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Neurology Clinical Trials

The Department of Neurology's clinical trials team at Indiana University School of Medicine specializes in clinical neurologic research of various disease states, which includes Alzheimer’s disease and related dementias, Huntington's disease, Parkinson’s disease, epilepsy, stroke, ALS, muscular dystrophy, myasthenia gravis and myotonic dystrophy.

Through support and partnerships with the National Institutes of Health, pharmaceutical sponsors and various academic medical centers across the country, the Neurology clinical trials team is able to advance research and treatment for patients living with these diseases of the nervous system.

Participate in research

Want to help researchers find new ways to diagnose and treat neurologic conditions? Contact the Neurology clinical trials team directly at 317-963-4823 or by email to get connected to ongoing studies.
2024 Clinical Research Data
296
new participants
69
open clinical trials
1,723
participant visits

What are clinical trials?

Clinical trials are research studies that involve people and are conducted to evaluate a new medical treatment, drug or medical device. They are designed to find new and improved ways to diagnose, treat or prevent different diseases. Researchers depend on patient volunteers to participate in clinical trials to determine whether a new drug or procedure is more effective compared to the current or standard method. Besides improving their own health, participants can make a life-saving difference in lives of others with similar conditions.

There are two main categories of trials: interventional and observational. Interventional trials involve studying a new medication. Observational studies do not have an interventional aspect such as medication, but they strive to learn more about the disease process to help develop treatments. The majority of interventional trials have a placebo group, or a group that does not receive active medication. This is to help make sure study results are accurate.

The Department of Neurology offers nearly 60 active clinical trials.

Alzheimer's disease and dementia

A research study to follow participants over several years for imaging (MRI and PET scans) and cognitive testing. There is no medication associated with this research study. Participants may or may not have a diagnosis of Alzheimer’s disease and may still participate with no concerns about their memory. All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.

You may be eligible if you are ages 55-90, have a study partner and are able to undergo an MRI. If receiving Aducanumab clinically, you must have received a consistent dose for 24 weeks prior to starting this research study.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Jared Brosch, MD

Clinical Trial Website

A research study for cognitive testing and imaging in participants with Frontotemporal Dementia (FTD). All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. There is no study medication in this clinical trial.

You may be eligible is you have a diagnosis of Frontotemporal Dementia and are able to undergo an MRI (do not have a pacemaker or severe claustrophobia).

For more information, email us at neuroct@iupui.edu or call 317-963-4823.

Faculty: David Clark, MD

A research study of study medication ALN-APP to remove amyloid plaques from the brains of individuals with Early Onset Alzheimer’s Disease.All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. Study medication or placebo is an injection given into the spinal column by a study physician.

You may be eligible if you are over 18 years of age, with symptoms of Alzheimer’s before the age of 65, are able to undergo an MRI (do not have a pacemaker or severe claustrophobia), and can receive a lumbar puncture (spinal tap) procedure.

For more information, email neuroct@iu.edu or call 317-963-4823. 

**Enrolling by invitation only

Faculty: Jared Brosch, MD

A research study of study medication CT1812 to improve cognition in participants with Alzhiemer’s disease.  Participants will take study medication or placebo orally, daily at home.  All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you, over approximately 18 months.

You may be eligible to participate if you are ages 50-85. Have a diagnosis of either Mild Cognitive Impairment (MCI) or Alzheimer’s disease, and are able to swallow capsules.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Jared Brosch, MD

Clinical Trial Website

A research study for participants with Alzheimer’s disease to study the effect of niacin (vitamin B3) on cognition.  =All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.  Participants will take study medication niacin (vitamin B3) or placebo once daily for approximately 60 days, and return to the research center at Day 30 and Day 60 for follow-up, including a lumbar puncture for cerebrospinal fluid (CSF) collection.

You may be eligible if you are ages 60-85, have a diagnosis of Alzheimer’s disease, and have a study partner that can attend study appointments with you. Please note, you will be asked to not take multivitamins or supplements (including Liquid IV and energy drinks) containing niacin/vitamin B3 for 90 days during your participation in the study.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Jared Brosch, MD

A research study to form a group of participants with Down Syndrome interested in research treatments in the future for dementia. All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. There is no study medication in this research study. Visits will consist of yearly questionnaires to follow participants over a period of time.

