Dorzagliatin in Pancreatic Insufficient Cystic Fibrosis

Launched by UNIVERSITY OF PENNSYLVANIA · May 20, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called dorzagliatin to see how it affects people with cystic fibrosis (CF) who have trouble with their pancreas and may also have issues with their blood sugar. The trial will give some participants dorzagliatin and others a placebo (a harmless pill with no active ingredients) for a week to compare how their bodies respond. Researchers hope that dorzagliatin will help improve blood sugar control and insulin production after meals.

To be eligible for this study, participants must be at least 18 years old and have a confirmed diagnosis of cystic fibrosis with pancreatic insufficiency, meaning they require enzyme replacements to help digest food. They should also have abnormal blood sugar levels. Participants will need to commit to the study procedures for the entire duration, and women who can become pregnant must use reliable birth control during the study. It's important to know that not everyone can join; some health conditions might exclude individuals from participating. If you qualify and choose to participate, you will help researchers learn more about this promising treatment for people with cystic fibrosis and related diabetes.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Provision of signed and dated informed consent form.
• • 2. Stated willingness to comply with all study procedures and availability for the duration of the study.
• • 3. Male or female, aged ≥18 years on date of consent.
• • 4. Confirmed diagnosis of CF, defined by positive sweat test or CFTR mutation analysis according to CFF diagnostic criteria.
• • 5. Pancreatic insufficiency defined by clinical requirement for pancreatic enzyme replacement.
• • 6. Abnormal glucose tolerance defined by OGTT criteria for EGI, IGT, or CFRD, or diagnosed CFRD.
• • 7. There will be no restriction on enrollment of individuals with CFRD but without fasting hyperglycemia (fasting hyperglycemia is defined as fasting glucose ≥126 mg/dL)
• • a. Individuals with CFRD and fasting hyperglycemia (defined as above or by the use of basal insulin therapy) must also have a HbA1c ≤8% and a random (non-fasting) C- peptide ≥1.2 ng/mL \[15\].
• • 8. For females of reproductive potential: use of highly effective contraception method for the during of study participation; oral contraceptives, intra-uterine devices, Norplant®, Depo- Provera®, and barrier devices with spermicide are acceptable contraceptive methods; condoms used alone are not acceptable.
• Exclusion Criteria:
• • 1. Established diagnosis of non-CF diabetes (e.g. type 1 diabetes).
• • 2. Pregnancy or lactation; a negative urine pregnancy test will be required at enrollment.
• • 3. Pulmonary exacerbation requiring IV antibiotics or systemic glucocorticoids within 4 weeks prior to randomization.
• • 4. Treatment with either CYP3A4 inhibitors (e.g. ketoconazole, itraconazole, voriconazole, posaconazole, clarithromycin, indinavir, ritonavir, saquinavir, telithromycin, boceprevir, nelfinavir, telaprevir, conivaptan, nefazodone, etc.) or inducers (e.g. phenobarbital, other barbiturates, carbamazepine, phenytoin, rifampicin, dexamethasone, etc.).
• • 5. Use of herbal remedies, including St. John's Wort within 14 days prior to dosing.
• • 6. Change in CFTR modulator therapy in the previous 3 months.
• • 7. History of clinically symptomatic pancreatitis within the last year.
• • 8. Prior lung, liver or another solid organ transplant.
• • 9. Abnormal kidney function: creatinine \>2x upper limit of normal (ULN) or potassium \>5.5mEq/L on non-hemolyzed specimen.
• • 10. Abnormal liver function: persistent elevation of liver function tests \>2.0 times ULN.
• • 11. Uncontrolled hyperlipidemia: triglycerides \>500 or cholesterol \>250 mg/dl.
• • 12. Hyperuricemia: serum uric acid \>1.5 times ULN.
• • 13. Anemia: hemoglobin \<10 g/dL.
• • 14. History of any illness or condition that, in the opinion of the investigator might confound the results of the study or pose an additional risk to the subject.