We are conducting a research study for children ages 6-17 with patchy Alopecia Areata (AA). The purpose of this research study is to learn more about the safety, tolerability and efficacy of an investigational drug called Baricitinib. This study will compare the investigational drug to a placebo (inactive substance) to see how well the investigational drug works.

This study is available at the Minnesota State Fair. The purpose of this research is to better understand how children learn language. Child participants will complete problem solving and language-based tasks. Parents will complete two questionnaires about the child and their development. Results of this study will be used to help children with language-learning weaknesses.

This is a study about fitness and nutrition content on social media. You will be asked to complete a survey that should take 10 minutes or less. It will ask about your beliefs about fitness and nutrition, as well as online fitness, and nutrition content that your child may engage with. Study is taking place only on Monday 9/2 from 9 AM - 2 PM in the D2D building at the State Fair

The study drug FP-001 (Leuprolide mesylate) is being developed for children that are suffering from central (gonadotropin-dependent) precocious puberty (CPP). Leuprolide has been approved in the United States (US) and the European Union (EU) as treatment for prostate cancer already, and other forms of Leuprolide from other companies have been approved for the treatment of CPP. In this clinical study, Leuprolide will be used in the form of a 6-month depot injection.

The purpose of this study is to evaluate the safety and ability of a transplant with your own gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. The goal is to determine if a sufficient amount of hemoglobin that prevents red blood sickling can be produced after the gene modified stem cells are returned to your body. This study may provide information on the potential usefulness of bb1111 for treatment of sickle cell disease

The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.

This study is to see if giving the medications rituximab-pvvr (Ruxience®) followed by abatacept can help preserve remaining insulin secretion if given within three months of being diagnosed with type 1 diabetes (T1D).

The goal of this study is to learn more about how children (ages 5-8 years) describe their gender identities. We are also interested in learning more about the ways children think and develop. We will be giving children several tasks designed as games to assess flexibility in thinking, creativity, and the ability to take another person’s perspective.

This study will seek to learn about barriers to management of Type 2 diabetes for children ages 10-17. This study is recruiting children, and their parent/guardian, who are managing with oral medication or insulin.

This is a new type 1 diabetes onset study for ages 12-35 years old. We are looking at JAK inhibitor drugs to see if they can preserve beta cell function.

Although allogeneic hematopoietic cell transplant (HCT) is standard treatment for severe aplastic anemia, the use of the lower intensity conditioning drugs with a personalized dosing strategy, low dose total body irradiation (TBI) with dosing based on age and bone marrow abnormalities, and use of the drug cyclophosphamide early after transplant is a newer approach. We are studying whether this new approach is safer and more effective than our previous approach.

In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.

Primary Immune Regulatory Disorders (PIRD disorders) are a group of diseases that cause the immune system to function abnormally and cause infections, autoimmunity or inflammation that can begin early in life. PIRD is usually caused by changes in genes in DNA. Researchers are trying to learn what types of medical problems patients with PIRD have and how these respond to treatment. Researchers also want to learn which genes cause PIRD and how it can cause the medical problems of PIRD.

CGD disorders are a group of diseases that cause the immune system to function abnormally, leading to infections, autoimmunity and/or inflammation that can begin early in life. CGD is usually caused by changes in certain genes in DNA. Researchers are trying to learn what types of medical problems patients with CGD have and how these respond to treatment, including bone marrow transplantation and gene therapy. Researchers also want to learn which genes cause CGD and how specific gene abnormalities lead to medical problems.

Humanitarian use devices are medical devices approved by the FDA for the treatment of medical conditions affecting fewer than 4,000 patients per year. The FDA reviewed the safety of the device and determined that the probable health benefits outweigh the risks of injury or illness from its use. Effective treatment of symptoms, however, has not been studied in formal clinical trials. Medtronic DBS Therapy delivers electrical stimulation to areas in the brain to help control symptoms of various movement disorders. You may be a candidate for Medtronic DBS Therapy for Dystonia if you have been diagnosed with chronic, intractable (drug refractory) primary dystonia. You or your third party payer (health insurance, Medicare, Medicaid or other) must provide payment for hospital, office and other medical costs related to this therapy.

The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. We will also check if the study drug works to improve your kidney function, if has an impact on your daily life and the amount of the study drug in your blood over a period of time (called pharmacokinetics)

The purpose of this project is to learn about the stereotypes and discriminatory treatment that Black patients encounter to develop communication skills training and tools for clinicians that reduces the likelihood that Black patients feel stereotyped and discriminated against. We will ask you to participate in a two-hour focus group, either in-person or virtually.

