Children's Hospital of Philadelphia leads or participates in hundreds of clinical research studies and clinical trials. Many of the studies that are currently enrolling patients can be found in this database. Use this finder to learn more about the purpose of these studies and clinical trials, find out who can participate, and tell us you're interested in enrolling. You can also look for studies that offer remote participation from home!
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Phase 1 open-label study to estimate the safety, manufacturing feasibility, and efficacy of intravenously administered, lentivirally transduced T cells expressing anti-CD123 in pediatric subjects with relapsed/refractory Acute Myeloid Leukemia (AML). Main study procedures include medical record review, apheresis, CART123 infusion, blood draws, physical exams, imaging, and disease assessments through bone marrows and lumbar punctures. If interested in the study or if you have any questions, please contact 267-426-0762.
This study wants to determine if racial differences in children that snore are related to factors that can be changed, such as child sleep habits, family factors, and provider unintentional racial bias. To accomplish this goal, we will compare measures of the sleepiness of children that snore and neurobehavioral impairments between Black and White children. We will also ask your child's healthcare provider to complete an implicit association test.
You are invited to take part in a voluntary research trial because you have type 2 diabetes and are between the ages of 10 and 17 years. You will be given a placebo or Ertugliflozin, which has been approved in the United States by the FDA for treatment of type 2 diabetes in adults but not in children. If you meet all criteria, you will be asked to come to CHOP for 8 study visits and have 4 phone calls for a total of approximately 60 weeks. Besides oral administration of the study product (a pill taken daily), the study procedures include: review of medical records, physical exam, EKG, blood and urine tests, renal ultrasounds, nutritional and physical activity counseling, and diary completion. You will be compensated for your time and effort committed to the study and reimbursed for your travel expenses.
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 purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease. This is an open-label study in which subjects will receive one dose of the Study Product. The goal of is to see if a single “dose” of the Study Product allows the body to increase the amount of hemoglobin F (HbF) while decreasing the effects of SCD. CTX001, which is created by changing (editing) the DNA in the subjects blood stem cells near a gene called BCL11A
You are invited to take part in a voluntary research trial because you have a genetic defect in vitamin D metabolism that causes elevated blood and/or urine levels of calcium and you are between the ages of 6 months and 65 years. You will be given a rifampin, which is an antibiotic drug that has been approved in the US by the FDA for treatment of many infections. Rifampin has not been approved for use in disorders of vitamin D metabolism. If you meet all criteria, you will be asked to come to CHOP for study visits and have phone calls for a total of approximately 48 weeks. Besides administration of the study product, the study procedures include: review of medical records, physical exam, ultrasound of the kidneys, blood and urine tests, tests of intestinal calcium absorption and nutritional assessment. Participants aged 3 years or older will also have a DXA scan performed. You will be compensated for your time and reimbursed for travel.
The Alterra study is for patients in need of pulmonary valve replacement. The Alterra Adaptive Prestent is a transcatheter stent designed for patients in need of a pulmonary valve replacement who do not want to have to undergo surgery, but have a pulmonary artery poorly suited for current approved transcatheter valves. Surgical placement of a new valve is the most common treatment for a nonworking pulmonary valve, however, there are many risks associated with open heart surgery and it is followed by months of recovery. We are conducting this study to see whether there is a less invasive approach by placing and Alterra Adaptive Prestent to make the pulmonary artery appropriate for transcatheter valve replacement. The Alterra Adaptive Prestent has not been approved by the FDA yet for commercial use and is only available by participating in the study. Please contact Dr. Matthew Gillespie (firstname.lastname@example.org) if you or your patient may be interested in participating in the trial of this device.
Cure Glomerulonephropathy Network (CureGN) is a multi-year study of 2,400 children and adults with four rare glomerular (kidney) diseases at over 50 centers in the US, Canada, and Italy. The purpose of CureGN is to create a patient research network to learn about four major causes of glomerular disease : 1. Minimal Change Disease (MCD) 2. Focal Segmental Glomerulosclerosis (FSGS) 3. Membranous Nephropathy (MN) 4. IgA Nephropathy (IgAN)
Children and adults with Beckwith-Wiedemann syndrome may be at increased risk for sleep apnea than other children. This risk may be due to a larger tongue size, but currently it is not known how to predict who need to be evaluated for sleep apnea. Some people with Beckwith-Wiedemann syndrome have tongue reduction surgery, but the effect of this procedure on sleep apnea is not known. This study seeks to understand how the upper airway anatomy contributes to sleep apnea in people with Beckwith-Wiedemann syndrome, including after tongue reduction surgery in people who have that procedure. The study includes a physical exam, medical record review (including surgical history), and upper airway MRI. For those participants who have not had a clinical sleep study, one will be done as part of this study.
Children and adults with PTEN Hamartoma Tumor Syndrome (PHTS) are at risk for specific long-term medical complications including autism spectrum disorder and cancer. While protocols for cancer screening exist, we do not yet have protocols for screening other complications of this syndrome, including neurobehavioral differences and immunologic changes. This pilot study will assess these outcomes in children and young adults with PHTS ages 1 to 20 years.