Dorzagliatin for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This clinical trial aims to evaluate how well dorzagliatin works in people with cystic fibrosis who have pancreatic insufficiency and difficulty managing blood sugar. The study compares dorzagliatin with a placebo (a pill with no active drug) to determine if it can improve blood sugar levels and insulin function. It seeks volunteers with cystic fibrosis and pancreatic insufficiency who experience blood sugar control issues. Participants will take the medication or placebo for 7 days, and researchers will closely monitor their body's response to the treatment. As a Phase 1 trial, this research focuses on understanding dorzagliatin's effects in people, offering participants the opportunity to be among the first to receive this new treatment.
Will I have to stop taking my current medications?
You may need to stop taking certain medications, especially if you are using CYP3A4 inhibitors or inducers, or herbal remedies like St. John's Wort. It's best to discuss your current medications with the study team to see if any changes are needed.
Is there any evidence suggesting that dorzagliatin is likely to be safe for humans?
Research has shown that dorzagliatin has undergone safety testing in people. Some studies have found it to be generally well-tolerated. However, reports indicate higher triglyceride levels (a type of fat in the blood) in those taking dorzagliatin compared to a placebo. The long-term safety, particularly concerning heart health, remains under investigation. As this study is in an early phase, it focuses on understanding the body's processing of the drug and its immediate effects. This phase aims to gather early safety information, so not all side effects may be known yet.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatments for cystic fibrosis, which often involve managing symptoms and preventing complications through antibiotics, mucus-thinning medications, and enzyme supplements, Dorzagliatin offers a novel approach. Researchers are excited because Dorzagliatin targets glucose metabolism, which is not a common focus in current cystic fibrosis treatments. This unique mechanism could potentially address an underlying aspect of the disease rather than just its symptoms, offering a new avenue for managing cystic fibrosis more effectively. Plus, the convenience of an oral administration twice daily for just seven days makes it a potentially accessible option for patients.
What evidence suggests that dorzagliatin might be an effective treatment for cystic fibrosis?
Research has shown that dorzagliatin helps control blood sugar by activating an enzyme called glucokinase. This activation could improve how the body manages sugar and insulin in people with cystic fibrosis who have blood sugar issues. In studies with individuals who have Type 2 diabetes, dorzagliatin effectively managed blood sugar levels. Although specific research for cystic fibrosis is ongoing, the mechanism of dorzagliatin suggests it could aid in blood sugar control for this condition. Early results in other conditions indicate it might also support better overall metabolic health. Participants in this trial will receive either dorzagliatin or a placebo to evaluate its effectiveness in cystic fibrosis.24678
Who Is on the Research Team?
Michael R Rickels, MD, MS
Principal Investigator
University of Pennsylvania
Are You a Good Fit for This Trial?
This trial is for individuals with pancreatic insufficient cystic fibrosis and abnormal glucose tolerance. Participants should be able to undergo a mixed-meal tolerance test.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive dorzagliatin 75 mg or placebo twice daily for 7 days in a cross-over design
Follow-up
Participants are monitored for safety and effectiveness after treatment, including pharmacokinetic and pharmacodynamic assessments
What Are the Treatments Tested in This Trial?
Interventions
- Dorzagliatin
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Pennsylvania
Lead Sponsor