This trial is an open label, randomized, stratified 2-arm Australian-led multicenter phase 3 clinical trial undertaken in two stages. Participants (age >= 11 years and <= 45 years) with intermediate and poor-risk metastatic germ cell tumors will be randomized into either a “standard BEP” group or “accelerated BEP” group. Participants will be assigned to the two treatment arms in a 1:1 ratio and evaluated weekly, and then for 5 years after completing the study to assess the long-term effects of the chemotherapy. Bleomycin, Etoposide, Cisplatin (BEP) administered 3-weekly x 4 remains standard 1st line chemotherapy for intermediate- and poor-risk metastatic germ cell tumours (GCTs). BEP is accelerated by cycling Cisplatin and etoposide 2-weekly instead of 3-weekly. The aim of this study is to determine if accelerated BEP is superior to standard BEP as first-line chemotherapy for intermediate and poor risk metastatic GCTs.

The primary purpose of this study is to gain information, especially disease free outcomes, using the tandem approach as compared to the historical information of using a single transplant. The data will be analyzed for transplant “milestones” such as time to blood count recovery and how patients are doing at 3 months and 1 year after the treatment. Participation in this study will not alter treatment or medical care. All information for this study will be collected from medical records.

The purpose of this research study is to collect timely and relevant data that will support the evolving research needs of the Inherited Retinal Disease community (IRD), in order to provide insights that can be used to improve patient management, and to inform development of future treatments. No visits, assessments, or procedures are mandated, and follow-up will be captured as part of your standard of care. The planned length of registry is of 8 years with a potential to extend the duration as needs evolve.

The clear layer at the front of the eye that covers the pupil and iris (colored part of the eye) is called the “cornea”. When the cornea is damaged, it normally heals within a few days but it may take up to 2 weeks depending on the size and depth of the defect (wound). Some corneal defects heal much slower than expected. A defect in the cornea that fails to heal within the normal time of 2 weeks despite using the best available medicines and procedures, is known as Persistent Corneal Epithelial Defect (or PCED for short). The purpose of this research study is to evaluate the safety, tolerability, and effectiveness (risks and benefits) of of NEXAGON ophthalmic gel for the treatment of PCEDs.

This is a study of an investigational drug (also known as the “study drug”) called allo-APZ2-OTS as a possible treatment for RDEB. The study drug contains allogeneic dermal ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs). These cells suppress inflammation and produce proteins that are important for wound healing. The study drug is expected to counteract local inflammation, improve wound healing and skin strength, and speed up wound closure.

The purpose of this research to enhance our understanding of adrenoleukodystrophy ALD and study biospecimens such assaliva, blood, urine and stool to identify potential biomarkers for early identification of dise. We invite people who have or are at risk to have ALD, including females who are known or at risk carriers of the mutation for ALD, to help us learn more.

The purpose of this study is to determine the dose of medication (Valacyclovir) needed to prevent an infant from developing herpes simplex virus (HSV) if the infant was potentially exposed to HSV at the time of delivery as they passed through the birth canal.

Researchers want to find out more about how physical, cognitive, and emotional factors affect eating.

This study is available only at the Minnesota State Fair. We want to learn more about children and teens strength, endurance, tiredness and level of physical activity. We want to find out how many times children and teens of different ages stand up and sit down in 30-seconds. It will take 10 minutes or less to take part in the study.

This phase II trial tests the safety and best dose of revumenib when given together with chemotherapy, and how well the treatment regimen works for infants and young children with leukemia that has come back (relapsed) or does not respond to treatment (refractory) and is associated with a KMT2A (MLL) gene rearrangement (KMT2A-R). Revumenib is an oral medicine that directly targets the changes that occur in a cell with a KMT2A rearrangement and has been shown to specifically kill these leukemia cells in test tubes and animals. Drugs used in chemotherapy, such as vincristine, prednisone, asparaginase, fludarabine and cytarabine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial is being done to find out if the combination of revumenib and chemotherapy may help to treat the cancer cells better than either treatment alone.

Skytrofa is approved in the U.S. for sale and use in children with growth hormone deficiency (GHD). This study is being done to find out how safe and useful Skytrofa is for long-term treatment. A child’s care will follow the normal treatment practices at the clinic. There is no new treatment or medicine involved and no additional visits will be performed.

To determine the prognostic factors for visual outcome for newly diagnosed NF1-OPGs. Hypothesis: Patients (<18 years of age) with tumors involving the optic tracts and/or radiations will demonstrate worse visual outcomes compared to those without optic tract involvement.

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.