Search Results

Humanitarian Use device application for DBS therapy for dystonia

Kelly Ryberg - rybe0010@umn.edu

The goal of this study is to provide comprehensive longitudinal assessments of a cohort of PD patients before, during, and after DBS surgery, including neurological, neurophysiological, and neuropsychological data.

Kelly Brown - ksbrown@umn.edu

The proposed research will measure and compare differences in visual performance and associated neural processing in participants who do or do not experience Visual Snow Syndrome, using a series of well-established psychophysical and imaging paradigms.

Schallmo Lab Study - schallmolab@umn.edu

To evaluate 3 year progression free survival (PFS) rate of high-risk neuroblastoma patients after treatment with a tandem consolidation of Thiotepa/ Cyclophosphamide and PBSC rescue followed by Carboplatin/Etoposide/ Melphalan (CEM) and PBSC rescue, as compared to historical controls of a single CEM consolidation course with PBSC rescue.

Holly Franceen - hfrancee@umn.edu

1.1 Purpose: This protocol will carry out Aim 2 (Experiments 1 and 3) of Udall Project 2, leveraging the novel (on-label, FDA-approved) local field potential measuring capability of the Medtronic Percept™ PC DBS system to study the effects of globus pallidus internus and globus pallidus externus (GPi, GPe) DBS on: the wash-out and wash-in dynamics of motor behavior and local field potentials (LFPs) and correlations between fluctuations in gait and LFPs during activities of daily living (recorded over 4 weeks).

Kristin Garland - garl0038@umn.edu

This protocol will characterize the effects of deep brain stimulation (DBS) location (both adverse and beneficial) on motor signs in people with Parkinson’s disease (PD). This information can be used to inform future DBS protocols to tailor stimulation to the specific needs of a patient. If targeted dorsal GP stimulation is shown to significantly improve motor features that are typically resistant to dopamine replacement therapy, these experiments will likely have major impact on clinical practice by providing a potential strategy to these medically intractable symptoms.

Kelly Ryberg - rybe0010@umn.edu

In this protocol, we will enroll pediatric, adolescent and adult patients diagnosed with adrenoleukodystrophy (ALD). These patients will include probands diagnosed by newborn screening and their relatives subsequently diagnosed, as well other patients who are diagnosed with ALD due to other presenting signs and symptoms and subsequently were confirmed to have ALD. We will ask consenting subjects to provide a medical history (with verification via medical records), to participate in a semi-annual health survey and provide consent to collect biospecimens. The overarching goal of this work is to engage with families affected by ALD and to assemble a resource of clinical, medical, and biological data that will allow of to better understand the natural history of ALD, and how this is affected by newborn screening. The initial focus will be on patients within Minnesota, but participation will be open to any family interested in the study, as this will be web-based. This registry and biobank, together with other research conducted in tandem, will possibly provide information describing the natural history of ALD and outcomes with interventions. It is anticipated that the data collected will further our understanding of the natural history of the disease, basic biology of adrenoleukodystrophy, diagnosis and outcomes. Ultimately, this research may lead to new avenues for early diagnosis and development of safer and more effective therapies for ALD.

Rachael Eye - eye00002@umn.edu

This is a prospective study aimed at quantifying walking and balance in Parkinson's Disease patients in the clinical setting. To accomplish this, we will use a portable motion capture system that is widely used to study biomechanics in humans. Once quantified, we want to test the effects of deep brain stimulation (DBS) frequency in patients who already have DBS systems in place. Thus, our objectives are: 1. Develop an anonymized database of quantitative postural responses and gait of PD patients. 2. Develop software that can quantify the postural response and gait of PD participants using only standard video camera footage. 3. Test the effectiveness of bilateral high and low frequency STN-DBS on the postural responses and gait of PD patients using the portable motion capture system. 4. We will passively record thalamic LFPs from patients with Medtronic Percept DBS devices while the DBS is OFF, set to low frequency, and set to high frequency.

