The objective of this study is to create an international registry with long-term follow-up to characterize and evaluate the safety of abatacept in juvenile idiopathic arthritis (JIA). The primary objective of the JIA registry is to describe the long-term safety of abatacept treatment for JIA by quantifying the incidence rates of serious infections, autoimmune disorders, and malignancies.

Bryce Binstadt - binstadt@umn.edu

This study is designed to assess the safety of GDA-201 + rituximab, as well as the maximum tolerated dose in patients with B cell lymphomas in phase I; in phase II, it will assess safety and efficacy of GDA-201 in cohorts of patients with follicular lymphoma, high grade B cell lymphoma (including diffuse large B-cell), high grade B cell lymphoma not otherwise specified, and primary mediastinal B cell lymphoma.

Veronika Bachanova - bach0173@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

The primary objective is to estimate overall survival (OS) at 3 years post-transplant for patients who received the radiation free preparative regimen BEAM.

Veronika Bachanova - bach0173@umn.edu

The DELIVER-MS study seeks to answer the important question: Does early treatment with highly effective DMT improve the prognosis for people with MS? This is an area of significant controversy and no data currently exist to guide treatment choices for patients and clinicians. The study results will help guide overall treatment philosophy and will be applicable not only to a wide range of existing therapies but also to new therapies, meeting a significant unmet need in patient decision making and aiding the decision for medication approval by third parties.

Beth Zander - zande001@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

This study is a double blinded crossover clinical trial evaluating the safety and effectiveness of incobotulinum toxin A or a placebo (before crossover) injections for the treatment of tinnitus.

Stephanie Standal - stan0621@umn.edu

Smokeless tobacco users who are unable or unwilling to quit tobacco use may be exposed to the potent oral and esophageal carcinogen NNN not only from tobacco itself, but also via its endogenous synthesis from nornicotine. The proposed study will lead to an understanding of the endogenous formation of NNN from nornicotine in humans, and will also investigate the effect of the reduction of nornicotine content in smokeless tobacco on the extent of endogenous NNN formation. The knowledge gained in this study will lead to the development of recommendations for the regulation, or potentially elimination, of nornicotine in smokeless tobacco products in order to minimize exposure to NNN in the users of these products.

Andrew Egbert - egber014@umn.edu

This is an early study of a new drug, called [225Ac]-FPI-1434, to treat solid tumors that have not responded to usual treatment. We are testing different doses of the drug and looking at how well it works for treating the cancer and side effects that occur.

Douglas Yee - yeexx006@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

Study objectives: -To characterize spontaneous and movement-related LFP changes in STN and GP in externalized patients under conditions that modulates the severity of tremor, bradykinesia and rigidity (off meds/off stim; on meds/off stim; off meds/on stim, on meds/on stim). -To characterize and compare the relative effect of different forms of closed loop stimulation (e.g., triggered at specific thresholds of low beta/HFO PAC or beta band activity) to standard isochronal high frequency DBS on motor signs and performance during movement.

Kelly Brown - ksbrown@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

Immunotherapies, such as immune checkpoint inhibitors, have greatly improved survival for many cancers and are now approved for over half of all cancer patients. However, many patients receiving immunotherapy experience Grade 3 and 4 toxicities, termed immune-related adverse events (IRAEs) which cause frequent hospitalizations, emergency department visits, impaired health-related quality of life (QOL) and often discontinuation of therapy. While clinical trials of immunotherapeutic drugs have reported on IRAEs over short time-periods, the real-life and long-term frequencies of and experiences with IRAEs outside of clinical trials, and the general experience of taking immunotherapies long-term remain unknown. The goal of this protocol is to build a prospective cohort study of cancer survivors who receive immunotherapies.

Maria Pecoraro - pecor011@umn.edu

This study will compare the effectiveness and durability of intensive behavioral counseling vs. medical management plus low-intensity behavioral counseling on BMI, body fat, cardiometabolic risk factors, and quality of life in adolescents with severe obesity. We hypothesize that Wegovy (semaglutide) plus low-intensity behavioral counseling will elicit superior reductions in BMI (primary efficacy endpoint) and body fat and greater improvements in cardiometabolic risk factors and quality of life compared to intensive behavioral counseling at 56 weeks.