You may be eligible if you have Down Syndrome, are able to undergo an MRI (do not have a pacemaker or severe claustrophobia) and are between the ages of 30-55.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: David Clark, MD and Jill Fodstad, PhD

Clinical Trial Website

Parkinson's disease and Huntington's disease

A registry research study for participants with Huntington’s disease.All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.There is no study medication in this research study.Visits will consist of yearly questionnaires to follow participants over a period of time.

You may be eligible to participate if you are 18 years of age or older and have a confirmed genetic mutation for Huntington’s disease.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Christopher James, MD

A research study to collect blood or saliva samples from participants with Parkinson’s disease to learn if they have a genetic variant causing Parkinson’s disease.All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. There is no study medication in this research study.Participants will sign a consent and provide a saliva sample either in clinic or at home, and receive a free genetic disclosure session after results are received.

You may be eligible if you are over the age of 18, have a diagnosis of Parkinson’s disease and do not have an active cancer diagnosis.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: S. Elizabeth Zauber, MD

Epilepsy

A research study of study medication RAP-219 to decrease seizure frequency for participants with refractory focal epilepsy and an implanted Neuropace Responsive Neurostimulator (RNS).  All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.  Participants will take the study medication orally at home daily, and complete a seizure diary daily.

You may be eligible if you are over the age of 18, have a diagnosis of focal epilepsy with an implanted RNS, have had at least 8 seizures in the last four weeks.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Vicenta Salanova, MD

A research study of XEN1101 in participants with generalized tonic clonic seizures or focal onset seizures to help control or limit seizures.All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.Participants will take the study medication or placebo orally, daily at home and complete a seizure diary.

You may be eligible if you are over the age of 12, have generalized tonic clonic seizures or focal onset seizures with onset prior to age 40, willing to keep a daily seizure diary and are not pregnant or planning to be pregnant.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Alison Kostandy, MD

Stroke

A research study to follow participants with recent stroke over four years for cognitive and functional changes. Participants are consented within six weeks after a stroke and complete a one time blood draw with cognitive testing every six months. Many assessments are completed over the phone. Any onsite assessment will be completed at the IU Health Neuroscience Center at no cost to you. No medication is associated with this research study.

You may be eligible if you are over the age of 18, were admitted to the hospital with a diagnosis of a stroke and have no history of dementia.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Jason Mackey, MD

A research study to evaluate a thrombectomy procedure (removal of a clot in a blood vessel in the brain) for those with a stroke scale score of less than six.Participants for this research are enrolled in the hospital setting and may receive a procedure to remove a clot in the brain during an active stroke.Participants will then have follow-up appointments at 30 days and 90 days for safety evaluation.

You may be eligible if you are over the age of 18 and have been admitted to the hospital for a stroke.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Kaustubh Limaye, MD

A research study to evaluate study medication Milvexian after a stroke, to try to prevent future strokes.Participants will be enrolled while admitted to the hospital for a stroke.After leaving the hospital, participants will take the study medication or placebo orally, daily at home.Participants will then return to the IU Health Neuroscience Center for safety evaluation and follow-up every three months for approximately two and a half years.

You may be eligible if you are over 40 years of age, have a diagnosis of stroke and do not have a history of increased bleeding risk.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Ann Jones, MD

ALS, Myasthenia Gravis, Myotonic Dystrophy, Muscular Dystrophy

A research study for participants with Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig’s disease) or participants with a first degree relative with ALS to study the genetics and biomarkers of the disease through blood and cerebrospinal fluid (CSF) collection. All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. Study medication could be an oral medication taken daily at home, or a subcutaneous shot.

You may be eligible if you are over the age of 18, have a diagnosis of ALS or are a first-degree relative of someone with ALS, and are willing to undergo a blood draw and optional lumbar puncture.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Cynthia Bodkin, MD

A research study for study medication AOC 1001 for participants with a diagnosis of Myotonic Dystrophy, type 1 (DM1) to hopefully help improve hand function and weakness. All study procedures will be conducted at the IU Health Neuroscience Center or University Hospital at no cost to you. Participants will receive an IV dose of study medication or placebo every eight weeks for approximately 12 months.

You may be eligible if you are ages 16 to 65, have a genetic diagnosis of DM1, and do not currently have a diagnosis of heart failure or high blood pressure.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Adam Comer, MD

A research study of study medication Amlentetug (LU AF82422) for participants with Multiple System Atrophy (MSA) to potentially slow progression of MSA. All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. Participants will receive a monthly infusion of study medication or placebo for about 18 months.

