Note: The Minneapolis Veterans Affairs (VA) Health Care Systems (MVAHCS) Institutional Review Board (IRB) will oversee this research study under the main protocol (#VAM-18-00364). This local protocol addendum addresses neuroimaging at the University of Minnesota Center for Magnetic Resonance Research (UMN CMRR). Please note that we have selected "No" to the Drugs question in ETHOS, as the VA IRB approval covers the Ketamine use; only neuroimaging at CMRR is included in the UMN IRB portion of this study.

The two primary aims of the study are: -To evaluate the efficacy of PEER and BWL on the target adolescent’s weight over the 18 months of the study, and -To evaluate the efficacy of PEER and BWL on the target adolescent’s emotion regulation skills and emotional eating over the 18-months of the study.

To assess whether there is superiority of overall survival (OS) when enzastaurin rather than placebo is added to the regimen of temozolomide with radiation therapy followed by temozolomide for the treatment of patients with newly diagnosed glioblastoma in DGM1 biomarker-positive patients, regardless of MGMT methylation status.

TRACK-FA is a multi-site, international, prospective, observational neuroimaging study in Friedreich’s Ataxia. The objective is to obtain natural history MRI and MRS data over time in the brain and spinal cord in a large cohort of FRDA patients and healthy control. This will permit validation of neuroimaging biomarkers in FRDA for future clinical trials. In addition to neuroimaging data, the study will collect data from clinical, cognitive and mood assessments as well as blood samples to measure blood markers (frataxin and neurofilament light chain). The present IRB protocol covers only subjects enrolled at the University of Minnesota.

This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.

This phase III trial studies whether inotuzumab ozogamicin added to post-induction chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves outcomes. This trial also studies the outcomes of patients with mixed phenotype acute leukemia (MPAL), and B-lymphoblastic lymphoma (B-LLy) when treated with ALL therapy without inotuzumab ozogamicin. Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a type of chemotherapy called calicheamicin. Inotuzumab attaches to cancer cells in a targeted way and delivers calicheamicin to kill them. The overall goal of this study is to understand if adding inotuzumab ozogamicin to standard of care chemotherapy maintains or improves outcomes in High Risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL). The first part of the study includes the first two phases of therapy: Induction and Consolidation. This part will collect information on the leukemia, as well as the effects of the initial treatment, in order to classify patients into post-consolidation treatment groups. On the second part of this study, patients will receive the remainder of the chemotherapy cycles (interim maintenance I, delayed intensification, interim maintenance II, maintenance), with some patients randomized to receive inotuzumab. Other aims of this study include investigating whether treating both males and females with the same duration of chemotherapy maintains outcomes for males who have previously been treated for an additional year compared to girls, as well as to evaluate the best ways to help patients adhere to oral chemotherapy regimens. Finally, this study will be the first to track the outcomes of subjects with disseminated B-cell Lymphoblastic Leukemia (B LLy) or Mixed Phenotype Acute Leukemia (MPAL) when treated with B-ALL chemotherapy.

The overall objective of this proposal is to determine the efficacy of a single vs. multiple sub-anesthetic IV ketamine infusions for patients with TRD. We plan to conduct a randomized controlled trial (RCT) comparing a single ketamine infusion preceded by 5 midazolam infusions vs. six ketamine infusions.

This is a 5-year, longitudinal, observational study of adult and pediatric patients (age 2 and above) undergoing IBD therapy designed to specifically address important clinical questions that remain incompletely answered from registration trials. Patients being prescribed medical therapy for IBD outside of a clinical trial will be eligible for enrollment. Treatment algorithms will follow each site’s local standard of care and no specific treatments, assessments, and or laboratory tests will be dictated by enrollment in TARGET-IBD. Enrolled patients will consent to the possibility of up to 3 years of retrospective, redacted medical record collection as well as prospective collection for up to 5 years. Medical records will include but will not be limited to: records of hospitalizations, laboratory reports, clinic notes, telephone contact reports, medication lists, reasons for medication initiation and/or discontinuation, endoscopy reports, biopsy results, and imaging results. Patients will also be asked to provide a blood sample for biomarker, anti-drug antibody, and DNA assays and complete patient reported outcome (PRO) surveys, although participation in these two portions of the study is not mandatory for study participation. Consent for linkage of patient health information (PHI) to external healthcare databases (such as patient support programs) will also be requested as an optional portion of the study.

This study aims to ovaluate the effect of hydradermabrasion for scalp health in patients with androgenetic alopecia, G1 to G4 according to Hamilton Norwood Classification with trichoscopic investigation.

a Phase 3, open-label, safety study of ataluren in patients who previously received ataluren at an investigational site in a prior PTC-sponsored clinical study or treatment plan.

• To classify patients (< 30 years old) with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight and loss of heterozygosity for chromosomes 1p and 16q, to thereby define eligibility for a series of therapeutic studies. • To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists.

This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective CFTR modulators and their impact in children with CF on endocrine growth factors, the gut microbiome, respiratory microbiome, liver and pancreatic function, lung function, sweat chloride, and inflammatory markers.

