Dorzagliatin for Cystic Fibrosis

Recruiting at 1 trial location

Overseen By Paola Alvarado, MSCR

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Pennsylvania

Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers, Herbal remedies

Disqualifiers: Non-CF diabetes, Pregnancy, Pulmonary exacerbation, others

Compensation: Varies

Number of Visits: Unknown

Duration: 7 days

15 Participants Needed

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. ^{1 2 3 4 5}

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. ^{2 4 6 7 8}

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

Provision of signed and dated informed consent form

Abnormal glucose tolerance defined by OGTT criteria for EGI, IGT, or CFRD, or diagnosed CFRD

I am willing and able to follow all study requirements.

Exclusion Criteria

I have been diagnosed with diabetes that is not cystic fibrosis-related.

Pregnancy or lactation; a negative urine pregnancy test will be required at enrollment

Hyperuricemia: serum uric acid >1.5 times ULN

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive dorzagliatin 75 mg or placebo twice daily for 7 days in a cross-over design

7 days

Follow-up

Participants are monitored for safety and effectiveness after treatment, including pharmacokinetic and pharmacodynamic assessments

2 months

What Are the Treatments Tested in This Trial?

Interventions

• Dorzagliatin

Trial Overview

The study tests dorzagliatin (75 mg twice daily) against a placebo over 7 days, measuring its effects on drug levels in the body, glucose control, insulin secretion, glucagon levels, and liver glycogen storage.

How Is the Trial Designed?

Group I: Dorzagliatin Experimental Treatment 2 Interventions

Dorzagliatin 75 mg orally twice daily for 7 days

Group II: Placebo Placebo Group 2 Interventions

matched-placebo orally twice daily for 7 days

Who Is Running the Clinical Trial?

University of Pennsylvania

Lead Sponsor

Trials

2,118

Recruited

45,270,000+

Citations

1.

withpower.com

withpower.com/trial/phase-1-fibrosis-6-2025-01d93

Dorzagliatin for Cystic Fibrosis · Info for Participants

We hypothesize that dorzagliatin administration will result in significant drug concentrations and improved glucose tolerance, early-phase insulin secretion, ...

2.

huamedicine.com

huamedicine.com/En/news-255

[News] Hua Medicine Announces 2025 Interim Results

A real-world study (BLOOM) is being conducted in 2,000 patients with Type 2 diabetes across 80 centers in China. BLOOM has already completed one ...

3.

drugpatentwatch.com

drugpatentwatch.com/p/drugs-in-development/drugname/Dorzagliatin?srsltid=AfmBOoqJMkfZCE12IUMdaWe43GIThzToAubDjK28rtwyOQybCj9w9J6M

Dorzagliatin clinical trial progress and patents: Drug ...

Dorzagliatin uniquely activates glucokinase, enhancing the body's intrinsic ability to regulate glucose, potentially offering both improved ...

4.

centerwatch.com

centerwatch.com/clinical-trials/listings/NCT06995651/dorzagliatin-in-pancreatic-insufficient-cystic-fibrosis

Dorzagliatin in Pancreatic Insufficient Cystic Fibrosis

We hypothesize that dorzagliatin administration will result in significant drug concentrations and improved glucose tolerance, early-phase ...

5.

prnewswire.com

prnewswire.com/news-releases/hua-medicine-announces-2025-interim-results-302540936.html

Hua Medicine Announces 2025 Interim Results

A real-world study (BLOOM) is being conducted in 2,000 patients with Type 2 diabetes across 80 centers in China. BLOOM has already completed one ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06995651

Dorzagliatin in Pancreatic Insufficient Cystic Fibrosis

This study is designed to determine the pharmacokinetic and pharmacodynamic response of dorzagliatin 75 mg twice daily following 7-day administration in ...

7.

drugpatentwatch.com

drugpatentwatch.com/p/drugs-in-development/drugname/Dorzagliatin?srsltid=AfmBOormyMYnd4YKEiSHGJP_LTMWxt3L0V0_Xo-MT7r_pRNlhJjtdKqF

Dorzagliatin clinical trial progress and patents: Drug ...

Risks include clinical trial failure, unforeseen safety issues (especially long-term cardiovascular effects), regulatory delays, and market ...

8.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11309680/

Efficacy and safety of dorzagliatin, a novel glucokinase ...

Regarding the safety profile of dorzagliatin, triglyceride levels were remarkable higher in the dorzagliatin group than in the placebo group [MD ...