Dorzagliatin for people with cystic fibrosis and pancreatic insufficiency

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled, crossover Phase 1 trial of dorzagliatin 50 mg given twice daily for 7 days in adults with cystic fibrosis and pancreatic insufficiency who have abnormal glucose tolerance or CFRD. Participants will receive dorzagliatin and placebo in separate treatment periods with washout between periods, and investigators will measure drug concentrations and glucose-related responses. Key pharmacodynamic measures include glucose tolerance during a standardized mixed-meal tolerance test, early-phase insulin secretion, glucagon suppression, and markers of hepatic glycogen storage. The study is conducted at the University of Pennsylvania clinical research sites and focuses on short-term safety and metabolic effects in this specific patient group.

Why it matters

Who can join: the full rules

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.

Where it's running

• Hospital of University of Pennsylvania
  Philadelphia, Pennsylvania, United States Recruiting
• University of Pennsylvania Center for Human Phenomic Science (CHPS)
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Principal investigator: Michael R Rickels, MD, MS — University of Pennsylvania
• Study coordinator: Paola Alvarado, MSCR
• Email: Paola.Alvarado@Pennmedicine.upenn.edu
• Phone: 215-746-2081

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.