Incretin Hormones for Cystic Fibrosis
Trial Summary
What is the purpose of this trial?
In recent years, diabetes has emerged as one of the most significant co-diseases that many Cystic Fibrosis (CF) patients develop. Type 1 (T1D) and Type 2 (T2D) diabetes results when either the body does not make enough insulin or the body does not respond correctly to this insulin, respectively. Insulin is a hormone which is made by cells in the pancreas and helps carry glucose (sugar) from the food we eat to the cells of the body for energy. While cystic fibrosis related diabetes (CFRD) has many features similar to both T1D and T2D, patients with CF may not have the same symptoms as either T1D or T2D patients. Currently, there is little understanding of CFRD and the best options for treatment remain unclear. 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. In particular, we plan to study the effects of incretin hormones that can enhance insulin production in CF patients. Enrollment is complete for the protocol as initially written. In order to further study the role of the incretin hormone on Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) function , we have received approval to extend our investigation to include the following study groups: * Cystic Fibrosis participants with normal glucose tolerance * Non-Cystic Fibrosis controls
Will I have to stop taking my current medications?
The trial protocol does not specify if you need to stop taking your current medications. However, you should not have had any changes in therapy, including antibiotics, within 6 weeks prior to the study.
What data supports the effectiveness of the drug for cystic fibrosis?
Research shows that GLP-1, a component of the drug, can enhance insulin secretion in people with cystic fibrosis, suggesting potential benefits. Additionally, semaglutide, a GLP-1 receptor agonist, has been used to help control blood sugar in cystic fibrosis-related diabetes, indicating its effectiveness in managing glucose levels.12345
Is the treatment with incretin hormones like tirzepatide and semaglutide generally safe for humans?
Tirzepatide and semaglutide, which are incretin hormones, have been studied for type 2 diabetes and are generally considered safe, with common side effects including nausea, vomiting, diarrhea, and constipation, especially at higher doses. Cardiovascular safety has been evaluated, showing no increased risk of major cardiovascular events.46789
How is the drug for cystic fibrosis unique?
The drug uses incretin hormones, GLP-1 and GIP, which are gut hormones that help control blood sugar by increasing insulin and decreasing glucagon. This approach is novel for cystic fibrosis as it targets glucose control, which is often impaired in these patients, and GLP-1 has shown potential benefits in enhancing insulin secretion specifically in cystic fibrosis.1231011
Research Team
Michael Rickels, MD, MS
Principal Investigator
University of Pennsylvania
Eligibility Criteria
This trial is for adults over 18 with cystic fibrosis and pancreatic insufficiency, who may have glucose tolerance issues but not severe diabetes. Women must test negative for pregnancy. Excluded are those with prior transplants, severe liver disease, abnormal kidney function, certain blood disorders or any condition that could interfere with the study.Inclusion Criteria
Exclusion Criteria
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive GLP-1 or GIP incretin hormone infusions and undergo GPA tests to measure insulin response
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- GIP (Hormone Therapy)
- GLP-1 (Hormone Therapy)
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Pennsylvania
Lead Sponsor
Children's Hospital of Philadelphia
Collaborator