What side effects are associated with this type of intervention?

Common treatments for cystinuria include increased fluid intake, dietary modifications, and medications such as thiol drugs (e.g., tiopronin and penicillamine) that help dissolve cystine stones. These medications can have side effects such as gastrointestinal upset, rash, and, in rare cases, more severe reactions like bone marrow suppression. SGLT-2 inhibitors, which are being studied for their potential use in cystinuria, are primarily used to treat diabetes and heart failure. Known side effects of SGLT-2 inhibitors include urinary tract infections, genital infections, dehydration, and, in rare cases, diabetic ketoacidosis. While the exact efficacy and safety of SGLT-2 inhibitors for cystinuria are still under investigation, these side effects should be considered.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically for cystinuria that utilize SGLT-2 inhibitors to increase glucose concentration in urine to prevent or reverse cystine stone formation. The mainstay of therapy for cystinuria involves breaking or preventing the cystine bond at the molecular level. While SGLT-2 inhibitors are FDA-approved for diabetes mellitus and heart failure, their use in cystinuria remains hypothetical and under investigation, with no published data confirming their effectiveness for this condition.

What other types of research exist?

As of now, there are no specific novel alternatives to SGLT-2 inhibitors explicitly mentioned for cystinuria treatment in the provided context. However, ongoing research into other molecularly targeted therapies or advancements in nonpharmacologic strategies, such as dietary modifications and increased fluid intake, may offer potential alternatives. Patients should consult with their healthcare provider to explore the most current and effective treatment options available in 2024.

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SGLT-2 Inhibitor

SGLT-2 Inhibitor for Cystinuria

Phase 2

Waitlist Available

Led By Marshall Stoller, MD

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

history of previous cystine kidney stones

History of previous cystine kidney stones

Must not have

Unable to give informed consent

Prior diagnosis of diabetes mellitus (type I or type II)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year and 3 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests whether taking dapagliflozin daily can help people with cystinuria, a genetic kidney disorder that causes painful kidney stones. Dapagliflozin increases sugar levels in the urine, which may help stop or break down the stones. The study will monitor patients to see if this treatment works.

Who is the study for?

This trial is for adults over 18 with a history of cystine kidney stones and confirmed cystinuria. Participants must be able to consent and not have diabetes, be pregnant or likely to become so without birth control, seriously ill, non-English speaking, incarcerated, or unable to give informed consent.

What is being tested?

The trial tests if Dapagliflozin, an FDA-approved drug for diabetes and heart failure that increases urine glucose levels can prevent or dissolve cystine stones in the kidneys by introducing enough glucose into the urine.

What are the potential side effects?

While not specified here, common side effects of SGLT-2 inhibitors like Dapagliflozin include yeast infections due to sugar in the urine, urinary tract infections (UTIs), increased urination and risk of dehydration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have had cystine kidney stones in the past.

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I have had cystine kidney stones in the past.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am unable to understand and agree to the study's procedures and risks.

Select...

I have been diagnosed with diabetes (type I or II).

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My primary language is not English.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year and 3 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year and 3 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year and 3 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in cystine stone size over time (1 year and 3 months)

Side effects data

From 2018 Phase 4 trial • 30 Patients • NCT03006471

20%

Cervicovaginal infection

13%

Urinary tract infections

100%

80%

60%

40%

20%

Study treatment Arm

Dapagliflozin

Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Study DrugExperimental Treatment1 Intervention

The study drug is Dapagliflozin

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Dapagliflozin

2014

Completed Phase 4

~64440

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Cystinuria is treated by preventing the formation of cystine stones in the kidneys. One common approach is to break or prevent the cystine bond at the molecular level, making it less likely to precipitate in the urine. SGLT-2 inhibitors, which are being studied for this purpose, work by increasing the concentration of glucose in the urine. This increased glucose may help prevent or reverse the formation of cystine stones by interfering with cystine's ability to form crystals. This mechanism is crucial for cystinuria patients as it offers a potential non-invasive treatment option to manage and reduce the recurrence of painful kidney stones.

Treatment of clinically normal and cystinuric dogs with 2-mercaptopropionylglycine.