonic) for at least 6 months before Visit 1; and an electroencephalogram (EEG) reporting diagnostic criteria for LGS at some point in their history (abnormal background activity accompanied by slow, spike and wave pattern <2.5 Hz)

Be at least 18 years old at the time of consent

Have experienced at least 2 drop seizures per week in the 4-week Baseline Period preceding randomization; the Baseline Period within the Prerandomization Phase is 4 weeks

Have been receiving 1 to 3 concomitant AEDs at a stable dose for at least 30 days before Visit 1 (vagal nerve stimulation and ketogenic diet do not count as an AED)

Compensation:Participants will receive $30 per visit for travel, for a total of up to $270 during the Core study and $240 during the Extension study.
Link:ClinicalTrials.gov
Site Contact:Kim Janko

A Randomized, Double Blind, Placebo Controlled, Dose Finding Study to Evaluate the Efficacy and Safety of Padsevonil as Adjunctive Treatment of Focal-Onset Seizures in Adult Subjects with Drug-Resistant Epilepsy (ARISE)

Researcher:
Anne F. Josiah, MD

Sponsor:
UCB Biopharma SPRL

IRB No:
2018-1077

Objective:
The purpose of this research study is to determine the safety and effectiveness of the study treatment, padsevonil or placebo, in adults (≥18 years of age) with drug-resistant epilepsy who continue to have uncontrolled focal-onset seizures despite treatment with at least four tolerated antiepileptic drugs. This study involves the administration of padsevonil in different dosing regimens in order to find the best dose (maximum efficacy with minimum side effects) when it is administered with up to three antiepileptic drugs.

Description:

 

Key Eligibility:

Site Contact:

Kim Janko

Adding betaquik® liquid beverage to the modified Atkins diet of women with seizures related to the menstrual cycle – how easy is it, and can it be tolerated?

Researcher:Elizabeth Felton, MD, PhD
Sponsor:Vitaflo International Ltd 
Protocol #:NA
IRB No:2018-0171
Objective:The purpose of this research study is to find out if adding betaquik® liquid beverage to the modified Atkins diet will be something that women with a catamenial seizure pattern (meaning seizures that are related to the menstrual cycle) are able to do and tolerate.
Description:Subjects are in the study for about 6 months. Aside from 3 study visits concurrent with regular clinic visits, study activities take place at home.

Initial Enrollment is expected to add about 1 hour to the regular clinic visit. At this visit, subjects will:

–participate in the consent process

–complete surveys about their menstrual cycle and the modified Atkins diet

–receive betaquik® and discuss how to use it

–have a progesterone level drawn if around day 22 of menses

During the first month, subjects do not drink betaquik® but do the following:

–3-day food record during the first 3 days of menses

–daily check of urine ketones

–blood draw for progesterone level if not done on day of  enrollment

For the remaining 5 months, subjects:

–Drink betaquik® for a specific 10 days each month

–Record betaquik® consumption, missed medications, carbohydrate intake, and twice weekly urine ketones (daily during betaquik® use)

–1-day food recall by phone each month

During checkup clinic visits (month 3 and month 6) subjects will complete the same surveys as the initial enrollment visit.

Key Eligibility:Adult women with epilepsy who have a catamenial seizure pattern, which means related to the menstrual cycle, who are already compliant on the modified Atkins diet.
Compensation:Subjects are paid up to $200
ClinicalTrials.gov
Site Contact:Kim Janko

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:

Kim Janko

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 – kalkach@neurology.wisc.edu

MORE INFORMATION

GENERAL NEUROLOGY

This is an accordion element with a series of buttons that open and close related content panels.

Phase I Trial of the Feasibility and Dose Tolerability of High Definition Transcranial Direct Current Stimulation in healthy adults and children with Down Syndrome

Researcher:Hrissanthi Ikonomidou, MD, PhD
Sponsor:University of Wisconsin-Madison, Department of Pediatric Neurology
IRB:2014-0262
Objective:The purpose of this research study is to find out if high definition transcranial direct current stimulation (HD-tDCS) is feasible, tolerable and safe in children with Down Syndrome.

