The goal of this study is to investigate the efficacy and safety of Canagliflozin (which is experimental) in children and adolescents ages 10 to 18 years old with Type 2 diabetes and poor control (i.e., an HbA1c of 6.5% to 10.5%). Study drug or placebo would be taken alone or with metformin or insulin as needed. Monitoring tests include blood, urine, physical exams, vital signs, blood sugar and ketone monitoring, diabetes management counseling.

The aim of this study is to evaluate safety, and tolerability of insulin LY3209590 following a single dose given to children with type 2 diabetes mellitus (T2DM). This will be the first study to evaluate LY3209590 in pediatric patients with T2DM.

Screening:

All participants will be screened up to 28 days prior to dosing.

Treatment and Assessment Period:

On Day 1, participants will receive a single 100-Unit subcutaneous dose of LY3209590 at the clinical research unit.

Follow up research visits will be performed on Days 3, 5, 7, 15, 29, 43, and 65. Blood sampling and safety assessments will be performed at each in clinic visit. Before each study visit, subjects will need to fast for at least 8 hours.

A follow-up telephone call will be performed on approximately Day 72. The total time for participation is the study is about 100 days.

This study is a randomized, double-blind, placebo-controlled study that will evaluate the safety and efficacy of dasiglucagon in children between the ages of 7 days and 12 months who have congenital hyperinsulinism. Dasiglucagon will be given as a subcutaneous infusion while patients are admitted to the hospital. Other treatments for hyperinsulinism may be changed as allowed while maintaining safe blood sugar levels. For the second part of the study, patients may be able to go home on the medication.

The goal of this study is to identify a safe dosing regimen for siplizumab in participants with type 1 diabetes. Siplizumab is a drug used to help treat recent onset type 1 diabetes mellitus (T1DM). This study is interested in participants aged 18-45 years with T1DM, who are within 18 months of receiving their diagnosis. Participants will be randomized (selected at random by the study team) to one of four possible treatment groups. All groups will receive weekly siplizumab doses administered subcutaneously (injected into the body through the skin) for a total of 12 weeks.

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa). The FDA has not approved this drug for the treatment of GSDIa but has approved us to use this drug as part of this clinical trial. The purpose of this research study is to see if DTX401 can help people with GSDIa have normal blood sugar levels and fewer episodes of hypoglycemia. DTX041 works by delivering copies of the G6PC gene to your liver through a one-time infusion. Participation in this study will last for 2 years and will involve 30 study visits. It is possible that treatment with the study drug infusion may improve your blood sugar levels. Travel costs will be covered by the study and you will receive compensation for participating.

This study will evaluate the safety and effectiveness of the MiniMed 780G insulin pump system used in combination with the DS5 CGM in type 1 diabetic pediatric participants in a home setting.

Our team has partnered with Moderna on a clinical trial for patients with glycogen storage disease type 1a (GSD1a). We would like to invite you to take part in a clinical trial for an mRNA investigational drug that could potentially correct the cause of GSD1a, by teaching your body to break down glycogen, correct low glucose, and avoid starch intake. The purpose of this trial is to see if a new investigational mRNA study drug, called mRNA-3745, is safe and if it works in the way researchers expect. mRNA technology uses messenger ribonucleic acid (mRNA), an instructional molecule that naturally occurs in the body and carries information to cells. In this case, we are studying if mRNA-3745 given through an IV infusion can instruct the body to make the protein that is missing in people with GSD1a.

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 doctors 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.

The purpose of this study is to find out if an investigational drug called setmelanotide (RM-493) can help control body weight in people with certain genetic modifications in their genes that play a key role in the regulation of body weight. Males and females, age 6 to 65, with obesity and certain genetic variants (MC4R pathway) may be eligible to participate. If you are confirmed eligible for the study, then participation will last up to 64 Weeks. Participants will complete Screening (Up to 8 Weeks), Double-Blind Treatment (Up to 52 Weeks), and Follow-Up Period (4 Weeks). Participants will complete research procedures including physical exams, blood draws, ECGs, and questionnaires. Participant will also need to self-inject the study drug once daily for the duration of their participation.

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.