You may be eligible if you are ages 40 to 55 with a diagnosis of Multiple System Atrophy, have study partner that can attend study infusions with you, and can receive an MRI.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Alex Barboi, MD

A research study for participants with Postural Orthostatic Tachycardia Syndrome (POTS) to improve symptoms of high heart rate and low blood pressure upon standing. All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. Participants will receive three subcutaneous injections of study medication or placebo, at two visits approximately one week apart. Participants will return to the research center every few weeks for two months in follow-up.

You may be eligible if you are ages 18 to 55 with a diagnosis of POTS, and do not have a history of or take medication for high blood pressure.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Alex Barboi, MD

A registry research study for participants with Pompe disease. There is no study medication for this trial. Participants will answer questions and be followed clinically by the research team to further understand Pompe disease and treatments. All study procedures will be conducted at the IU Health Neuroscience Center. Participants will be followed for up to 5 years.

You may be eligible to participate if you are over the age of 18, have a diagnosis of Pompe disease and receive medical therapy, and are not currently receiving a study medication in another trial for Pompe disease.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Cynthia Bodkin, MD

A research study for participants with ALS. This research study has different study medication options available for participants with ALS. All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you. Study medication could be an oral medication taken daily at home, or a subcutaneous shot.

You may be eligible if you are over the age of 18, have an onset of ALS symptoms within the last 36 months and are able to swallow pills and liquids for the duration of the trial.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Cynthia Bodkin, MD

A research study to evaluate DNTH103 in participants with Generalized Myasthenia Gravis.  All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.  Participants will come to the research center every two weeks to receive an injection of DNTH-103 or placebo.  After 12 weeks, there is an opportunity to continue dosing with only active study medication DNTH-103 (no placebo) for up to one year.

You may be eligible if you are ages 18-75, have a diagnosis of Generalized Myasthenia Gravis (gMG), have not received IVIg within the last 4 weeks, and do not have a history of thymic malignancy.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Cynthia Bodkin, MD

A research study for participants with generalized myasthenia gravis to complete assessments via a smart phone application and in-person to determine if remote assessment and symptom reporting assisting with doctor-patient communication.

You may be eligible to participate if you are ages 18 to 60, own and able to use a smart phone with iOS v14 or Android v8 or higher and able to read the language used in the application (English, Spanish, or French available).

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Sara Takacs, MD

A research study of medication EDG-5506 in participants with Becker Muscular Dystrophy to help prevent muscle breakdown and disease progression. All study procedures will be conducted at the IU Health Neuroscience Center or Methodist Hospital at no cost to you. Participants will take one dose of study medication or placebo each night before bed and return to the research center for follow-up for 12 months.

You may be eligible if you are ages 18 to 50 with documented Becker Muscular Dystrophy, can still walk (with or without assistive devices), and was assigned male sex at birth.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Cynthia Bodkin, MD

A research study to evaluate Ibudilast to reduce inflammation in participants with ALS.All study procedures will be conducted at the IU Health Neuroscience Center at no cost to you.Participants will take the study medication or placebo orally, daily at home and return to the research center for follow-up.Some visits can be complete over the phone.After 12 months of treatment, participants are eligible to receive active study medication (no placebo group).

You may be eligible if you are ages 18 to 80, have a diagnosis of ALS and are able to swallow capsules.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Cynthia Bodkin, MD

Neuro-oncology

An open-label clinical trial for participants with recurrent glioblastoma, for use of the Sonocloud-9 device in conjunction with chemotherapy to better distribute medication to the tumor bed. All study procedures will be conducted at the IU Health Neuroscience Center or Methodist Hospital.  Participants will undergo standard tumor resection, and those chosen to receive the Sonocloud-9 device in the trial will have it implanted at the time of surgery.  Some participants will not receive the Sonocloud-9 device and will be followed as they receive standard therapies.  Participants will be seen at the Neuroscience Center approximately every three weeks over the course of six months for treatment and follow-up.

You may be eligible if you are over the age of 18 and have a glioblastoma brain tumor, and are able to undergo surgery for partial removal of your tumor and placement of the Sonocloud-9 device.

For more information, email neuroct@iu.edu or call 317-963-4823.

Faculty: Kathryn Nevel, MD 

Neurology clinical trials contacts
headshot of Sheryl Lynch

Sheryl Lynch

Director of Clinical Research

Email

headshot of lauren perrey-moore

Lauren Perrey-Moore

Clinical Research Manager

Email