Cystic Fibrosis-related DiabetesPhase 2NCT04530383
Metformin for People With CFRD on CFTR Modulator Therapy to Improve Ion Channel Function
A Phase 2 study testing Metformin Hydrochloride in people with Cystic Fibrosis-related Diabetes and Cystic Fibrosis, run by University of Kansas Medical Center at 1 site in United States.
Trial summary
The purpose of this study is to assess the efficacy of metformin to improve airway ion channel function in those with CF-related diabetes (CFRD) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis-related Diabetes, Cystic Fibrosis
- What is being tested?
- Metformin Hydrochloride
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Feb 2022to 15 Jul 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age >18 years with a prior diagnosis of CF.
- Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor or vanzacaftor/tezacaftor/deutivacaftor for 30 days prior to day 0
- Diagnosis of CFRD with evidence of continued glucose intolerance at least 6 months after starting qualifying modulator therapy will be based upon one of the following:
- Insulin use
- Hemoglobin A1C >6.5%
- Fasting glucose >126 mg/dl
- Non-fasting glucose >200 mg/dl (random or as part of a 2-hr OGTT)
Who cannot join
- Prior lung or liver transplant
- Use of supplemental oxygen
- BMI <18
- CF pulmonary exacerbation requiring hospitalization or intravenous antibiotics in the preceding 30 days
- Systemic corticosteroid or regular non-steroidal anti-inflammatory use in the preceding 30 days
- Cardiac, renal (creatinine clearance <45 mL/minute), neurologic, psychiatric, endocrine or neoplastic diseases that are judged to interfere with participation in the study
Show all 14Show fewer
- Alanine aminotransferase, aspartate aminotransferase or alkaline phosphatase >1.5X the upper limit of normal; bilirubin >3 mg/dL
- Taking medications that interact with metformin.
- Vitamin B12 deficiency
- Pregnancy or lactation
- Inability or unwillingness to comply with an approved contraceptive method during the study period (females of childbearing age)
- Use of medications known to be strong CYP inducers or moderate to strong CYP inhibitors
- In the opinion of the investigator any severe or acute or chronic condition or laboratory abnormality that may increase the risk associated with trial participation or make the subject inappropriate for enrollment
- Participation in another interventional trial that, in the opinion of the investigator, has the potential to affect the primary outcome
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Matthias A Salathe, M.D., 9135886000, msalathe@kumc.edu
Carolina Aguiar, 9139459295
About this study
Official title: An Open Label Clinical Trial of Metformin in Those With CFRD on CFTR Modulator Therapy to Improve Ion Channel Function
The purpose of this study is to assess the efficacy of metformin to improve airway ion channel function in those with CF-related diabetes (CFRD)
Read the detailed description
Up to 30 patients with CFRD on highly effective CFTR modulator therapy who meet criteria and agree to participation in the study will be placed on metformin. There will be a dose-escalation starting with 500mg twice daily for a week, followed by 500mg in the AM and 1000mg in the PM for another week and finally followed by 1000mg twice daily for 14 weeks. To minimize risk of B12 deficiency, a known side effect of long-term metformin use, we will also provide a supplement of 1000 μg oral cyanocobalamin daily for the duration of the trial
- Conditions
- Cystic Fibrosis-related Diabetes, Cystic Fibrosis
- Being tested
- Metformin Hydrochloride
- Keywords
- Metformin
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Metformin HydrochlorideDrug
Also called Glucophage
1000 mg twice daily
| Study group | Role | What they receive |
|---|---|---|
| Metformin dose regimen A | Experimental | patients with CFRD on highly effective CFTR modulator therapy who meet criteria and agree to participation in the study will be placed on metformin. There will be a dose-escalation starting with 500mg twice daily for a week, followed by 500mg in the AM and 1000mg in the PM for another week and finally followed by 1000mg twice daily for 14 weeks. Receives: Drug: Metformin Hydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in BK channel gene expression
Levels of LRRC26 (big potassium channel regulatory subunit) mRNA will be measured by polymerase chain reaction from nasal cells acquired via brushing
Measured: Baseline through week 14 of metformin treatment
Secondary outcomes (9)
Change in BK function, as measured by nasal potential difference testing
Nasal potential difference testing measures direct BK current in the nasal epithelium, with greater current indicating greater BK function
Measured: Baseline through week 14 of metformin treatment
Change in receptor for receptor for advanced glycation end products (RAGE) gene expression
Levels of RAGE mRNA will be measured by polymerase chain reaction from nasal cells acquired via brushing
Measured: Baseline through week 14 of metformin treatment
Change in advanced glycation end products (AGE)
Plasma levels of AGE, receptor for AGE (RAGE), soluble RAGE and S100A12 will be quantified by ELISA
Measured: Baseline through week 14 of metformin treatment
Change in sweat chloride
Measured as a secondary marker of CFTR function, with lower levels indicating greater CFTR function
Measured: Baseline through week 14 of metformin treatment
Change in lung function
Measured by percent predicted forced expiatory volume in one second captured on spirometry (FEV1)
Measured: Baseline through week 14 of metformin treatment
Change in Quality of Life (CFQ-R)
