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Research Studies

Division of Endocrinology and Diabetes Research Studies

Find research studies available to children cared for by the Division of Endocrinology and Diabetes team.

Appointments and referrals

18F-DOPA PET/CT scan for Hyperinsulinism

This study is sponsored by Children's Hospital of Philadelphia Department of Radiology. The purpose of this research is to study 18F-L-Fluoro-DOPA (F-DOPA), a tracer, which is used to take pictures of the pancreas. This imaging test is called an F-DOPA PET scan. The F-DOPA tracer is used to highlight areas of the pancreas that make too much insulin on the PET scan images. The test includes a CT scan (computerized tomography) done at the same time on the same machine, to help define exactly where the highlighted area is located within the pancreas and determine the location of blood vessels and other vital structures. Who can participate:

  • Infants, children and adults with hyperinsulinemic hypoglycemia, diagnosed by a fasting test and/or response to glucagon stimulation
  • Any age, from birth on
  • Requiring surgery to remove part or all of the pancreas

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, MRI/Imaging Studies, One-Time Visit Studies

Bone Health in DMD

Dr. Weber at The Children's Hospital of Philadelphia is looking for boys and young men with Duchenne Muscular Dystrophy between 5-20 years old to participate in a study to learn more about bone health. Study participation includes completing two study visits: one baseline visit and one 12-month follow-up visit. To be eligible, participants must have used steroids for a minimum of 12 months by the time they complete their baseline visit. Eligible study participants will be paid for each study visit. If interested in hearing more about this study or being screened for eligibility, please contact Dr. David Weber at davidweberresearch@chop.edu.

Phase: Pilot

Actively recruiting: Yes

Category: Children

Conditions: Muscular Dystrophy

CFRD-Pilot Study

The purpose of this research study is to examine and understand the various mechanisms that contribute to CFRD and gain a better understanding of potential means to treat CFRD. Participation will include 2-3 visits, home monitoring, and will last about 2 years.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children, Healthy Controls

CODA: COVID-19 and Diabetes Assessment

The goals of this research study are to find out how having COVID-19 recently affects blood sugar, inflammation, and insulin in people with type 1 and type 2 diabetes. We also want to see if COVID-19 changes blood vessel health or makes blood clot more easily. We will also look at how things like genes, lifestyle, and environment might affect these issues.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

Conditions: Type 1 Diabetes, Type 2 Diabetes

DISCOVERY Study

The DISCOVERY Study is a new, national effort to learn about the causes of type 2 diabetes in children. DISCOVERY is for children 9-14 years old who are at higher risk of developing type 2 diabetes. We want to figure out why some children get type 2 diabetes and others do not.

Phase: N/A

Actively recruiting: Yes

Category: Children

HM15136 for the Treatment of Congenital Hyperinsulinism Research Study

The purpose of this study is to look at the safety and tolerability (how well you will react) of the study drug (HM15136) and to determine if it is effective for the treatment of Congenital Hyperinsulinism (HI). HM15136 is an experimental drug which is not approved by the FDA for the treatment of HI. HM15136 is stable and has a long effect in the body, only requiring it to be given (by injection) once a week. HM15136 is designed to act like glucagon, a hormone produced by the pancreas that helps the body maintain normal blood sugar levels by increasing the glucose produced in the liver and breaking down glycogen (a form of stored glucose) into the usable glucose form.

Participation lasts for up to 22 weeks and will involve up to 13 study visits. Study procedures include taking the study drug for 8 weeks, having electrocardiograms, wearing a continuous glucose monitor (CGM) and using a handheld glucose meter, having blood and urine tests, and completing an electronic diary.

The main risks of this study include: nausea, vomiting, diarrhea, abdominal pain, injection site reactions (swelling, itching, bleeding, or pain), loss of appetite, weight loss, high blood sugar. You may benefit if the study drug proves to be more effective than your current treatment plan in treating low blood sugar.

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Congenital Hyperinsulinism

Phentermine/Topiramate in Hypothalamic Obesity

The purpose of this study is to evaluate how safe and effective the drug Qsymia (Phentermine/Topiramate of Ph/T) is for children, adolescents, and young adults with hypothalamic obesity. We will also evaluate if Ph/T helps with weight loss and changes in hunger in patients with hypothalamic obesity.

If you are determined to be eligible, then your participation will last for approximately 8 months. Participants will be asked to complete up to 5 in-person visits and 5 telephone safety check-ins. During the study you will be randomly assigned to study drug (phentermine/topiramate) or placebo for 28 weeks.

You will complete study procedures including blood tests, ECGs, mobile cardiac outpatient telemetry, body composition scan, and questionnaires. Compensation and reimbursement of limited travel expenses is available.

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Hypothalamic Obesity

Utilizing the Bionic Pancreas in Cystic Fibrosis Related Diabetes

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.

Phase: Phase III, Phase IV

Actively recruiting: Yes

Category: Adults, Children

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