Acetazolamide for Type 1 Diabetes

Overseen By Todd May, MS

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of California, San Diego

Must not be taking: Diuretics

Disqualifiers: Liver disease, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 10 weeks

25 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether acetazolamide can benefit kidneys in people with Type 1 Diabetes while minimizing side effects. Participants will begin with either acetazolamide or a placebo, then switch after ten weeks. The goal is to understand acetazolamide's impact on kidney function. Individuals with Type 1 Diabetes who have been stable on their medication for at least three months might be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot use certain types of diuretics (medications that help remove excess water from the body). It's best to discuss your specific medications with the study team.

Is there any evidence suggesting that acetazolamide is likely to be safe for humans?

Research has shown that acetazolamide is usually safe for people with type 1 diabetes. In studies, it temporarily reduced kidney function, but this change was reversible. Importantly, it did not affect blood sugar levels.

These findings suggest that while acetazolamide might temporarily alter kidney function, it doesn't appear to cause lasting harm. The treatment remains under investigation, and researchers continue to study its safety. This ongoing research helps ensure a thorough understanding of any possible side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Type 1 Diabetes?

Most treatments for Type 1 Diabetes focus on insulin replacement or regulating blood sugar levels. However, Acetazolamide is unique because it targets a different mechanism: it influences the body's acid-base balance and can have anti-inflammatory effects. Researchers are excited about Acetazolamide because it might offer a new way to protect and preserve insulin-producing cells in the pancreas, potentially reducing the need for insulin injections. This approach could provide a novel and complementary strategy to manage Type 1 Diabetes beyond conventional therapies.

What evidence suggests that acetazolamide might be an effective treatment for Type 1 Diabetes?

This trial will compare the effects of acetazolamide on kidney health in people with Type 1 Diabetes. Research has shown that acetazolamide may protect the kidneys by temporarily lowering their blood filtration rate, potentially reducing stress. Once the drug is stopped, the kidneys return to normal function. Importantly, acetazolamide does not affect blood sugar levels. Early results from studies in both mice and humans support these findings. Therefore, acetazolamide appears promising for kidney health in Type 1 Diabetes. Participants in this trial will receive acetazolamide in different sequences to evaluate its effectiveness. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for people with Type 1 Diabetes who are interested in how a drug called acetazolamide might help their kidneys. The details on specific eligibility criteria are not provided, but typically participants would need to meet certain health standards and not be taking conflicting medications.

Inclusion Criteria

eGFR (estimated glomerular filtration rate) ≥ 45ml/min/1.73m2

Negative urine toxicology screen

Able to provide written informed consent approved by an Institutional Review Board (IRB)

Exclusion Criteria

I have liver disease or drink more than 14 alcoholic beverages a week.

Serum hemoglobin A1c > 10.0%

Serum hemoglobin concentration of <8 g/dL

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 250mg acetazolamide or placebo twice daily for 10 weeks in a crossover design

10 weeks

Regular visits for drug administration and monitoring

Washout

Participants receive no study drug for 2 weeks between treatment periods

2 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Acetazolamide

Trial Overview The study is testing the effects of acetazolamide versus a placebo on kidney function in individuals with Type 1 Diabetes. It's a crossover trial, meaning each participant will receive both the drug and the placebo at different times to compare results.

How Is the Trial Designed?

Group I: Treatment Group B Experimental Treatment 2 Interventions

Placebo Comparator administered twice a day for 10 weeks followed by 250mg Acetazolamide administered twice a day for 10 weeks.

Group II: Treatment Group A Experimental Treatment 2 Interventions

250mg Acetazolamide administered twice a day for 10 weeks followed by Placebo Comparator administered twice a day for 10 weeks.

Who Is Running the Clinical Trial?

University of California, San Diego

Lead Sponsor

Trials

1,215

Recruited

1,593,000+

Breakthrough T1D

Collaborator

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39466253/

Acetazolamide Therapy and Kidney Function in Persons with ... Low-dose acetazolamide reversibly lowered GFR in persons with type 1 diabetes mellitus, suggesting a possible role in relieving glomerular ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05473364

Acetazolamide in Persons With Type 1 Diabetes - Dose ... This is a dose finding trial where participants will receive escalating doses of acetazolamide, each for a 2-week dosing period followed by a 2-week washout ...

3. journals.lww.com journals.lww.com/jasn/fulltext/2025/03000/acetazolamide_therapy_and_kidney_function_in.16.aspx

Acetazolamide Therapy and Kidney Function in Persons ... Low-dose acetazolamide reversibly lowered GFR in persons with type 1 diabetes mellitus, suggesting a possible role in relieving glomerular hyperfiltration. Low ...

4. breakthrought1d.org breakthrought1d.org/grants/united-states/california/acetazolamide-and-tubuloglomerular-feedback-in-persons-with-type-1-diabetes-a-clinical-trial/

Acetazolamide and Tubuloglomerular Feedback in ... Preliminary studies in mice, as well as in humans with T1DM demonstrate that acetazolamide can lower glomerular pressure. However, these studies have all been ...

5. healio.com healio.com/news/nephrology/20241010/acetazolamide-may-reduce-gfr-without-impacting-glucose-metabolism-in-type-1-diabetes

Acetazolamide may reduce GFR without impacting ... Acetazolamide was associated with an acute, reversible drop in GFR without impacting glucose metabolism for adults with type 1 diabetes and preserved kidney ...