The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

This study is looking at the liver function of people who have bariatric surgery. People who want to be in this trial must be obese, have abnormal liver function tests & prediabetes or type 2 diabetes. The study includes assistance with diet and exercise for a year after surgery.

This study will test whether adding in daily weighing (5-7 days per week) is helpful in weight loss. In adults, it has been found to be helpful to have individuals who are obese to be more successful with their weight loss efforts if they weigh themselves at home every day. We want to see if this could be helpful in children as well. This research will last for about 12 weeks (3 months).

This study is being done to look at how pregnancy affects the health of women with cystic fibrosis (CF) and to look at the health of babies born to women with CF. It is expected that most but not all women will be taking the newest CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator medication called TRIKAFTA®. It is not known how this medication will affect the health of mother and infant.

This study is to investigate if robotic assisted laparoscopy (small incision surgery), is worse than open surgery (otherwise known as a laparotomy) when performing a radical hysterectomy for cervical cancer. Previous research has been done and shown that patients receiving laparoscopy had an increased risk of recurrence of their cervical cancer. But since that time a lot has been learned and improvements have been made, hence why we are relooking at the differences between the two surgical approaches.

The purpose of this research is to compare any good and bad effects of using radium-223 along with docetaxel chemotherapy (at a lower dose) treatment versus using docetaxel alone (at the usual dose). The addition of radium-223 to docetaxel could be a better cancer treatment than just docetaxel alone, but it could also cause additional side effects. This study will allow the researchers to know whether this different approach is better, the same, or worse than the usual approach.

A Phase I/II trial of single agent intravenous CBL0137 in pediatric patients (≥ 12 months and ≤ 30 years) with relapsed/refractory solid tumors, including CNS tumors and lymphoma.

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.

This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients (≥1 year and < 22 years ) with CD22 positive B acute lymphoblastic leukemia that has come back or does not respond to treatment. Immunotoxins, such as inotuzumab ozogamicin, are antibodies linked to a toxic substance and may help find cancer cells that express CD22 and kill them without harming normal cells.

The purpose of this study is to learn whether the use of Pregnyl with the drug ruxolitinib is able to reduce the need for high dose steroids to treat severe acute Graft versus Host Disease (GVHD).

This study aims to identify independent blood-based risk factors for postoperative complications and near-term events among patients undergoing CABG surgery. In particular, this study will be using proteomics, the study of all proteins produced by the cells found in blood, as well as genetic analysis to identify potential predictive markers of postoperative complications. We will collect blood samples from before and after surgery. This study does not involve any medical treatment other than the one prescribed by your doctor, nor does it involve any additional medical procedures.

This study is intended to find the safest dose of a new combination of drugs (ALX148 and T-DXd) and to start to determine how effective it is at treating advanced or metastatic breast cancer. This study is an addition to the ongoing ISPY study program.

This phase III trial compares the effect of open thoracic surgery (thoracotomy) to thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery is a type of surgery done through a single larger incision (like a large cut) that goes between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest surgery where the doctor makes several small incisions and uses a small camera to help with removing the cancer. This trial is being done evaluate the two different surgery methods for patients with osteosarcoma that has spread to the lung to find out which is better.

Prospective study to compare safety of upper versus lower extremity injection agitated saline (bubble study) using echocardiography of the heart.

This research is being done to collect blood and placenta samples to better understand how pregnancy impacts the health of mother and child. The goal of this research is to better understand what causes some pregnancy complications and how this impacts the longer term health of mothers and children.

The purpose of our research study is to increase our understanding on the human lung immune response to infection. We aim to use this knowledge to develop novel approaches on the prevention and treatment of lung infections. To accomplish this, we plan to obtain cells from the airways and blood of healthy volunteers to perform immune experiments.

The purpose of this study is to understand whether blood levels of adalimumab (brand name: Humira) can be used to predict how well uveitis will respond to adalimumab.

The Muscular Dystrophy Association (MDA) wants to collect information about individuals with neuromuscular disease to better understand the disease progression and ultimately improve the medical care, quality of life, and survival of those with neuromuscular disease. To collect this information, MDA has created a data registry called the Neuromuscular Observational Research Data Hub (referred to as the “MOVR Data Hub”).

The purpose of the study is to test new biomarkers of amyotrophic lateral sclerosis (ALS) and primary lateral sclerosis (PLS) using MRI scans at 3 tesla (3T). Identifying biomarkers of a disease can lead to a better understanding of the disease as well as improved treatments.

This research is being completed to examine the cells, brain imaging, and speech in individuals with REM Sleep Behavior Disorder who are taking serotonergic medications such as Citalopram, Escitalopram, Fluoxetine, Fluvoxamine, Paroxetine and Sertraline. The purpose of examining these is to try and see if we can predict signs of Dementia with Lewy bodies (a progressive form of dementia with an increase in decline of thinking, reasoning, and other functions). This may benefit others by enabling us to diagnose Dementia with Lewy Bodies sooner rather than later.

The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug. We are doing this research to find out if different treatments have an effect on Amyotrophic Lateral Sclerosis (ALS). We also want to find out if these treatments are safe to take without causing too many side effects.

We are studying a new drug, INCB054707, used to treat people who have hidradenitis suppurativa which is a chronic skin condition characterized by lumps or boils in places such as the armpits or groin. The skin lesions develop because of inflammation of the follicle. We are studying two doses of the drug and we will compare the effectiveness and side effects that occur. We will also have a group that receives an inactive medication (placebo). After the first 12 weeks of taking the drug or placebo, all participants will receive the active drug. The study will last for about 62 weeks.

This study will be measuring brain activity using EEG and assessing motor skills and speech in children following cancerous brain tumor resection. No direct cancer treatments or objectives are being targeted.

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.

This study is being done to see how safe an investigational drug is and how well it will work to help people with obesity, or overweight with weight-related conditions like hypertension, dyslipidemia, obstructive sleep apnea, non-alcoholic fatty liver disease, or diabetes. If you qualify to be in the study, you will be given frequent lifestyle and behavioral counseling for the first 12 weeks of the study. The counseling will consist of advice on physical activities and dietary advice on healthy eating. During the treatment period, you will receive either tirzepatide or placebo. Placebo is a solution that looks like the study drug but has no medicine. The chance that you will get the study drug is 2 in 3. This phase will last about 72 weeks.

This study will provide the most accurate and reliable estimates to date on disease progression and clinical events in evolving chronic pancreatitis. We also hope to develop from the results of this study some lab tests that will help us with early diagnosis of chronic pancreatitis and also to discover any genetic factors that may affect your chances of developing chronic pancreatitis.

Levosimendan has not been approved by the FDA to treat people who have PH-HFpEF or approved to be taken by mouth (orally). In this study, we will measure the amount of levosimendan in blood at various times and evaluate the change in participants 6-Minute Walk Distance.

This is a 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.

We are looking at how well letrozole with or without paclitaxel and carboplatin works in treating patients with stage II-IV low-grade serous carcinoma of the ovary, fallopian tube, or peritoneum. Letrozole is an enzyme inhibitor that lowers the amount of estrogen made by the body and may stop the growth of tumor cells that need estrogen to grow. We will compare the effectiveness of the two different treatments.

Currently, more than half of all women who are able to quit smoking cigarettes during pregnancy start smoking again within six months after they give birth. We want to find out if the drug bupropion (a commercially-available medicine) can help women who quit smoking during pregnancy to continue not smoking after they give birth. All study visits can be completed either in-person or virtually.