Thomas Lisko - tlisko@umn.edu

This is a study to assess the safety and efficacy of PR001A, an Aden-associated (AAV9) viral vector to treat neuronopathic Gaucher disease type 2 (GD2) in infants. PRA001A will be administered via suboccipital injection to the cisterna magna during a single neurosurgical session. GD2 is a fatal disease of early infancy that does not have any therapeutic options beyond palliative care. This study will enroll infants 0-24 months of age.

Brenda Diethelm-Okita - dieth001@umn.edu

This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.

Kyriakie Sarafoglou - saraf010@umn.edu

The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

Colleen Correll - corr0250@umn.edu

The purpose of the study is to test new biomarkers of amyotrophic lateral sclerosis (ALS) and primary lateral sclerosis (PLS) using MRI scans at 3 tesla (3T). Identifying biomarkers of a disease can lead to a better understanding of the disease as well as improved treatments.

Valerie Ferment - ferm0016@umn.edu

The objective of this study is to evaluate the safety and efficacy of the AccuCinch Ventricular Restoration System in patients with symptomatic heart failure with reduced ejection fraction (HFrEF).

Julie Longman - longm021@umn.edu

The proposed study uses a recombinant AAV2/6 vector encoding the cDNA for human α-Gal A (ST-920). The α-Gal A produced by this cDNA has an identical amino acid sequence to the native enzyme, and also to Fabrazyme® (agalsidase beta or equivalent), a clinically approved recombinant protein product. The ST-920 construct encodes a liver-specific promoter, the human α-1-antitrypsin (hAAT) promoter and includes liver-specific regulatory elements. In addition, rAAV2/6 exhibits liver tropism thus providing the potential for long-term hepatic production of α-Gal A in Fabry disease subjects. Studies of ST-920 in a Fabry disease mouse model administered rAAV2/6 encoding hGLA cDNA by intravenous (IV) injection show generation of therapeutic circulating levels of α-Gal A. The one-time treatment with ST-920 minimizes the risk of infusion--related reactions. The goal of ST-920 is to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3.

Brenda Diethelm-Okita - dieth001@umn.edu

A single visit study with muscle and/or skin biopsy / blood draw, performed to determine whether a molecular or cellular defect can be attributed to cells of FSHD muscle. This study is recruiting both individuals with genetically confirmed FSHD as well as unaffected healthy (control) individuals.

Ana Mitanoska - mitan001@umn.edu

Having PTSD is associated with a higher risk of developing Cardiovascular Disease (CVD), which presents a major health risk for women, who are twice as likely as men to develop PTSD. The purpose of this study is to learn more about the mechanisms behind the relationship between PTSD and increased cardiovascular risk. Ultimately, our goal is to use the knowledge gained from this research study to help develop intervention and treatment strategies to protect the cardiovascular health of women with PTSD.

Nest Lab Study - nestlab@umn.edu

This is a prospective, longitudinal cohort study involving data collection regarding performance of children with ALD and typically developing (TD)children on neurocognitive testing and collection of neuroimaging data. The first goal of this study is to understand more about how ALD affects a child’s brain and development in childhood as they take part in their normal medical care and monitoring. This is important to identifying the best ways to detect and treat manifestations of ALD such as cerebral ALD. The second goal is to learn about how ALD affects caregivers, so that clinicians can offer better support to families in the future.

Brain Study - brainstudy@umn.edu

One treatment for certain types of chronic pancreatitis is total pancreatectomy with islet autotransplantation (TPIAT). In this procedure, the pancreas is removed (eliminating the source of the pain) and the islets, which produce insulin and other important hormones, are taken from the pancreas and transplanted in to the liver. This is a small study to evaluate a new procedure for transplanting some islets to a new location in the body.

Gregory Beilman - beilman@umn.edu

This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.

Timothy Krepski - tkrepsk1@fairview.org

The objectives of this study are to assess the following: - Incidence of sustained donor engraftment at day 42 post transplant - Incidence of transplant related mortality (TRM) at day 100 - Overall survival at day 100 and 1 year - Acute GVHD after this second transplant at day 100 and 6 months - Chronic GVHD after this second transplant at day 12 and 24 months

Timothy Krepski - tkrepsk1@fairview.org

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.

