Search Results

The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.

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 about finding out if long-term use of iptacopan (LNP023) is safe and can help others who have C3 Glomerulopathy (C3G) or idiopathic immune-complex membranoproliferative glomerulonephritis (IC-MPGN). This study if for people who have completed either the IC-MPGN Phase 3 CLNP023B12302 “APPARENT” study or the C3G Phase 3 CLNP023B12301 “APPEAR-C3G” study.

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.

This study will assess how effective and safe the use of the medication named Upadacitinib is for the treatment of signs and symptoms of severe hair loss in adults.

The goal of this observational study is to learn more about associations between respiratory infections, changes in the microbiome of the nose and upper throat, and juvenile idiopathic arthritis (JIA).

While hematopoietic stem cell transplant (HSCT) is an effective therapy, as many as 25% of patients develop problems with their lungs as a result of this treatment. Bronchiolitis obliterans (BO) is a type of lung injury after HSCT due to graft versus host disease. BO is commonly diagnosed late in patients, when lung injury is hard to treat and can be irreversible, leading to long-term lung disease or even death. The purpose of this research is to learn more about ruxolitinib as an early treatment for lung injury and BO after HSCT. Patients who are diagnosed with early lung dysfunction will be eligible for this research study.

This research is being done to learn if a study drug called vosoritide can help children who are shorter than should be for their age to grow.

A study to test a medicine (fitusiran) injected under the skin for preventing bleeding episodes in male adolescent or adult participants with severe Hemophilia.

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.

In this study, we want to find out more about secondhand smoke from shisha smoking in the home. We want to help families learn more about the risks of shisha smoke in the home and find ways to stop smoking at home.

This phase I trial tests the safety, side effects, and best dose of imetelstat in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) that has not responded to previous treatment (refractory) or that has come back after a period of improvement (recurrent). Imetelstat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as 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. Giving imetelstat in combination with fludarabine and cytarabine may work better in treating patients with refractory or recurrent AML, MDS, and JMML.

This study aims to reduce the radiotherapy (RT) dose necessary to successfully treat patients with intracranial germ cell tumors who are in a state of complete response (CR) following chemotherapy. In this study, a further reduction in whole ventricular irradiation (WVI) will be tested. The primary aim of the study is to determine whether 12 Gy of WVI, and 12 Gy tumor boost, would be successful. Event-free survival (EFS) in patients with central nervous system germinoma, who meet criteria for CR or continued complete response (CCR) following chemotherapy/second-look surgery, would be the primary measurement of success.

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 is a study of persons who do and do not stutter between ages 4 and 65 years. This study is trying to figure out how attention skills influence memory, language, and speech fluency skills.

The purpose of RESTORATiVE303 is to see if the study drug, which is called VE303, is safe and effective in preventing another episode of Clostridioides Difficile Infection (CDI). VE303 is an investigational drug that has 8 strains of live bacteria, called “commensals.” Commensals are the type of bacteria that live in harmony with the body, without harming health. These specific bacteria are often found in the intestines of normal, healthy people. They were selected for inclusion in VE303 because they rarely infect humans (mostly in very weakened patients), they do not carry any toxins that can make one sick, and they are not known to carry any risk of creating or spreading resistance to antibiotics.

To determine the prevalence, type, and severity of chronic health conditions (CHC) in survivors of Down syndrome-associated acute leukemia (DS-AL), and to compare CHC with frequency-matched DS individuals that have no cancer history.

The purpose of this study is to help investigators learn more about lung problems after bone marrow transplant including what are the best methods for diagnosing lung problems and follow-up care. The lung problems that may develop after transplant varies from patient to patient, and we don’t exactly know what risk factors influence who develops them or how patients respond to pulmonary (breathing system) therapies. Also, we wish to improve how we monitor lung function and quality of life after transplant, especially in children and young adults.

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 characterize balance and postural control in children with autism spectrum disorder and those with typical development. Participants will be asked to complete one lab visit. During this visit, parents will be asked to complete questionnaires about their family demographics and child behaviors, and their child's balance performance will be assessed using a virtual reality-based balance assessment. We are recruiting two groups of parents and children with ages ranging between 7-15 years to participate in this study. One group will include children with a diagnosis of autism spectrum disorders and their parents and the other group will be typically developing children who have no developmental, neurological or medical conditions and their parents.

This research study is for creating a registry of all ages with conditions in endocrine and both health. Registries are used very often these days by doctors and scientists to collect information and use to perform research into rare conditions. This registry will be part of a global registry, called "GloBE-Reg" with the University of Glasgow (Scotland) and with the University of MInnesota.

The purpose of this research study is to test the safety and effectiveness of Budesonide in low and high dose extended- release tablets in pediatric participants with active, mild to moderate Ulcerative Colitis and to evaluate the level of budesonide that remains in the blood after taking it. Participants will be asked to take an oral (by mouth) form of Budesonide or a placebo once daily for 8 weeks. A placebo is a tablet that does not contain any active study drug (Budesonide).

Nephrotic syndrome is a condition which affects the kidneys causing them to leak protein from the blood into the urine. Nephrotic syndrome is a disease that can improve (remission) and worsen (relapse) at different times throughout childhood. By collecting health information and laboratory samples, our goal is to learn more about these kidney diseases and find better ways to prevent and treat people with nephrotic syndrome. New knowledge will be shared with researchers and the public.

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse.

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

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 study is being conducted in order to further understand how we perceive the world, and if and how this may be different in individuals on the autism spectrum. We hope to further understand sensory processing differences in autism.

The purpose of this study is to develop a questionnaire specifically designed for children and adolescents, which will help health care providers to better measure how bothersome symptoms of cGVHD are for children and adolescents living with cGVHD. Another purpose of this study is to design a caregiver companion questionnaire, to allow caregivers to measure how bothersome the symptoms of cGVHD are for their child/adolescent.

The purpose of this study is to help investigators learn more about what are the best methods for diagnosing and monitoring lung problems.

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.