A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients with Generalized Myasthenia Gravis Save

Date Added
September 24th, 2019
PRO Number
Pro00092083
Researcher
Amy Chen

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Keywords
Muscle, Nervous System
Summary

Myasthenia gravis (MG) is a serious, sometimes life threatening, debilitating condition associated with numerous symptoms including muscular weakness and fatigue. This study is to see how effective and safe rozanolixizumab is in adult patients experiencing moderate to severe symptoms of generalized myasthenia gravis (gMG).

The study consists of a Screening Period of up to 4 weeks, followed by a 6-week double-blind Treatment Period and an Observation Period of 8 weeks. During this 18 weeks period there will be 14 visits to the study clinic.

Institution
MUSC
Recruitment Contact
Aparna Choudhury
843-792-7450
choudhur@musc.edu

Development of Wearable Stimulation App to Increase Hand Functional Recovery in Patients with Neurological Movement Save

Date Added
June 4th, 2019
PRO Number
Pro00088976
Researcher
Na jin Seo

List of Studies


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Keywords
Aging, Central Nervous System, Movement Disorders, Muscle, Nerve, Nervous System, Physical Therapy, Rehabilitation Studies, Stroke, Stroke Recovery
Summary

Post-stroke hand impairment diminishes stroke survivors' ability to perform activities of daily living. Motor recovery has been shown to improve through peripheral sensory stimulation. This study aims to determine if vibration from a smartwatch improves post-stroke hand function.

Institution
MUSC
Recruitment Contact
Andrew Fortune
843-792-8970
fortunea@musc.edu

HELIOS-A: A Phase 3 Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) Save

Date Added
May 14th, 2019
PRO Number
Pro00088330
Researcher
Katherine Ruzhansky

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Keywords
Muscle, Nervous System, Rare Diseases
Summary

This is a global phase 3 open-label study designed to evaluate the efficacy and safety of ALN-TTRSC02 in adult patients (18 - 85 years of age) with hATTR amyloidosis. The estimated time on the study is approximately 3 years, including 42 days of Screening, an 18 month Treatment Period and an 18 month Treatment Extension Period.

Institution
MUSC
Recruitment Contact
John Keller
843-792-2209
kellej@musc.edu

Neuroplasticity Associated with Extended Daily Use of a Sensorimotor Priming Vibration System to Improve Hand Function After Stroke Save

Date Added
March 5th, 2019
PRO Number
Pro00086207
Researcher
Na jin Seo

List of Studies


Profiles_link
Keywords
Aging, Central Nervous System, Movement Disorders, Muscle, Nerve, Nervous System, Physical Therapy, Rehabilitation Studies, Stroke, Stroke Recovery
Summary

The objective is to determine if continuous use of TheraBracelet in the home has a clinically meaningful effect in chronic stroke survivors. The study design is a double-blinded randomized controlled trial. We will enroll 40 chronic stroke survivors with moderate hand impairment. Subjects will be randomly assigned to the treatment or control group (n=20 per group). All subjects will wear the TheraBracelet device on the paretic wrist for 8 hours/day every day during their normal daily activity for 1 month. The device will deliver vibration (treatment) or no vibration (control). Double-blinding is possible because the treatment vibration is imperceptible (i.e., subthreshold). Measures of neural plasticity, the amount of the paretic arm use in daily living, clinical hand function, biomechanical grip control, and self-reported abilities for activities of daily living will be assessed at baseline, once a week during the month of wearing the device, and for 3-month follow-up, allowing determination of the efficacy and persistence.

Institution
MUSC
Recruitment Contact
Amanda Vatinno
843-792-8970
vatinno@musc.edu

A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients with Myasthenia Gravis Having Generalized Muscle Weakness Save

Date Added
January 22nd, 2019
PRO Number
Pro00084665
Researcher
Katherine Ruzhansky

List of Studies

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Keywords
Muscle, Nervous System
Summary

Patients who have been diagnosted with Myasthenia Gravis having generalized muscle weakness may qualify to participate in this study.

Subjects that volunteer to participate will undergo a screening visit to assess their health. The purpose of the screening visit is to determine if the requirements to take part in this study are met. If you meet all of the requirements, you will be randomly assigned (like the flip of a coin) to either the study drug that is being researched called ARGX-113 or a placebo treatment group. You will have a one in two chance of being placed in either group. Neither you nor your study doctor will know what group you will be in. If you are in the placebo group, you will receive a substance that looks like ARGX-113 but contains no active medication. This is called a placebo. During the study, both treatment groups will be given the ARGX-113 or placebo through a vein in their arm, called an infusion. Participants will continue to take their current medicines for gMG.