This phase I/II trial tests the safety, best dose, and whether BAY 1895344 (elimusertib) monotherapy works in treating patients (≥ 12 months and ≤ 30 years) with solid tumors that have come back (relapsed) or does not respond to treatment (refractory). Elimusertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

This study will be a prospective analysis of a post-operative transplant cohort in the PICU to determine whether using magnesium sulfate as an analgesic adjuvant can decrease overall opiate requirement in this patient population. It will indirectly also look at opiate-induced side effects, effects on overall PICU course, and applicability/safety of a magnesium infusion in pediatric patients. It is well known that post-operative analgesia in children is one of many challenges faced by surgeons and intensivists, both due to the invasiveness of procedures as well as the biopsychosocial variance in these populations. TPIAT (total pancreatectomy and islet autotransplantation) and liver transplant patients at our institute have protocols designed for their management, part of which includes continuous opiate dosing, other adjuvants (such as tylenol, ketorolac, ketamine), and sometimes paravertebral nerve blocks. All of these medications, despite their benefits, come with their own unique side effect profile. Opiates remain no stranger to this, in addition to a distinct growing shortage nationwide. Magnesium sulfate has been cited as a potential source of adjuvant analgesia by its action on the NMDA receptor. Pediatric populations where magnesium has shown potential analgesic benefit include post-tonsillectomy, post-osteotomy (cerebral palsy), post-operative scoliosis repair, sickle cell, and hsevere headache management. Literature also supports use in adult populations, which includes more expansive operative cohorts. Added benefit of magnesium is its overall safety profile (symptoms not present until levels significantly above normal indices), cost-effectiveness, and incidental overall prevalence of hypomagnesemia within PICU populations. We plan to implement a magnesium therapy protocol to all of our liver and TPIAT transplant children in the pediatric ICU with dosing that has been used both efficaciously (in comparison to available adult data) and safely (in comparison to other pediatric studies). This will be done via a bolus dose in the operating room followed by infusion dosing for the next 48 hours. Magnesium levels will be checked serially to ensure they remain below toxic levels. We will track opiate dosage metrics throughout their post-operative ICU admission, as well as other secondary outcomes listed elsewhere. The control will be a retrospective chart review of the same primary and secondary outcome measures from previous post-transplant patients in this PICU. The study protocol has been approved by the U.S Food & Drug Administration, which will also be involved in monitoring of the study.

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.

This multinational, multicenter, observational, prospective, long-term registry is designed to collect data on epidemiology, HPP history, clinical course, symptoms (including multi-systemic aspects of disease), and burden of disease from patients of all ages who have a diagnosis of HPP. In addition, the Registry will collect data on asfotase alfa dosing, effectiveness of treatment, SAEs, immunogenicity, pregnancy and neonatal outcome data (for patients treated with asfotasealfa only), and targeted EOI.

This registry is open for people of all ages who have either received or donated an organ at the University of Minnesota. This study aims to evaluate organ transplant outcomes among people who have been part of the transplant program.

This partially randomized phase II/III trial studies how well cisplatin and combination chemotherapy works in treating children and young adults (≤ 30 years of age) with hepatoblastoma or liver cancer after surgery. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy after surgery may kill more tumor cells.

This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen followed by a related or unrelated donor stem cell infusion. It is a modification of the treatment plan which has been studied extensively over the last 10+ years which has shown consistent engraftment and low transplant related mortality (TRM).

This phase II trial studies OST31-164 as a single agent every 3 weeks for 48 weeks, with 4 doses constituting 1 treatment cycle (12 weeks per cycle). Each patient will receive treatment at a dose of 1x109 CFU until Week 48 or until disease progression, unacceptable toxicity, or the patient meets any other treatment discontinuation criteria. Following treatment discontinuation, all patients will enter a 3-year survival follow-up period. The primary endpoints are disease control during the first 12 months after enrollment and safety assessments (adverse events [AEs], physical examinations, clinical laboratory tests, vital sign measurements, performance status, and tests to monitor for the persistence of Lm).

This is a prospective randomized clinical trial, to determine whether dose-intensive tandem Consolidation, in a randomized comparison with single cycle Consolidation, provides an event-free survival (EFS) and overall survival (OS). The study population (children < 120 months) will be high-risk patients (non-Wnt and non-Shh sub-groups) with medulloblastoma, and for all patients with central nervous system (CNS) embryonal tumors completing "Head Start 4" Induction. This study will further determine whether the additional labor intensity (duration of hospitalizations and short-term and long-term morbidities) associated with the tandem treatment is justified by the improvement in outcome. It is expected that the tandem (3 cycles) Consolidation regimen will produce a superior outcome compared to the single cycle Consolidation, given the substantially higher dose intensity of the tandem regimen, without significant addition of either short-term or long-term morbidities.

This study is one of three studies on an NIH-funded project addressing the effectiveness of parents in buffering children and adolescents from the physiological and brain responses to stress. This study uses MRI scanning to measure the brain response to social evaluative stress (giving a speech and doing math problems in front of a panel of judges) as well as the impact of the presence of various social partners (no one, researcher, or parent) in buffering the physiological and brain responses to social evaluative stress.

TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in patients with unresectable, locally advanced or metastatic solid tumors determined to harbor specific oncogenic genomic alterations or who are TMB-high as identified by a validated NGS assay.

This is planned as a multicenter project that will enroll 25 elective Cesarean section patients and gather demographic data, perioperative opioid requirements and perform genetic testing to seek preliminary evidence linking the extremes of opioid requirements (high and low) to genetics. This is intended as a pilot project to demonstrate proof of concept and the feasibility of a much larger multicenter study, with the eventual goal of defining the genomics of postoperative opioid requirements needed for pain control.

The purpose of this study is to assess the severity of illness and associated outcomes of neurological intact survival in patients treated with extracorporeal membrane oxygenation with central and peripheral cannulation techniques.

ARCADIA is a multicenter, biomarker-driven, randomized, double-blind, active-control, phase 3 clinical trial of apixaban versus aspirin in patients who have evidence of atrial cardiopathy and a recent stroke of unknown cause. Eleven hundred subjects will be recruited over 2.5 years at 120 sites in the NINDS StrokeNet consortium. Subjects will be followed for a minimum of 1.5 years and a maximum of 4 years for the primary efficacy outcome of recurrent stroke and the primary safety outcomes of symptomatic intracranial hemorrhage and major hemorrhage other than intracranial hemorrhage.