Clinical Research Finder

Children's Hospital of Philadelphia leads or participates in hundreds of clinical trials. Use this database to learn the purpose of these trials, find out who can participate and more.

Find a Clinical Research Study

141 - 150 of 152

A Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Subjects With Severe Sickle Cell Disease

The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 18 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

ILAR Reclassification Study

This study hopes to revise the currently used criteria for diagnoses of Juvenile Idiopathic Arthritis (JIA). This study aims to follow children newly diagnosed with JIA for up to five years. We will ask for a small blood sample for the first two visits, and for participants to fill out a back assessment. We will also record disease information from your medical record.

Upadacitinib trial for polyarticular JIA

The purpose of this study is to see if a new investigational drug (Upadacitinib) is safe and well tolerated and also to determine the appropriate dose for subjects with polyarticular course juvenile arthritis (pcJIA). The study is a two part study; in Part 1 subjects will be categorized based on age group, and the dose administered for seven consecutive days will be based on four different body weight categories. Subjects who complete Part 1 and are benefiting from study drug with no ongoing adverse events of special interest or serious adverse events, based on investigator's clinical judgment and with subject/family's agreement, will have the option to enroll in Part 2 to receive open-label upadacitinib. Participants will remain in part 2 for up to 156 weeks. Researchers will also be testing the pharmacokinetics (PK) of the study drug, which looks at how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body. Subjects will be asked to come into the Rheumatology clinic for blood and urine testing, a joint exam, and administration of study drug.

Distracted Driving App Study

This study invites teens 16-18 years old and their parent/guardian to partner with the Center for Injury Research and Prevention as we investigate a potential mobile app to prevent smartphone use while driving. Parents/caregivers and teens will both be participants in this study. Both parent and teen must have an Android smartphone. All participants will answer an initial questionnaire, install the investigational app on their Android smartphone, and answer additional periodic questionnaires.

Health disparities in pediatric sleep

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.

Car seat safety study

This study invites parents/guardians age 18-75 of children between 6 and 24 months of age to assess a new feature for child car seats. Participation consists of a brief online survey and a 1-time visit to the Center of Injury Research and Prevention during which the study team will take measurements of your child, and ask you to harness your child into different car seats and share your thoughts about the car seats and their features. Participants are compensated for their time and effort.

BoneGN

Glomerular disease is a major cause of chronic kidney disease associated with substantial morbidity; children and adults with glomerular disease face many threats to bone health. The purpose of this research study is to understand more about bone strength in children, adolescents and adults with Glomerular disease. This and future research will help us develop treatment guidelines for this population in order to promote bone health and prevent fractures and osteoporosis later in life. We will be evaluating bone quality in 150 participants with Glomerular disease and 120 healthy participants. The study requires 2 visits for a total of 3 hours and that the participants be 5 to 55 years old. The study procedures include blood draws, bone density measures and questionnaires. Compensation will be provided. If interested in participating contact the research coordinator at [TBD]

The CARRA Registry

This is an observational registry of children and young adults with pediatric-onset rheumatic conditions such as juvenile idiopathic arthritis (JIA) and Systemic Lupus Erythematosus (SLE) and SLE-like conditions. The registry will be used to answer questions about how safe medications are, how well medications work, and how well patients do over time. Patients who agree to participate will allow their health information to be used for research, complete a brief questionnaire during their clinic visits, and in some cases, be asked to give a sample of blood, urine, cheek swabs, stool, leftover (from clinical procedure) joint fluid, and leftover (from clinical procedure) tissue biopsy.

Vitamin E Supplementation in Hyperinsulinism/Hyperammonemia (HI/HA) Syndrome

Our team is working on a study to see if children with HI/HA Syndrome tolerate Vitamin E supplementation. This is an experimental, non-FDA approved use of Vitamin E which will lead to further studies to evaluate if Vitamin E can be used as a treatment for HI/HA. The study involves taking Vitamin E once a day for two weeks at home, completing a tolerability questionnaires, and two, one-day visits to the CHOP outpatient Center for Human Phenomic Science (one visit before and one visit after taking the Vitamin E supplement for two weeks). During each study visit, fasting oral protein tolerance will be performed and blood samples will be drawn. If you're interested in learning more, please contact us at ackermanna@email.chop.edu or rosenfelde@email.chop.edu.


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