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
Moderna mRNA Clinical Trial for Patients with GSD 1a
mRNA-3725 is not approved by the FDA; the FDA has allowed us to provide this investigational medicine to patients enrolled in this phase 1/2 trial. Phase 1/2 clinical trials like this one aim to test the safety, side effects, and best dose of an investigational medication. In the phase 2 part, participants usually receive the highest dose of treatment discovered in the phase 1 part. This is the first study in which humans are administered this study drug.
If successful, mRNA-3745 would enable certain organs in the body to effectively break down glycogen and avoid low glucose levels and starch intake. If mRNA-3745 could correct the cause of GSD1a, it would help keep your glucose levels where they need to be all the time.
Participation in this clinical trial could last up to approximately two years, depending on which stage you enroll in and whether or not you decide to participate in the long-term follow-up period of the study. Some of the visits in this trial will require you to stay overnight at the hospital, and some of the visits will require you to visit the hospital or doctor’s office. Most of the visits in this clinical trial can be done at your home, where a home health nurse will perform the assessments needed.
All study-related procedures, travel, and compensation are provided.
MRI Assessment of NAD+
MRI Muscle Metabolism in Different Age Groups
Prior to the completing the MRI scan, the study team will review the health status of individuals.
NCGD
PETITE-T1D
Previous studies in adults and children older than 8 years of age suggest that taking teplizumab, a drug that affects your child’s 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.
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.
Research Study for Individuals with Hyperinsulinism, Type 1 Diabetes Mellitus, and Healthy Children
Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in children with hyperinsulinism, including HI/HA syndrome, and type 1 diabetes mellitus. The study involves a one-day visit to The Children's Hospital of Philadelphia and The University of Pennsylvania for a specialized magnetic resonance imaging scan (MRI) of the brain, an electroencephalogram (EEG), blood draw, and completion of questionnaires to evaluate development and behavior. The entire study visit is expected to take about five hours. If you are interested in learning more, please contact us at HIResearch@email.chop.edu.
Research Study for Patients with GSD III
You or your child are being asked to take part in thisresearch study because you have Glycogen Storage Disesase Type III (GSD III).The purpose of this research study is to better understand blood sugarvariability, muscle strength and function, and impact of Glycogen StorageDisease Type III (GSD III) on overall health. You will not receive anytreatment for your GSD III as part of this study, but the information youprovide will be helpful for the study sponsor, Ultragenyx Pharmaceutical Inc.,to develop a new therapy for GSD III.
If you agree to take part, your participation will last for about26 weeks and will involve up to 2 study visits to the hospital along with up to 26 phone calls. There will be no changes to your treatment regimen for GSD IIIas part of this study.
As a participant in the research you will:
- come to the hospital for 1-2 in person visits tocomplete interviews and questionnaires,
- wear a blinded continuous glucose monitor (CGM) 3months and an unblinded CGM for 3 months,
- complete fingerstick blood sugar checks, maintain a nutrition diary for 3 or more weeksthroughout the study,
- maintain a hypoglycemia symptom diary, and
- upload CGM and HHG device data to astudy-provided laptop weekly.
The main risks of this study are from hypoglycemia related toyour diagnosis of GSD III and a loss of confidentiality. There are no directbenefit to you for participating in this study but the information collected aspart of this study will help researchers better understand GSDIII.