Define natural history, validate patient reported outcome and share knowledge on congenital disorders of glycosylation. We will recruit and enroll patients with CDG in this study evaluating clinical variation and natural history when a patient is being seen as part of routine clinical care.

The National Institutes of Health (NIH)-sponsored collaborative APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) is charged with prospectively assessing the effects of renal-risk variants (RRVs) in the apolipoprotein L1 gene (APOL1) on outcomes for kidneys from donors with recent African ancestry and the recipients of their kidneys, after deceased- and living-donor renal transplantation. For the purposes of APOLLO, recent African ancestry is defined as individuals with similar genetic make-up to those currently residing in Africa. APOLLO will also study the impact of APOL1 RRVs on the health of living kidney donors with recent African ancestry.

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.

Comparison of microfracture (knee surgery) with GelrinC (a glue like solution to augment the tissue regeneration of microfracture) for cartilage defects of the knee with historical control. This is a multi center, open label, controlled, non-randomized, double arm trial with prospective treatment arm (Gelrin C) and a historical control arm (microfracture). We hope to help the sponsor in learning more information regarding treatment of cartilage defects in the knee.

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.

A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.

This is a Phase III randomized, open label, multi-center study that will examine the impact of very low nicotine content (VLNC) cigarettes in a complex tobacco and nicotine product marketplace simulating a real world environment.

Study I8F-MC-GPGN (GPGN), also known as SURPASS-CVOT, is a Phase 3, event-driven, multicenter, international, randomized, double-blind, active comparator, parallel-group study. This study will assess the effect of QW tirzepatide (up to 15 mg) versus dulaglutide (1.5 mg) on CV outcomes when added to the standard of care in patients with T2DM with established cardiovascular disease and elevated risk for MACE.

• 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.

The objective of this study is to evaluate the efficacy and safety of sotatercept treatment (plus background P AH therapy) versus placebo (plus background PAH therapy) at 24 weeks in adults with PAH.

The primary objective of this study is to investigate the safety and efficacy of theta burst stimulation (TBS) for the management of post-traumatic headaches to improve outcomes and quality of life for individuals who have suffered a traumatic brain injury (TBI). To improve tolerability and logistical burden, we have developed a novel design whereby participants will receive three doses of TBS on alternate days of the week. This design will allow us to assess efficacy while leveraging an accelerated treatment course (nine stimulation sessions per week). We have three specific aims: Specific Aim 1. To determine the efficacy and safety of TBS for the treatment of post-traumatic headache among individuals who have sustained a mild TBI. Hypothesis 1a: TBS will be safe, well-tolerated, and reduce the number of headache days. Hypothesis 1b: TBS will improve function and quality of life outcomes. Specific Aim 2: To determine the efficacy and safety of an accelerated time-course of TBS for the management of post-traumatic headache. Hypothesis 2a: The accelerated-time course will be safe, welltolerated, and improve quality of life outcomes. Hypothesis 2b: The accelerated time-course will produce greater and faster improvement in headache symptoms than that reported in the literature for standard repetitive transcranial magnetic stimulation (rTMS) protocols. Specific Aim 3: To examine the durability of treatment response to accelerated TBS during a one-month observational period. Hypothesis 3: Accelerated TBS will result in enduring treatment response of posttraumatic headache symptoms over the follow-up period.

Drug study - Maribavir in HSCT patients with CMV infections

The Sponsor of this study, ModernaTX, is studying the mRNA-1273 vaccine for the prevention of COVID-19 in children. This study is being conducted to learn about the safety, any side effects, and how your child’s body responds to the study vaccine (the “immune response”).

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.

The overall goal of this study is to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, and to find out what effects, good and/or bad, the drug gilteritinib has when given with chemotherapy to children and young adults with newly diagnosed AML and the FLT3/ITD mutation or non-ITD FLT3 activating mutations.

