NCT06995651 · University of Pennsylvania
Dorzagliatin in Pancreatic Insufficient Cystic Fibrosis
What this study is about
This study is designed to determine the how the drug moves through the body and how the drug affects the body response of dorzagliatin 50 mg twice daily following 7-day administration in individuals with pancreatic insufficient cystic fibrosis and abnormal glucose tolerance when compared to randomly assigned, where neither patients nor doctors know which treatment is given 7-day administration of placebo in a cross-over fashion. We hypothesize that dorzagliatin administration will result in significant drug concentrations and improved glucose tolerance, early-phase insulin secretion, glucagon suppression, and hepatic glycogen storage assessed during a standardized mixed-meal tolerance test.
View original scientific description
This study is designed to determine the pharmacokinetic and pharmacodynamic response of dorzagliatin 50 mg twice daily following 7-day administration in individuals with pancreatic insufficient cystic fibrosis and abnormal glucose tolerance when compared to randomized, double-blind 7-day administration of placebo in a cross-over fashion. We hypothesize that dorzagliatin administration will result in significant drug concentrations and improved glucose tolerance, early-phase insulin secretion, glucagon suppression, and hepatic glycogen storage assessed during a standardized mixed-meal tolerance test.
Interventions
DRUG
Dorzagliatin
Randomized, double-blind, cross-over study of Dorzagliatin 50 mg orally twice daily for 7 days compared to matched-placebo orally twice daily for 7 days.
DRUG
Placebo
Randomized, double-blind, cross-over study of Dorzagliatin 50 mg orally twice daily for 7 days compared to matched-placebo orally twice daily for 7 days.
Primary outcome measures
Drug concentrations (PK; Cmax)
Time frame: 2 months
Assessed by the peak glucose during a standardized mixed-meal tolerance test following 7-day administration of study drug
Glucose tolerance (PD)
Time frame: 2 months
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Provision of signed and dated informed consent form.
- Stated willingness to comply with all study procedures and availability for the duration of the study.
- Male or female, aged ≥18 years on date of consent.
- Confirmed diagnosis of CF, defined by positive sweat test or CFTR mutation analysis according to CFF diagnostic criteria.
- Pancreatic insufficiency defined by clinical requirement for pancreatic enzyme replacement.
- Abnormal glucose tolerance defined by OGTT criteria for EGI, IGT, or CFRD, or diagnosed CFRD.
- 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 \[15\].
- 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
- Established diagnosis of non-CF diabetes (e.g. type 1 diabetes).
- Pregnancy or lactation; a negative urine pregnancy test will be required at enrollment.
- Pulmonary exacerbation requiring IV antibiotics or systemic glucocorticoids within 4 weeks prior to randomization.
- 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.).
- Use of herbal remedies, including St. John's Wort within 14 days prior to dosing.
- Change in CFTR modulator therapy in the previous 3 months.
- History of clinically symptomatic pancreatitis within the last year.
- Prior lung, liver or another solid organ transplant.
- Abnormal kidney function: creatinine \>2x upper limit of normal (ULN) or potassium \>5.5mEq/L on non-hemolyzed specimen.
- Abnormal liver function: persistent elevation of liver function tests \>2.0 times ULN.
- Uncontrolled hyperlipidemia: triglycerides \>500 or cholesterol \>250 mg/dl.
- Hyperuricemia: serum uric acid \>1.5 times ULN.
- Anemia: hemoglobin \<10 g/dL.
- 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
- Philadelphia, Pennsylvania
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 9, 2026 · Source of record for eligibility and locations