Nina Jacobs - njacobs@umn.edu

This protocol aims to characterize the safety and tolerability of loncastuximab tesirine in combination with gemcitabine, lenalidomide, polatuzumab vedotin, or umbralisib, and to identify the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) for any of the combinations in subjects with relapsed or refractory B-cell Non-Hodgkin Lymphoma. This project aims to address the resistance mechanisms to single agent therapies and enhance efficacy by engaging different targets, in synergistic or additive manner.

Marie Hu - hu000322@umn.edu

This is a study designed to evaluate the safety, tolerability, and efficacy of a one-time gene therapy (BBP-631) for adult patients diagnosed with classic congenital adrenal hyperplasia (CAH). The goal of gene therapy for CAH is to give the body a functioning CYP21A2 gene using a vector (an agent used to deliver a gene into the body). Having a functioning CYP21A2 gene in the adrenal gland may allow the body to naturally produce its own cortisol and aldosterone. The study treatment and follow-up lasts 1 year with a long-term follow-up of 4 more years.

Kyriakie Sarafoglou - saraf010@umn.edu

This is an open-label, multi-center, Phase 1/2 study of oral LOXO-292 in pediatric patients with an activating RET alteration and an advanced solid or primary CNS tumor.

Allison Fullenkamp - fulle631@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).

Joshua De Kam - jadekam@umn.edu

Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK)trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.

Amy Hanson - amhanson@umn.edu

The purpose of this study is to find out if using a computer program (called iDose) to guide infliximab dosing is more effective and safer than using standard infliximab dosing over 52 weeks. All patients in this study will be receiving infliximab as part of their medical care, this study is only looking at two different methods of determining the dose and timing of administration.

Beiqing Wu - wu000948@umn.edu

PRI-VENT FSGS is a phase III, multicenter, randomized, open label, clinical trial to test the hypothesis that plasmapheresis plus rituximab prior to kidney transplantation can prevent recurrent FSGS in children and adults.

Michelle Rheault - rheau002@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

This is a study of individuals older than 18, undergoing abdominal surgery, and are amenable to fat samples being collected during their surgical procedure, with the option to participate in other tests that can provide information on insulin sensitivity and fat distribution. This study is trying to figure out how fat tissue is related to an individual's health status and health conditions, and the analysis of the aging of cells that make up the human body.

Allison Wolf - wolfx494@umn.edu

This is a Phase 1/2 multicenter, open-label study to evaluate palbociclib in combination with either irinotecan (IRN) and temozolomide (TMZ) or topotecan (TOPO) and cyclophosphamide (CTX) chemotherapy in children, adolescents and young adults with recurrent or refractory solid tumors. The study consists of a non- randomized Phase 1 portion for recurrent or refractory solid tumors followed by potential non- randomized tumor specific cohort(s) and a randomized, Phase 2 portion for recurrent or refractory EWS.

Allison Fullenkamp - fulle631@umn.edu

This will be a Phase II, two-arm, nonrandomized, non-comparative, open-label study in participants ≥ 1 year of age with iobenguane avid, recurrent or progressive high-risk neuroblastoma. Participants not eligible for vorinostat treatment may receive 131I-MIBG as monotherapy.

Allison Fullenkamp - fulle631@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

This project is a data and specimen repository for developmental disorders. Participants provide biological samples and permission to store their health-related data. The purpose is collect and manage these materials for use in biomedical research related to developmental disorders.

Williams Dobyns - wbdobyns@umn.edu

The primary objective is to evaluate whether the addition of adjuvant Pembrolizumab following surgical resection improves disease free survival compared with observation following surgical resection in patients with stage I non-small cell lung cancer (NSCLC) with primary tumors between 1-4 cm in size, regardless of PD-L1 TPS score.

Amit Kulkarni - kulkarni@umn.edu

The overall objective of this study is to determine the feasibility of identifying transdiagnostic biomarkers of cognitive function mediated by neuromodulation of the dorsolateral prefrontal cortex that are translatable across disease groups in order to more accurately phenotype clusters of cognitive dysfunction. Completing behavioral paradigms with electrophysiology and TMS is a challenging frontier. This study focuses on the feasibility of such an endeavor for those with chronic pain or depression as well as healthy controls.

Matthew Maple - maple036@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