Measured by Patient Reported Outcome measurement tool called CFQ-R (validated)
Measured: Baseline through week 14 of metformin treatment
Change in airway inflammatory markers
Inflammatory markers (interleukin-1beta, interleukin-6, interleukin-8, transforming growth factor beta1, tissue necrosis factor-alpha, matrix metalloproteinase-9 and cyclooxygenase-2) collected from nasal fluid will be measured by enzyme linked immunosorbent assay (ELISA)
Measured: Baseline through week 14 of metformin treatment
Safety of metformin
Number of adverse events during study period
Measured: Baseline through week 14 of metformin treatment
Pharmacokinetics of metformin
Plasma levels of metformin will be quantified by liquid chromatography-mass spectrometry
Measured: Week 14 of metformin treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2020
- Study start14 Feb 2022
- Primary completion30 Dec 2027 (estimated)
- Study completion15 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis-related DiabetesStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Matthias Salathe, MD, University of Kansas Medical Center
- Investigators
- Matthias A Salathe, M.D., Principal investigator, Professor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04530383
- Sponsor's protocol ID
- STUDY00146063/STUDY00161734
- Other IDs
- R01HL157942-01
- Status last verified
- Apr 2026
- First submitted
- 24 Aug 2020
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Clinical Trial of Metformin in Those With CFRD on CFTR Modulator Therapy to Improve Ion Channel Function
- ClinicalTrials.gov ID
- NCT04530383
- Acronym / sponsor ID
- STUDY00146063/STUDY00161734
- Conditions
- Cystic Fibrosis-related Diabetes, Cystic Fibrosis
- Keywords
- Metformin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Study start
- 14 Feb 2022 (actual)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 15 Jul 2028 (estimated)
- First posted
- 28 Aug 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the efficacy of metformin to improve airway ion channel function in those with CF-related diabetes (CFRD)
Detailed description
Up to 30 patients with CFRD on highly effective CFTR modulator therapy who meet criteria and agree to participation in the study will be placed on metformin. There will be a dose-escalation starting with 500mg twice daily for a week, followed by 500mg in the AM and 1000mg in the PM for another week and finally followed by 1000mg twice daily for 14 weeks. To minimize risk of B12 deficiency, a known side effect of long-term metformin use, we will also provide a supplement of 1000 μg oral cyanocobalamin daily for the duration of the trial
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Metformin dose regimen A | Experimental | Drug: Metformin Hydrochloridepatients with CFRD on highly effective CFTR modulator therapy who meet criteria and agree to participation in the study will be placed on metformin. There will be a dose-escalation starting with 500mg twice daily for a week, followed by 500mg in the AM and 1000mg in the PM for another week and finally followed by 1000mg twice daily for 14 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age >18 years with a prior diagnosis of CF.
- Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor or vanzacaftor/tezacaftor/deutivacaftor for 30 days prior to day 0
- Diagnosis of CFRD with evidence of continued glucose intolerance at least 6 months after starting qualifying modulator therapy will be based upon one of the following:
Key exclusion criteria (14 listed)
- Prior lung or liver transplant
- Use of supplemental oxygen
- BMI <18
- CF pulmonary exacerbation requiring hospitalization or intravenous antibiotics in the preceding 30 days
- Systemic corticosteroid or regular non-steroidal anti-inflammatory use in the preceding 30 days
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Metformin HydrochlorideAlso: Glucophage1000 mg twice daily | Drug | Metformin dose regimen A |
Primary outcome
Change in BK channel gene expression
Time frame: Baseline through week 14 of metformin treatment
Levels of LRRC26 (big potassium channel regulatory subunit) mRNA will be measured by polymerase chain reaction from nasal cells acquired via brushing
9 secondary outcomes
Change in BK function, as measured by nasal potential difference testing
Time frame: Baseline through week 14 of metformin treatment
Change in receptor for receptor for advanced glycation end products (RAGE) gene expression
Time frame: Baseline through week 14 of metformin treatment
Change in advanced glycation end products (AGE)
Time frame: Baseline through week 14 of metformin treatment
Change in sweat chloride
Time frame: Baseline through week 14 of metformin treatment
Change in lung function
Time frame: Baseline through week 14 of metformin treatment
Change in Quality of Life (CFQ-R)
Time frame: Baseline through week 14 of metformin treatment
Change in airway inflammatory markers
Time frame: Baseline through week 14 of metformin treatment
Safety of metformin
Time frame: Baseline through week 14 of metformin treatment
Pharmacokinetics of metformin
Time frame: Week 14 of metformin treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Matthias A Salathe, M.D. principal investigator
Professor
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Metformin Hydrochloride in people with Cystic Fibrosis-related Diabetes and Cystic Fibrosis, run by University of Kansas Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Matthias A Salathe, M.D. (9135886000, msalathe@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in BK channel gene expression (time frame: Baseline through week 14 of metformin treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.