Transcranial direct current stimulation (tDCS) is a method which enables noninvasive (doesn’t puncture the skin) electrical stimulation of the brain.  tDCS works by placing electrodes on the head (over the hair). tDCS has been shown to improve motor learning, coordination, boost memory in humans and was found to be well tolerated in children with psychiatric disorders. tDCS is being evaluated in stroke rehabilitation in ongoing clinical trials and has already been shown to improve recovery in stroke victims.  In this study, a different type of transcranial direct current stimulation will be used called “high-definition” transcranial direct current stimulation (HD-tDCS). So far, the Federal Drug Administration (FDA) has not cleared tDCS treatment for these indications in humans.

HD tDCS allows for more precise generation of electrical fields over selected brain areas using multiple electrodes.

The child will receive HD-tDCS by placing a cap on their head while they read, watch TV, or use a tablet or computer at the University Hospital or the Health Sciences Learning Center. The caps are made of soft material and have 13 rubber electrodes incorporated in them. These rubber electrodes will be attached to the scalp. Gel will be placed on the child’s scalp before placing the electrodes over their head.

The sessions will be individual, so only the child and the study personnel will be present in the room. The electrodes will be placed every day by study staff.  The sessions will be carefully watched by trained study staff that will remain on site.  During the study visits, the child may request a break any time.

Eligibility:–5-10-year old children with Down Syndrome.

–Study participation will take place at the University Wisconsin Health Sciences Center or the University Hospital and will last up to 14 weeks and involves; 3 study visits before, during, and after a 4-10-week session with high definition transcranial direct current stimulation (HD-tDCS).

Compensation:Participants will be compensated $1000 upon completion of participation. The sum of $100 will be paid after enrollment, $400 after 10 sessions have been completed, $250 after 15 sessions have been completed and the remainder of the payment up to 6 weeks after visit #2 is complete.
Link:ClinicalTrials.gov
Contact:Kim Janko

PARKINSON'S DISEASE AND MOVEMENT DISORDERS

This is an accordion element with a series of buttons that open and close related content panels.

A multicenter, randomized, double-blind, parallel-group, placebo-controlled, fixed-dosage, Phase 2a study comparing 3 dosages of LY3154207 with placebo for 12 weeks in subjects with mild-to-moderate dementia due to Parkinson’s disease

Researcher:Kathleen M. Shannon, MD, FAAN, FANA
Sponsor:Eli Lilly and Company
Protocol No:I7S-MC-HBEH
IRB No:2018-0959
Objective:The purpose of this research study is to find out if a drug called LY3154207 can stop or slow mild-to-moderate Parkinson’s disease dementia. We also want to find out if LY3154207 is safe to use to treat mild-to-moderate Parkinson’s disease dementia. LY3154207 is an investigational drug. This means that the US Food and Drug Administration (FDA) has not approved LY3154207 for the treatment of mild-to-moderate Parkinson’s disease dementia, and LY3154207 can only be given in a research study.

What will happen during the study?
People in this study will be assigned by chance to get either the investigational drug, LY3154207 at 1 of 3 doses (10, 30, or 75 mg) or placebo. The chance you will receive any one of the LY3154207 doses is 3 in 4, or placebo is 1 in 4. You will not be able to choose which drug you get. Study treatment will also be “blinded.” This means that, during the study, you and the researchers will not know if you are getting LY3154207 or placebo. Blinded study treatment is a way to keep people’s expectations from influencing the study results.

Both drugs are pills that you take once each day by mouth.

You will also have blood and urine tests, physical exams, and electrocardiograms (ECGs).

How much time will I spend on the study?
You will come to University Hospital and UW Health Clinics once every 1 to 2 weeks for 12 visits. Visit 1 will take 2-4 hours, Visit 2, Visit 8, and Visit 11 will take about 6 hours, Visit 3 will take about 8 hours, Visits 4 through 7, Visits 9 and 10, and the final follow-up visit will take about 2 hours.

You can be on study treatment until you have bad side effects, your disease gets worse, or you or the study doctor decide you should stop.

Key Eligibility:–40 to 85 years of age
— Have an existing diagnosis of Parkinson’s disease
–Be experiencing a decline in memory and thinking
–Have a study partner (a close friend, relative or partner) who is willing to attend clinic appointments with the study participant
–Study participation will take place the University Hospital and will last up to 17 to 18 weeks and involves 12 study visits
Compensation:Participants or their study partners will be compensated up to $3675 upon completion of participation. Subjects will receive payments throughout the course of the study.
Link:clinicaltrials.gov
Site Contact:Kim Janko
Feedback
Feedback
Do you have any feedback?
Submit
Thank you for submitting your feedback!