-Characterize patient factors, such as pre-existing comorbidities, cancer type and treatment, and demographic factors, associated with short- and long-term outcomes of COVID-19, including severity and fatality, in cancer patients undergoing treatment. -Describe cancer treatment modifications made in response to COVID-19, including dose adjustments, changes in symptom management, or temporary or permanent cessation. -Evaluate the association of COVID-19 with cancer outcomes in patient subgroups defined by clinico-pathologic characteristics.

The study hypothesis is that the administration of Viralym-M to patients with virus-associated HC will demonstrate superiority for the time to resolution of HC (as measured by resolution of macroscopic hematuria) compared to patients treated with placebo. The primary hypothesis will be tested in patients with BKV viruria to demonstrate superiority over placebo in this population (BK Intent-to-Treat [ITT] Population). A supplementary analysis will be conducted in all patients with any viral-associated HC (BKV, JCV, AdV, EBV, CMV, and/or HHV-6) in order to evaluate efficacy in this broader population (ITT Population). Further detail is provided in the statistical section below and will be described in the Statistical Analysis Plan (SAP).

In this study, subjects who had a cataract removed but due to their eye structure, are not able to have a traditional intraocular lens (IOL) implanted, receive a special type of lens called the ARTISAN IOL. The objective of this study is to determine the effectiveness of the ARTISAN IOL and to precisely define the associated risks and, if possible, identify particular groups of patients who may be at high risk of developing complications resulting from the surgical procedure of implanting the lens.

This is a prospective, observational, non-interventional patient registry designed to document product safety and clinical outcomes for 10 years in patients treated with Cholbam/Kolbam, including those who have been using Cholbam/Kolbam for at least one month (existing users) and those who start Cholbam/Kolbam treatment at enrollment (new users). Patients who have been using Cholbam/Kolbam for less than one month and those who had previously been treated and who restart treatment will also be included but will not be counted in the existing or new users groups.

This is a randomized, double-blind, dose-ranging study of the efficacy and safety of DEX when used with PRO as needed, for procedural sedation of pediatric subjects ≥1 month to <17 years of age undergoing MRI scans in the US and Japan.

The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo-controlled clinical trial. All participants will receive close monitoring for progression of T1D. Participants will receive hydroxychloroquine or placebo and close monitoring for progression to Stage 2 (abnormal glucose tolerance) or Stage 3 (clinically overt) T1D. To assess the efficacy, safety and mode of action of hydroxychloroquine to prevent progression from Stage 1 to Stage 2 or Stage 3 of T1D.

Phase 1: To evaluate the safety and tolerability of JSP191 and to determine Phase 2 doses of JSP191 as a conditioning agent prior to allogeneic hematopoietic cell transplantation (HCT) in two populations of subjects with severe combined immunodeficiency (SCID): • SCID subjects with history of prior allogeneic HCT but with poor graft function • SCID subjects who are HCT-naïve Phase 2: • To evaluate the efficacy of JSP191 conditioning to enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism • To evaluate the efficacy of JSP191 conditioning to enable immune reconstitution determined by the production of naïve T cells

This is an open-label study for patients rolling over from VX18-445-104. This study will evaluate the long-term safety and efficacy of VX-445 combination therapy in subjects with CF.

This study is a randomized phase 3 study comparing selumetinib to Carboplatin and Vincristine (CV) in previously untreated NF1-associated LGG. This study will compare both the event-free survival (EFS) and visual functional outcomes between the 2 randomized arms.

The overall goal of this phase 3 non-inferiority study is to assess if selumetinib works as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with low-grade glioma (LGG).

This is a single site, 2-staged sequential multiple assignment randomized trial (SMART) that will systematically examine: 1) the optimal timing (12- versus 24 weeks) for identifying non-responders to lifestyle modification therapy (LSMT) before starting adjunct pharmacotherapy with phentermine and 2) for non-responders to LSMT+phentermine, the relative effect of adding topiramate to LMST+phentermine versus switching to LSMT+topiramate monotherapy. All participants will receive a total of 48 weeks of intervention.

This is a randomized, double-blind, placebo-controlled, parallel-group study with an optional open-label extension testing the safety and efficacy of inebilizumab in participants with Myasthenia Gravis.