Research Studies Finder

This study assesses whether early chelation with deferiprone is effective at delaying the progression of iron overload in infants and young children. Patients with beta thalassemia who are over 6 months old and less than 10 years old, receive regular transfusions, but who have not yet started chelation therapy, are eligible for this study. Subjects will be randomly assigned to take either deferiprone or a placebo three times daily for up to a year. Regular blood tests will be performed to track iron levels and monitor safety. Physical exams, blood and urine tests, and interviews will be done throughout the study to monitor safety and compliance with the medicine.

The purpose of this study is to evaluate the safety and pharmacokinetics (drug levels in the blood) of teplizumab in children less than 8 years old. The study will also assess if your child stays in Stage 2 T1D or progresses to Stage 3 T1D. Your child may join the study only if they are in Stage 2 T1D. Previous studies in adults and children older than 8 years of age suggest that taking teplizumab, a drug that affects your childs immune system, may stop, delay, or decrease the attack on the cells that make insulin. This slowing or blocking of the immune attack may reduce the risk of short-term and long-term consequences of T1D. To join this study, your child must have been diagnosed with Stage 2 T1D based on two positive T1D-related antibodies and confirmed dysglycemia (abnormal blood sugar levels) based on an oral glucose tolerance test, fasting plasma glucose test, or an increase in a blood test called the hemoglobin A1c. The study is open-label. That means that if your child qualifies for the study, your child will receive teplizumab. There is no placebo in this study.

This study will provide early access to an investigational drug which does not yet have marketing authorization for human use, Arimoclomol, for a group of patients suffering from Niemann-Pick Disease Type C that are not eligible for or able to participate in other clinical trials.

The purpose of this study is to help children who are tube-fed transition to eating orally. The goal is to evaluate the effectiveness of treatment with an experimental drug (megestrol) as part of a 24-week behavioral feeding protocol in transitioning from tube to oral feedings in a pediatric population. All children will receive feeding skills coaching to help them learn to eat by mouth, and children will follow a tube weaning protocol to transition from tube to oral feeds. More information can be found at www.ikaneat.org

Insulin therapy, commonly given as multiple daily injection therapy, is the only recommended treatment for Cystic Fibrosis Related Diabetes (CFRD). Traditional therapy for CFRD requires an intense daily effort related to diabetes care on top of the already burdensome management of Cystic Fibrosis. In this study, we will assess the safety and effectiveness of the iLet Bionic Pancreas, an automated insulin delivery system, which uses a continuous glucose monitor (CGM), an insulin pump, and a control formula that activates insulin delivery based on CGM glucose data.

Immune Thrombocytopenia (ITP) is a blood disorder in which the immune system produces antibodies that attack and destroy the body's own blood platelets. The Panzyga Study is looking at a potential medicine (experimental drug) to see if it can result in increasing platelet counts and may potentially treat ITP. You will be infused with the experimental drug once or twice during the treatment period and will be followed up for 32 days. Testing will include blood tests, urine tests, pregnancy tests and physical exams. You may be able to take part in the study if you are below 18 years of age and have been diagnosed with chronic ITP.

The goal of this study is to see if women with sickle cell disease (SCD) experience any difference in their pain while using the birth control injection, Depo-Provera, as compared to when not using this method. Depo-Provera is FDA-approved.

The duration of the study is 6 months with 6-7 in-person visits at the University of Pennsylvania that include a urine pregnancy test, blood draw, and surveys. The study duration is divided into a 3-month "baseline" period, where you would not use any hormonal contraception, and 3 months with the Depo-Provera injection. You would also complete weekly messages that ask about pain and menstrual cycle.

We are seeking women who are between the ages of 18 and 50 who have been diagnosed with SCD, have a regular menstrual cycle, and experience at least one vaso-occlusive pain episode per month.