Teresa Bekkala - tkivist1@fairview.org

The purpose of this study is to study the evolution of early life risk factors that may lead to cancer and other conditions. This is a prospective cohort study of families who reside in Minnesota.

Clemen Wilcox - clemenw@umn.edu

The purpose of the research is to find out if atrasentan delays worsening of kidney function in IgAN, FSGS, and Alport Syndrome.

Anael Kuperwajs Cohen - kuper029@umn.edu

Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

Allison Johnston - joh21779@umn.edu

This study is a prospective, non-randomized, observational multi-center study, utilizing a quality improvement methodology to facilitate systematic care coordination, interstage cardiovascular monitoring, and nutritional monitoring into every day practice. Utilizes a national registry to document the impact of these changes on various care processes and outcomes. The aim of this Phase II project intends to: 1) develop and support a robust national registry to gather clinical care process, outcome and developmental data on infants with HLHS between diagnosis and the first year of life, 2) engage pediatric cardiology and cardiac surgery programs in using the registry, and 3) use data from the registry to support and monitor the implementation of QI strategies to standardize and improve care for these infants.

Brittany Faanes - grego318@umn.edu

This study sets out to validate a simple, inexpensive blood test to identify Diastolic dysfunction (DD). Currently, diagnosis depends on costly, time-consuming imaging procedures that are only undertaken after symptoms develop. We have shown in the heart tissues of DD animals (mice and monkeys) and humans that S-glutathionylated cardiac myosin binding protein C (cMyBP-C) is likely responsible for reduced relaxation in DD and is elevated in the blood of each species when DD is present. Specific to the heart, cMyBP-C has been developed as a blood test to predict myocardial infarction. We hypothesize that modified S-glutathionylated cMyBP-C will be a blood marker for DD. We propose to do a non-interventional human clinical study to validate our animal and preliminary human data.

Samuel Dudley - sdudley@umn.edu

The purpose of this project is to provide new knowledge of the relationship between structural and functional changes in cortico-basal ganglia pathways and the severity of motor and non-motor deficits in humans with PD.

Madison Aasen - aasen056@umn.edu

The purpose of this study is to critically assess the current treatment that patients are undergoing by reviewing routine data collected and adding one additional outcome questionnaire solely for research purposes

Lydia Golden - golde406@umn.edu

This is pilot study designed to test the hypothesis that maternal probiotic supplementation is associated with infant gut microbiome variation and improved neurodevelopmental outcomes as measured by ERP performance. The primary aim is to determine if maternal probiotic supplementation during pregnancy and lactation is associated with improved recognition memory performance in infants of diabetic mothers (IDMs). This will involve recruitment and enrollment of pregnant mothers who have been diagnosed with gestational diabetes and randomization to an intervention or control group. Women in the intervention group will receive a standardized probiotic supplement during the third trimester of pregnancy through the first month of lactation. We will compare the IDMs who are exposed to probiotics via maternal supplementation or not with respect to auditory and visual ERPs at 1 and 6 months of age to determine if probiotic supplementation is associated with improved hippocampus function in infancy. The secondary aim is to examine whether maternal probiotic supplementation during pregnancy and lactation is associated with differences in maternal milk and infant fecal microbiome signatures as well as maternal milk and infant serum inflammatory protein levels. Microbial analysis will be performed on infant stool and maternal breast milk samples at one and six months of age. Infant serum and maternal breast milk inflammatory protein levels will be measured at one and six months postpartum.

Maria Bunch - mbunch@umn.edu

A single-center, single-arm, non-interventional natural history study to evaluate the longitudinal clinical course, functional outcome measures, and candidate biomarkers for individuals with DNA repair disorders, including Cockayne syndrome (CS), xeroderma pigmentosum (XP), and trichothiodystrophy (TTD).

Erin Aguero - aguer008@umn.edu