Metformin for People With CFRD on CFTR Modulator Therapy to Improve Ion Channel Function

NCT04530383 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: STUDY00146063/STUDY00161734R01HL157942-01
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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)

Conditions

Cystic Fibrosis-related Diabetes; Cystic Fibrosis

Keywords

Metformin

Outcome Measures

Primary Outcomes (1)

• 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

  Baseline through week 14 of metformin treatment

Secondary Outcomes (9)

• Change in BK function, as measured by nasal potential difference testing

  Baseline through week 14 of metformin treatment

• Change in receptor for receptor for advanced glycation end products (RAGE) gene expression

  Baseline through week 14 of metformin treatment

• Change in advanced glycation end products (AGE)

  Baseline through week 14 of metformin treatment

• Change in sweat chloride

  Baseline through week 14 of metformin treatment

• Change in lung function

  Baseline through week 14 of metformin treatment

Study Arms (1)

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.

Drug: Metformin Hydrochloride

Interventions

Metformin Hydrochloride DRUG

1000 mg twice daily

Also known as: Glucophage

Metformin dose regimen A

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• University of Kansas Medical Center: lead

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Metformin

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Biguanides; Guanidines; Amidines; Organic Chemicals

Study Officials

• Matthias A Salathe, M.D.

  Professor

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Matthias A Salathe, M.D.

CONTACT

9135886000; msalathe@kumc.edu

Carolina Aguiar

CONTACT

9139459295; caguiar@kumc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 24, 2020
• First Posted: August 28, 2020
• Study Start: February 14, 2022
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): July 15, 2028
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)