The trial will include a Screening period of a maximum of 2 weeks, a first Treatment Cycle and a maximum of 2 of subsequent Treatment Cycles given on an "as needed basis". Each Treatment Cycle has 9 visits over 8 weeks consisting of a Treatment period of 4 weekly infusions and a Follow-up period of 5 weeks. There are also Inter-treatment Cycle Visits every 2 weeks between Treatment Cycles. The study will last for approximately 28 weeks, and qualified participants will need to come to MUSC at least 19 times over this period.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu

A Phase 3, Open-Label, Multi-Center Trial to Evaluate the Long-Term Safety and Efficacy of Repeat Treatments of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia (ASPEN-OLS) Save

Date Added
December 11th, 2018
PRO Number
Pro00078648
Researcher
Christine Cooper

List of Studies

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Keywords
Central Nervous System, Drug Studies, Movement Disorders, Muscle, Nerve
Summary

The purpose of this study is to evaluate the safety and effectiveness of daxibotulinumtoxinA for injection (a new investigational study drug) compared to placebo in the treatment of cervical dystonia (CD). DaxibotulinumtoxinA for injection is composed of purified botulinum toxin type A, formulated with a small protein RTP004, and will be used for injection.

If you are eligible and choose to be in the study, the dose of study drug you receive (125 units or 250 units) will depend on your clinical assessment, the severity of your disease, and whether you have had any toxin before.

The study may last up to 55 weeks, including 3 weeks of screening. You will come to the study center up to 15 times.

The study is being done at approximately 80 sites. Approximately 290 adults with moderate to severe isolated CD, who are 18-80 will take part study-wide and 4 will take part at this institution.

Institution
MUSC
Recruitment Contact
Timothy Sheehan
843-792-0372
sheehant@musc.edu

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Trial to Evaluate the Efficacy and Safety of a Single Treatment of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia (ASPEN-1) Save

Date Added
November 13th, 2018
PRO Number
Pro00078538
Researcher
Christine Cooper

List of Studies

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Keywords
Central Nervous System, Drug Studies, Movement Disorders, Muscle, Nerve
Summary

The purpose of this study is to evaluate the safety and effectiveness of daxibotulinumtoxinA for injection (a new investigational study drug) compared to placebo in the treatment of cervical dystonia (CD). DaxibotulinumtoxinA for injection is composed of purified botulinum toxin type A, formulated with a small protein RTP004, and will be used for injection. Placebo means it doesn't contain botulinum toxin type A.

If you are eligible and choose to be in the study, the dose of study drug you receive will depend on the group that you will be put into after randomization at the time of your entry.

You will be assigned, by chance, to 1 of the 3 groups below:
? Group 1: High-dose (250 Units of daxibotulinumtoxinA for injection)
? Group 2: Low-dose (125 Units of daxibotulinumtoxinA for injection)
? Group 3: Placebo (a substance that looks like daxibotulinumtoxinA for injection but has no drug in it)

Study lasts aproximately 39 weeks, including 3 weeks of screening. You will come to the study center up to 12 times during the research study.

The study is being done at approximately 80 sites. Approximately 300 people will take part study-wide and 4 will take part at this institution.

Institution
MUSC
Recruitment Contact
Timothy Sheehan
843-792-0372
sheehant@musc.edu

PROMISE-MG: Prospective Multicenter Observational Cohort Study of Comparative Effectiveness of Disease-Modifying Treatments for Myasthenia Gravis Save

Date Added
September 24th, 2018
PRO Number
Pro00077927
Researcher
Katherine Ruzhansky

List of Studies

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Keywords
Muscle, Nervous System
Summary

This is an observational study to develop a research registry to collect information from subjects with Myasthenia Gravis (MG) to evaluate the effects of the treatments they receive and to understand how their medical condition and treatment affects their daily life.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu

Direct measurement of motor cortical responses to transcranial direct current stimulation Save

Date Added
May 15th, 2018
PRO Number
Pro00073545
Researcher
Nathan Rowland

List of Studies

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Keywords
Brain, Central Nervous System, Movement Disorders, Muscle, Nerve, Nervous System, Parkinsons, Surgery
Summary

Transcranial direct current stimulation (tDCS) has shown the potential to improve symptoms in patients with motor deficits, however its effects have not been consistent in randomized studies to date, limiting widespread adoption of this technology. A critical gap in our knowledge is a detailed understanding of how tDCS affects motor areas in the brain. We propose using tDCS while recording directly from motor cortex using subdural electrocorticography (sECoG) in patients undergoing deep brain stimulation surgery. We expect this novel approach to broaden our understanding of tDCS application and possibly lead to therapeutic advances in this population.

Institution
MUSC
Recruitment Contact
Sanicqua Robinson Smalls
843-792-8553
robinsst@musc.edu

Fluid Biomarkers with Deep Phenotyping in Patients with ALS Save

Date Added
June 21st, 2016
PRO Number
Pro00054504
Researcher
Amy Chen

List of Studies

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Keywords
Movement Disorders, Muscle, Nervous System
Summary

You are invited to volunteer for a research study if you have been diagnosed with Amyotrophic Lateral Sclerosis (ALS) within 2 years (24 Months) prior to screening.

This is a non interventional, longitudinal study in patients with ALS. There will be four (4) subject visits in this study: Baseline, month 6, month 12, and month 18. Subjects will have blood and cerebrospinal fluid (a clear fluid found in your brain and spine) collected, and be evaluated with assessment tools that focus on upper and lower motor skills and strength as well as cognitive function. Researchers will use these samples to study ALS, motor neuron disease and other medical conditions.

Institution
MUSC
Recruitment Contact
Christine Hudson
843-792-3790
hudsoncm@musc.edu

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